☆ 4.1 Review

Innovations in the Treatment of Dystrophic Epidermolysis Bullosa (DEB): Current Landscape and Prospects

THERAPEUTICS AND CLINICAL RISK MANAGEMENT (2023)

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Article Dermatology

Dupilumab zur Behandlung von Genodermatosen: Eine systematische Übersicht

Po‐Chien Wu et al.

JOURNAL DER DEUTSCHEN DERMATOLOGISCHEN GESELLSCHAFT (2023)

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Article Pediatrics

Our experience of using Losartan for esophageal stenosis in children with dystrophic form of congenital epidermolysis bullosa

Vladislav Oldakovskiy et al.

Summary: This study evaluated the efficacy of losartan in preventing restenosis after balloon dilatation (BD) for esophageal stenosis in children with dystrophic epidermolysis bullosa (DEB). The results showed that children treated with losartan had a lower rate of requiring repeat dilatation and lower indicators of nutritional deficiency and disease severity. Losartan demonstrated safety and contributed to an improvement in the nutritional status of children with DEB, reducing the frequency of restenosis.

JOURNAL OF PEDIATRIC SURGERY (2023)

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Article Dermatology

Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa: results from the phase III randomized double-blind phase of the EASE study

Johannes S. Kern et al.

Summary: The EASE study aimed to determine whether daily use of Oleogel-S10 could accelerate wound healing in epidermolysis bullosa (EB). The results showed that patients treated with Oleogel-S10 had a higher proportion of first complete closure of target wounds within 45 days compared to the control group (41.3% vs 28.9%). The efficacy of Oleogel-S10 in accelerating EB wound healing was confirmed and it has been approved by the European Commission for the treatment of wounds in EB.

BRITISH JOURNAL OF DERMATOLOGY (2023)

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Article Dermatology

Interleukin-17A immune pattern across genetic acantholytic and blistering disorders

Asal Haghighi Javid et al.

CLINICAL AND EXPERIMENTAL DERMATOLOGY (2023)

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Article Dermatology

Dystrophic Epidermolysis Bullosa Pruriginosa: Successfully Treated With Dupilumab

Ling Yu et al.

Dermatitis (2023)

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Article

Control del prurito refractario en epidermólisis ampollosa pruriginosa con dupilumab

B. Roque Quintana et al.

Actas Dermo-Sifiliograficas (2023)

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Article Biochemistry & Molecular Biology

COL7A1 Editing via RNA Trans-Splicing in RDEB-Derived Skin Equivalents

Bernadette Liemberger et al.

Summary: Mutations in the COL7A1 gene can cause skin problems by affecting type VII collagen (C7) in the basement membrane zone. The researchers used an RNA therapy called spliceosome-mediated RNA trans-splicing (SMaRT) to correct mutations in COL7A1. They successfully corrected mutations in RDEB keratinocytes and RDEB skin equivalents by introducing a repair molecule called RTM-S6m.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2023)

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Article Dermatology

The epidemiology of epidermolysis bullosa in England and Wales: data from the national epidermolysis bullosa database

Gabriela Petrof et al.

Summary: This study provides accurate epidemiological data for EB in England and Wales, showing a decrease in the birth incidence of severe types of EB, possibly due to the uptake of genetic counseling advice, while the decrease in milder types of EB may be related to delayed presentation. There is a slight trend towards longer survival in babies with JEB-S, which may be attributed to improved multidisciplinary care.

BRITISH JOURNAL OF DERMATOLOGY (2022)

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Letter Dermatology

Successful use of tofacitinib in epidermolysis bullosa pruriginosa

K. -J. Chen et al.

CLINICAL AND EXPERIMENTAL DERMATOLOGY (2022)

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Article Dermatology

Transcriptomic profiling of recessive dystrophic epidermolysis bullosa wounded skin highlights drug repurposing opportunities to improve wound healing

Alexandros Onoufriadis et al.

Summary: This study identified gene expression profiles and pathways in RDEB wounds, identifying potential drugs or compounds to restore wound healing. The findings highlight the clinical value of reverse transcriptomics data analysis in RDEB and the potential of this approach in discovering or repurposing drugs for other diseases.

EXPERIMENTAL DERMATOLOGY (2022)

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Article Biochemistry & Molecular Biology

5′RNA Trans-Splicing Repair of COL7A1 Mutant Transcripts in Epidermolysis Bullosa

Elisabeth Mayr et al.

Summary: This study demonstrates the potential of endogenous 5' trans-splicing to correct mutations in the COL7A1 gene and modulate the RDEB phenotype, addressing the needs of patients.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)

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Letter Dermatology

A case of dystrophic epidermolysis bullosa pruriginosa treated with dupilumab

F. Caroppo et al.

JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY (2022)

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Article Genetics & Heredity

The clinical efficacy and safety of anti-IgE therapy in recessive dystrophic epidermolysis bullosa

Fuying Chen et al.

Summary: Patients with recessive dystrophic epidermolysis bullosa (RDEB) and elevated IgE levels, especially those with intense pruritus, showed significant clinical improvement with anti-IgE therapy, including enhanced wound healing, reduction in disease severity, and amelioration of skin inflammation. IgE may play a potential role in the pathogenesis of inflammatory conditions associated with RDEB, as indicated by this study.

CLINICAL GENETICS (2022)

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Article Dermatology

Losartan treatment improves recessive dystrophic epidermolysis bullosa: A case series

Mohammad Reza Pourani et al.

Summary: This case series reports the beneficial effects of losartan on RDEB as a potentially novel treatment. Losartan can improve the clinical features and severity of the disease, and enhance the quality of life for patients with RDEB.

DERMATOLOGIC THERAPY (2022)

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Article Dermatology

Dystrophic epidermolysis bullosa pruriginosa: a new case series of a rare phenotype unveils skewed Th2 immunity

D. Darbord et al.

Summary: The study confirmed the stereotyped presentation of DEB-Pr with significant intra-familial variability in disease expression. Mast cell infiltration, elevated IgE levels, and increased Th2 subset without atopy strongly support a role of Th2-mediated immunity in DEB-Pr.

JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY (2022)

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Article Biochemistry & Molecular Biology

In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial

Irina Gurevich et al.

Summary: This study evaluated the use of an engineered viral vector, B-VEC, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) patients with wounds. The results showed that B-VEC promotes wound healing and is well-tolerated.

NATURE MEDICINE (2022)

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Article Dermatology

Molecular and Clinical Outcomes After Intravenous Gentamicin Treatment for Patients With Junctional Epidermolysis Bullosa Caused by Nonsense Variants

Daniel Mosallaei et al.

Summary: This study evaluated the safety and efficacy of intravenous gentamicin readthrough therapy in JEB patients. The results showed that gentamicin treatment induced readthrough of nonsense variants, restored functional laminin 332 expression, and promoted wound healing and closure in a 3-month period.

JAMA DERMATOLOGY (2022)

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Article Dermatology

Evaluation of Systemic Gentamicin as Translational Readthrough Therapy for a Patient With Epidermolysis Bullosa Simplex With Muscular Dystrophy Owing to PLEC1 Pathogenic Nonsense Variants

Lucia Martinez-Santamaria et al.

Summary: This case report focused on a woman with EBS-MD who received gentamicin treatment for two courses, showing improvements in mucocutaneous involvement, muscle weakness, and quality of life. Laboratory tests and clinical scales showed positive outcomes, with gentamicin treatment leading to plectin expression in the skin for at least 5 months, and improvements in skeletal and respiratory muscle function.

JAMA DERMATOLOGY (2022)

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Article Medicine, General & Internal

Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa

Shireen V. V. Guide et al.

Summary: Topical administration of B-VEC was more effective in achieving complete wound healing at 3 and 6 months in patients with dystrophic epidermolysis bullosa compared to placebo. Patients treated with B-VEC experienced pruritus and mild systemic side effects. Longer and larger trials are needed to evaluate the durability and side effects of B-VEC for this disease.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

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Article Multidisciplinary Sciences

The C4EB study-Transvamix (10% THC / 5% CBD) to treat chronic pain in epidermolysis bullosa: A protocol for an explorative randomized, placebo controlled, and double blind intervention crossover study

Nicholas H. B. Schraeder et al.

Summary: This study aims to evaluate the efficacy of cannabinoid-based medicines in patients with epidermolysis bullosa (EB) in reducing pain. The study design is a randomized, double-blind, placebo-controlled crossover trial, using pain scores and self-efficacy as primary outcomes. Additionally, functional magnetic resonance imaging (fMRI) will be used to assess brain connectivity related to pain circuits.

PLOS ONE (2022)

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Letter Dermatology

Losartan as therapy for recessive dystrophic epidermolysis bullosa: Report of three cases

Maria Relvas et al.

DERMATOLOGIC THERAPY (2022)

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Article Multidisciplinary Sciences

ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation

Adam Sheriff et al.

Summary: This study demonstrates the use of the adenine base editor ABE8e to correct gene mutations in RDEB patient fibroblasts. The results show efficient correction of the pathogenic allele and restoration of protein expression. This research lays the foundation for developing base editing strategies to treat RDEB patients.

SCIENTIFIC REPORTS (2022)

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Article Dermatology

295 Collagen 7 (C7) protein replacement therapy (PTR-01) durably reduces wound size and symptoms in patients with recessive dystrophic epidermolysis bullosa (RDEB)

A. Bruckner et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2022)

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Article Biotechnology & Applied Microbiology

Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa

Sung-Ah Hong et al.

Summary: In this study, researchers identified and corrected pathogenic mutations in the COL7A1 gene using adenine base editors and prime editors in patients with recessive dystrophic epidermolysis bullosa (RDEB). The edited patient-derived skin equivalents showed positive outcomes in terms of C7 deposition and anchoring fibril formation, suggesting the feasibility of ex vivo gene editing as a potential treatment for RDEB.

MOLECULAR THERAPY (2022)

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Article Cell Biology

Mesenchymal Stem Cell-Derived Extracellular Vesicles as an Advanced Therapy for Chronic Wounds

Eric R. Bray et al.

Summary: Chronic wounds pose a significant challenge, and the use of mesenchymal stem cell (MSC) therapies and their secreted extracellular vesicles (EVs) show promise in promoting healing. This review explores the targeting of MSC-EVs to the underlying processes of chronic wounds and highlights the potential for optimizing their use in patient care.

COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY (2022)

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Article Genetics & Heredity

Clinical characteristics, healthcare use, and annual costs among patients with dystrophic epidermolysis bullosa

James A. Feinstein et al.

Summary: This study comprehensively examined the diagnoses, healthcare use, and annual costs in patients with dystrophic epidermolysis bullosa (DEB). The study found that DEB patients have various comorbidities, multiple healthcare visits, prescription medications, and high treatment costs.

ORPHANET JOURNAL OF RARE DISEASES (2022)

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Article Genetics & Heredity

Understanding the socioeconomic costs of dystrophic epidermolysis bullosa in Europe: a costing and health-related quality of life study

A. Angelis et al.

Summary: This study analyzed the burden of DEB in Europe and found that the economic costs for DEB patients are higher than the overall EB patient population, mainly due to high direct non-medical costs, with the majority of patients requiring support from caregivers at home.

ORPHANET JOURNAL OF RARE DISEASES (2022)

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Article Genetics & Heredity

Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa

Jodi Y. So et al.

Summary: This Phase 1/2a open-label study evaluated the long-term efficacy and safety of gene-corrected autologous keratinocyte grafts (EB-101) for chronic RDEB wounds. The results showed that treatment with EB-101 appears to be safe and effective, producing long-term improvements in wound healing, pain, and itch for RDEB patients.

ORPHANET JOURNAL OF RARE DISEASES (2022)

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Article Dermatology

Improved erythema and decreased blister formation in dominant dystrophic epidermolysis bullosa following treatment with pulsed dye laser

Anna Cristina Garza-Mayers et al.

Summary: This case report describes a patient with DDEB who showed significant improvement in erythema and had fewer and less severe blistering episodes after treatment with PDL.

PEDIATRIC DERMATOLOGY (2022)

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Article Multidisciplinary Sciences

The C4EB study-Transvamix (10% THC/5% CBD) to treat chronic pain in epidermolysis bullosa: A protocol for an explorative randomized, placebo controlled, and double blind intervention crossover study

Nicholas H. B. Schrader et al.

Summary: This study aims to investigate the use of cannabinoid-based medicines for alleviating pain symptoms in patients with epidermolysis bullosa. Functional magnetic resonance imaging will be used to assess the brain's pain circuits, and secondary outcomes such as pain self-efficacy and adverse events will also be measured.

PLOS ONE (2022)

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Article Dermatology

Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds

C. L. Ebens et al.

Summary: This study evaluated the effects of allogeneic epidermal skin grafts on wound healing and durability in patients with RDEB. The results showed significant reductions in wound area, successful fusion of epidermal grafts, and improvement in RDEB pseudosyndactyly.

BRITISH JOURNAL OF DERMATOLOGY (2021)

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Review Dermatology

Prevalence, pathophysiology and management of itch in epidermolysis bullosa

M. Papanikolaou et al.

Summary: Epidermolysis bullosa (EB) is a group of inherited skin disorders caused by mutations in genes encoding proteins of the dermoepidermal junction. Itch is a common symptom in EB and may be due to skin inflammation, wound healing cascades, and dysregulated activation of sensory nerve endings. Understanding these mechanisms is crucial for optimizing treatments and improving quality of life for patients with EB.

BRITISH JOURNAL OF DERMATOLOGY (2021)

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Article Dermatology

QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study

Olivier Bornert et al.

Summary: Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII, with recessive DEB (RDEB) and dominant DEB (DDEB) being the main types. The antisense oligonucleotide QR-313, targeting exon 73, shows promise for treating both RDEB and DDEB, restoring collagen VII levels in the skin.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2021)

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Article Dermatology

Epidermolysis bullosa pruriginosa treated with dupilumab

Amanda G. Zhou et al.

Summary: These two cases demonstrate the effectiveness of dupilumab in treating patients with EBP, improving pruritus symptoms and skin findings, and helping patients enhance their quality of life. However, sometimes an increased dosage is needed to achieve optimal control of the disease.

PEDIATRIC DERMATOLOGY (2021)

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Article Medicine, Research & Experimental

Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients

Sang Eun Lee et al.

Summary: This study evaluated the safety and potential clinical efficacy of intravenous infusion of allogeneic human umbilical cord blood-derived mesenchymal stem cells in patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). The results showed that the treatment was well tolerated and led to transient clinical benefits, including improvements in wound conditions, pain, itch, and quality of life. Inflammatory skin conditions and C7 expression also showed some improvement after treatment.

JCI INSIGHT (2021)

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Review Biochemistry & Molecular Biology

Applications of Mesenchymal Stem Cells in Skin Regeneration and Rejuvenation

Hantae Jo et al.

Summary: MSCs play a key role in promoting repair of damaged and aging skin by increasing cell proliferation, neovascularization, collagen and elastic fiber production. iPSC-derived MSCs are considered advanced MSCs for cell therapy.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Letter Dermatology

A case of recessive dystrophic epidermolysis bullosa treated with a cultured epidermal autograft

Ryosuke Mochizuki et al.

JOURNAL OF DERMATOLOGY (2021)

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Article Genetics & Heredity

Impact of low-dose calcipotriol ointment on wound healing, pruritus and pain in patients with dystrophic epidermolysis bullosa: A randomized, double-blind, placebo-controlled trial

Christina Guttmann-Gruber et al.

Summary: In this study, the efficacy and safety of low-dose calcipotriol, a vitamin D3 analogue, in promoting wound healing and reducing itch and pain in patients with dystrophic epidermolysis bullosa (DEB) were evaluated. The results showed that topical treatment with low-dose calcipotriol can accelerate wound closure and significantly reduce itch, making it a safe and readily-available option to improve local wound care in DEB patients.

ORPHANET JOURNAL OF RARE DISEASES (2021)

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Letter Dermatology

Intravenous allogeneic multilineage-differentiating stress-enduring cells in adults with dystrophic epidermolysis bullosa: a phase 1/2 open-label study

Y. Fujita et al.

JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY (2021)

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Review Biochemistry & Molecular Biology

Impaired Wound Healing, Fibrosis, and Cancer: The Paradigm of Recessive Dystrophic Epidermolysis Bullosa

Grace Tartaglia et al.

Summary: RDEB is a devastating skin blistering disease caused by mutations in the C7 gene, leading to rapid fibrosis development, chronic wounds, and squamous cell carcinoma. It serves as a model for understanding the molecular basis of fibrosis and rapidly developing aggressive cancer.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Review Pharmacology & Pharmacy

Antisense Oligonucleotide-Based Therapy of Viral Infections

Woan-Yuh Tarn et al.

Summary: Nucleic acid-based therapeutics, particularly Antisense Oligonucleotides (ASOs), have shown effectiveness in treating genetic disorders, cancer, and viral infections. ASOs can be quickly developed to target newly emerging viruses and their variants, making them a promising tool in antiviral therapy.

PHARMACEUTICS (2021)

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Review Cell & Tissue Engineering

Mesenchymal stromal cells in wound healing applications: role of the secretome, targeted delivery and impact on recessive dystrophic epidermolysis bullosa treatment

Julia Riedl et al.

Summary: Mesenchymal stromal cells (MSCs) are promising candidates for tissue engineering and regenerative medicine applications in wound healing due to their self-renewal and regenerative capacities. However, clinical studies have shown variable results, prompting further research into the specific mechanisms of MSCs in influencing the local microenvironment, particularly through their secretome.

CYTOTHERAPY (2021)

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Letter Dermatology

Amelioration of dystrophic epidermolysis bullosa pruriginosa symptoms with dupilumab: A case report

Yangbin Wang et al.

DERMATOLOGIC THERAPY (2021)

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Article Genetics & Heredity

Cannabinoid use and effects in patients with epidermolysis bullosa: an international cross-sectional survey study

Nicholas H. B. Schrader et al.

Summary: The study evaluated the use of cannabinoid-based medicines (CBMs) among EB patients and found that most patients reported a decrease in pain and pruritus after using CBMs, as well as improvements in overall symptoms and wound healing. Additionally, a majority of participants reported reduced use of pain medications. However, further controlled studies are needed to determine the risks and benefits of using CBMs in EB patients.

ORPHANET JOURNAL OF RARE DISEASES (2021)

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Letter Dermatology

Epidermolysis Bullosa Pruriginosa Treated With Baricitinib

Xingyuan Jiang et al.

JAMA DERMATOLOGY (2021)

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Article Cell & Tissue Engineering

ABCB5+dermal mesenchymal stromal cells with favorable skin homing and local immunomodulation for recessive dystrophic epidermolysis bullosa treatment

Julia Riedl et al.

Summary: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare, incurable skin disease caused by biallelic mutations in type VII collagen (C7), and advancements in treatment have been made by utilizing the immunomodulatory potential of mesenchymal stem cells (MSCs). A unique subset of skin-derived MSCs expressing ABCB5 have superior skin homing ability and may induce wound repair through increased expression of HOXA3 gene. Further exploration of the immunomodulatory mechanisms among MSC populations may have broader implications in regenerative medicine beyond RDEB treatment.

STEM CELLS (2021)

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Article Dermatology

Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs

Mark J. Osborn et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2020)

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Article Dermatology

Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa

Ellie Rashidghamat et al.

JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY (2020)

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Review Dermatology

Consensus reclassification of inherited epidermolysis bullosa and other disorders with skin fragility

C. Has et al.

BRITISH JOURNAL OF DERMATOLOGY (2020)

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Letter Dermatology

Treatment of epidermolysis bullosa pruriginosa-associated pruritus with dupilumab

W. Shehadeh et al.

BRITISH JOURNAL OF DERMATOLOGY (2020)

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Article Multidisciplinary Sciences

High-mobility group box 1 fragment suppresses adverse post-infarction remodeling by recruiting PDGFRα-positive bone marrow cells

Takasumi Goto et al.

PLOS ONE (2020)

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Article Biotechnology & Applied Microbiology

Exposed CendR Domain in Homing Peptide Yields Skin-Targeted Therapeutic in Epidermolysis Bullosa

Toini Pemmari et al.

MOLECULAR THERAPY (2020)

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Review Cell Biology

RNA delivery by extracellular vesicles in mammalian cells and its applications

Killian O'Brien et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2020)

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Article Dermatology

306 Topical QR-313, an Antisense Oligonucleotide, in the Treatment of Dystrophic Epidermolysis Bullosa

M.P. Marinkovich et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2020)

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Article Biochemistry & Molecular Biology

Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1

Kristin A. Ham et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Article Medicine, General & Internal

Epidermolysis bullosa

Ajoy Bardhan et al.

NATURE REVIEWS DISEASE PRIMERS (2020)

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Letter Dermatology

Successful use of cyclosporine in epidermolysis bullosa pruriginosa

Akanksha Kaushik et al.

DERMATOLOGIC THERAPY (2020)

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Article Surgery

Extracellular Vesicles, Apoptotic Bodies and Mitochondria: Stem Cell Bioproducts for Organ Regeneration

Natalia Gebara et al.

CURRENT TRANSPLANTATION REPORTS (2020)

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Article Dermatology

Epidermolysis bullosa simplex generalized severe induces a T helper 17 response and is improved by apremilast treatment

E. Castela et al.

BRITISH JOURNAL OF DERMATOLOGY (2019)

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Review Plant Sciences

The Wound Healing Properties of Betulin from Birch Bark from Bench to Bedside

Armin Scheffler

PLANTA MEDICA (2019)

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Article Dermatology

Revertant mosaic fibroblasts in recessive dystrophic epidermolysis bullosa

K. Twaroski et al.

BRITISH JOURNAL OF DERMATOLOGY (2019)

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Article Dermatology

1021 Intravenous gentamicin therapy for recessive dystrophic epidermolysis bullosa patients harboring nonsense mutations

D. Woodley et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2019)

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Review Dermatology

Mechanism of Oleogel-S10: A triterpene preparation for the treatment of epidermolysis bullosa

Agnes Schwieger-Briel et al.

DERMATOLOGIC THERAPY (2019)

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Review Cell & Tissue Engineering

The Clinical Trials of Mesenchymal Stem Cell Therapy in Skin Diseases: An Update and Concise Review

Ali Golchin et al.

CURRENT STEM CELL RESEARCH & THERAPY (2019)

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Article Multidisciplinary Sciences

Search-and-replace genome editing without double-strand breaks or donor DNA

Andrew V. Anzalone et al.

NATURE (2019)

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Article Dermatology

Combined tetrahydrocannabinol and cannabidiol to treat pain in epidermolysis bullosa: a report of three cases

N. H. B. Schrader et al.

BRITISH JOURNAL OF DERMATOLOGY (2019)

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Editorial Material Dermatology

Therapies for epidermolysis bullosa: delivery is key

R. F. L. O'Shaughnessy

BRITISH JOURNAL OF DERMATOLOGY (2019)

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Article Biochemistry & Molecular Biology

Decorin counteracts disease progression in mice with recessive dystrophic epidermolysis bullosa

Francesca Cianfarani et al.

MATRIX BIOLOGY (2019)

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Article Multidisciplinary Sciences

CRISPR/Cas9-based targeted genome editing for correction of recessive dystrophic epidermolysis bullosa using iPS cells

Joanna Jackow et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

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Article Medicine, Research & Experimental

Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa

Su M. Lwin et al.

JCI INSIGHT (2019)

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Article Medicine, Research & Experimental

Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa

Shaundra Eichstadt et al.

JCI INSIGHT (2019)

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Article Biochemistry & Molecular Biology

Dual mechanism of type VII collagen transfer by bone marrow mesenchymal stem cell extracellular vesicles to recessive dystrophic epidermolysis bullosa fibroblasts

Jeffrey D. McBride et al.

BIOCHIMIE (2018)

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Article Dermatology

Cultured allogeneic fibroblast injection vs. fibroblasts cultured on amniotic membrane scaffold for dystrophic epidermolysis bullosa treatment

H. Moravvej et al.

BRITISH JOURNAL OF DERMATOLOGY (2018)

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Meeting Abstract Dermatology

A phase 1/2 study of genetically-corrected, collagen VII expressing autologous human dermal fibroblasts injected into the skin of patients with recessive dystrophic epidermolysis bullosa (RDEB)

M. Marinkovich et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2018)

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Article Dermatology

EB2017-Progress in Epidermolysis Bullosa Research toward Treatment and Cure

Jouni Uitto et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2018)

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Article Dermatology

Self-initiated use of topical cannabidiol oil for epidermolysis bullosa

Malcolm P. Chelliah et al.

PEDIATRIC DERMATOLOGY (2018)

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Article Multidisciplinary Sciences

Impaired lymphoid extracellular matrix impedes antibacterial immunity in epidermolysis bullosa

Alexander Nystroem et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)

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Review Immunology

High-mobility group box 1 protein (HMGB1) operates as an alarmin outside as well as inside cells

Ulf Andersson et al.

SEMINARS IN IMMUNOLOGY (2018)

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Article Medicine, Research & Experimental

Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

Araksya Izmiryan et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

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Article Multidisciplinary Sciences

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

Westley J. Friesen et al.

PLOS ONE (2018)

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Article Multidisciplinary Sciences

Low-dose calcipotriol can elicit wound closure, anti-microbial, and anti-neoplastic effects in epidermolysis bullosa keratinocytes

Christina Guttmann-Gruber et al.

SCIENTIFIC REPORTS (2018)

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Review Genetics & Heredity

Base editing: precision chemistry on the genome and transcriptome of living cells

Holly A. Rees et al.

NATURE REVIEWS GENETICS (2018)

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Article Multidisciplinary Sciences

The administration of high-mobility group box 1 fragment prevents deterioration of cardiac performance by enhancement of bone marrow mesenchymal stem cell homing in the delta-sarcoglycan-deficient hamster

Takashi Kido et al.

PLOS ONE (2018)

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Article Cell Biology

Minimal information for studies of extracellular vesicles 2018 (MISEV2018): a position statement of the International Society for Extracellular Vesicles and update of the MISEV2014 guidelines

Clotilde Thery et al.

JOURNAL OF EXTRACELLULAR VESICLES (2018)

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Review Dermatology

RNA-based therapies for genodermatoses

Olivier Bornert et al.

EXPERIMENTAL DERMATOLOGY (2017)

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Article Medicine, Research & Experimental

Gentamicin induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients

David T. Woodley et al.

JOURNAL OF CLINICAL INVESTIGATION (2017)

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Article Dermatology

Amlexanox Enhances Premature Termination Codon Read-Through in COL7A1 and Expression of Full Length Type VII Collagen: Potential Therapy for Recessive Dystrophic Epidermolysis Bullosa

Velina S. Atanasova et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2017)

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Article Medicine, Research & Experimental

Rapid generation of Col7a1-/- mouse model of recessive dystrophic epidermolysis bullosa and partial rescue via immunosuppressive dermal mesenchymal stem cells

Beau R. Webber et al.

LABORATORY INVESTIGATION (2017)

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Article Multidisciplinary Sciences

Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage

Nicole M. Gaudelli et al.

NATURE (2017)

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Article Dermatology

Betulin-Based Oleogel to Improve Wound Healing in Dystrophic Epidermolysis Bullosa: A Prospective Controlled Proof-of-Concept Study

Agnes Schwieger-Briel et al.

DERMATOLOGY RESEARCH AND PRACTICE (2017)

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Article Dermatology

Treatment of dystrophic epidermolysis bullosa with bone marrow non-hematopoeitic stem cells: a randomized controlled trial

Mohammad El-Darouti et al.

DERMATOLOGIC THERAPY (2016)

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Article Medicine, General & Internal

Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa

Zurab Siprashvili et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2016)

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Article Dermatology

Targeted Exon Skipping Restores Type VII Collagen Expression and Anchoring Fibril Formation in an In Vivo RDEB Model

Sandrina Turczynski et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2016)

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Article Biotechnology & Applied Microbiology

Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy

Olivier Bornert et al.

MOLECULAR THERAPY (2016)

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Article Multidisciplinary Sciences

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

Alexis C. Komor et al.

NATURE (2016)

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Article Multidisciplinary Sciences

Site-specific genome editing for correction of induced pluripotent stem cells derived from dominant dystrophic epidermolysis bullosa

Satoru Shinkuma et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

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Article Medicine, Research & Experimental

Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa

Jeroen Bremer et al.

Molecular Therapy-Nucleic Acids (2016)

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Article Medicine, Research & Experimental

Losartan ameliorates dystrophic epidermolysis bullosa and uncovers new disease mechanisms

Alexander Nystroem et al.

EMBO MOLECULAR MEDICINE (2015)

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Article Dermatology

Intravenously Administered Recombinant Human Type VII Collagen Derived from Chinese Hamster Ovary Cells Reverses the Disease Phenotype in Recessive Dystrophic Epidermolysis Bullosa Mice

Yingping Hou et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2015)

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Letter Dermatology

Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa

Gabriela Petrof et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2015)

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Article Pediatrics

Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Mark B. Geyer et al.

JOURNAL OF PEDIATRICS (2015)

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Review Cell Biology

Mesenchymal stem cell exosomes

Ruenn Chai Lai et al.

SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY (2015)

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Article Pediatrics

Case Report: Rapidly Healing Epidermolysis Bullosa Wound After Ablative Fractional Resurfacing

Andrew C. Krakowski et al.

PEDIATRICS (2015)

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Editorial Material Dermatology

Long-term Follow-up of Cultured Epidermal Autograft in a Patient with Recessive Dystrophic Epidermolysis Bullosa

Satoru Shinkuma et al.

ACTA DERMATO-VENEREOLOGICA (2014)

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Article Biochemistry & Molecular Biology

Monozygotic twins discordant for recessive dystrophic epidermolysis bullosa phenotype highlight the role of TGF-β signalling in modifying disease severity

Teresa Odorisio et al.

HUMAN MOLECULAR GENETICS (2014)

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Article Biotechnology & Applied Microbiology

Aminoglycosides Restore Full-length Type VII Collagen by Overcoming Premature Termination Codons: Therapeutic Implications for Dystrophic Epidermolysis Bullosa

Jon Cogan et al.

MOLECULAR THERAPY (2014)

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Article Dermatology

Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds: results of a randomized, vehicle-controlled trial

G. Petrof et al.

BRITISH JOURNAL OF DERMATOLOGY (2013)

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Letter Dermatology

Intravenously Injected Recombinant Human Type VII Collagen Homes to Skin Wounds and Restores Skin Integrity of Dystrophic Epidermolysis Bullosa

David T. Woodley et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2013)

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Article Dermatology

A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa

Supriya S. Venugopal et al.

JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY (2013)

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Article Medicine, General & Internal

Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix

Jakub Tolar et al.

LANCET (2013)

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Article Biotechnology & Applied Microbiology

A Reporter-Based Screen to Identify Potent 3' Trans-Splicing Molecules for Endogenous RNA Repair

Eva M. Murauer et al.

HUMAN GENE THERAPY METHODS (2013)

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Article Biochemistry & Molecular Biology

A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays

Stuart P. McElroy et al.

PLOS BIOLOGY (2013)

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Letter Dermatology

siRNA-Mediated Allele-Specific Inhibition of Mutant Type VII Collagen in Dominant Dystrophic Epidermolysis Bullosa

Valerie Pendaries et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2012)

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Review Biochemistry & Molecular Biology

Sense from nonsense: therapies for premature stop codon diseases

Laure Bidou et al.

TRENDS IN MOLECULAR MEDICINE (2012)

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Article Dermatology

Functional Correction of Type VII Collagen Expression in Dystrophic Epidermolysis Bullosa

Eva M. Murauer et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2011)

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Letter Dermatology

HB-EGF Induces COL7A1 Expression in Keratinocytes and Fibroblasts: Possible Mechanism Underlying Allogeneic Fibroblast Therapy in Recessive Dystrophic Epidermolysis Bullosa

Nikoletta Nagy et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2011)

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Article Multidisciplinary Sciences

PDGFRα-positive cells in bone marrow are mobilized by high mobility group box 1 (HMGB1) to regenerate injured epithelia

Katsuto Tamai et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)

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Article Cell & Tissue Engineering

Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa

Paulette Conget et al.

CYTOTHERAPY (2010)

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Article Biotechnology & Applied Microbiology

Long-Term Type VII Collagen Restoration to Human Epidermolysis Bullosa Skin Tissue

Zurab Siprashvili et al.

HUMAN GENE THERAPY (2010)

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Article Dermatology

Low-level laser therapy for the treatment of epidermolysis bullosa: A case report

Eliana Maria Minicucci et al.

JOURNAL OF COSMETIC AND LASER THERAPY (2010)

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Letter Dermatology

Revertant Mosaicism in Recessive Dystrophic Epidermolysis Bullosa

Noor Almaani et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2010)

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Article Biotechnology & Applied Microbiology

SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa

Matthias Titeux et al.

MOLECULAR THERAPY (2010)

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Article Medicine, General & Internal

Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa

John E. Wagner et al.

NEW ENGLAND JOURNAL OF MEDICINE (2010)

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Article Hematology

Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells

Jakub Tolar et al.

BLOOD (2009)

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Article Dermatology

Epidermolysis bullosa and the risk of life-threatening cancers: The National EB Registry experience, 1986-2006

Jo-David Fine et al.

JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY (2009)

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Article Pathology

Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance

Takenao Chino et al.

AMERICAN JOURNAL OF PATHOLOGY (2008)

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Article Dermatology

Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa

Tracy Wong et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2008)

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Article Dermatology

Targeted skipping of a single exon harboring a premature termination codon mutation: Implications and potential for gene correction therapy for selective dystrophic epidermolysis bullosa patients

Maki Goto et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2006)

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Article Dermatology

Fibroblasts show more potential as target cells than keratinocytes in COL7A1 gene therapy of dystrophic epidermolysis bullosa

Maki Goto et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2006)

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Article Biochemistry & Molecular Biology

Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa

DT Woodley et al.

NATURE MEDICINE (2004)

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Article Biochemistry & Molecular Biology

Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors

C Baldeschi et al.

HUMAN MOLECULAR GENETICS (2003)

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Article Dermatology

Similarly potent action of 1,25-dihydroxyvitamin D3 and its analogues, tacalcitol, calcipotriol, and maxacalcitol on normal human keratinocyte proliferation and differentiation

H Takahashi et al.

JOURNAL OF DERMATOLOGICAL SCIENCE (2003)

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Article Medicine, Research & Experimental

Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue

S Ortiz-Urda et al.

JOURNAL OF CLINICAL INVESTIGATION (2003)

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Article Dermatology

Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone

DT Woodley et al.

JOURNAL OF INVESTIGATIVE DERMATOLOGY (2003)

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Article Genetics & Heredity

Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa

M Chen et al.

NATURE GENETICS (2002)

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Article Biochemistry & Molecular Biology

Development and characterization of a recombinant truncated type VII collagen minigene - Implication for gene therapy of dystrophic epidermolysis bullosa

M Chen et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2000)

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