相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study
Olivier Bornert et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2021)
Fabry Disease: Molecular Basis, Pathophysiology, Diagnostics and Potential Therapeutic Directions
Ken Kok et al.
BIOMOLECULES (2021)
EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa
S. Gaucher et al.
BRITISH JOURNAL OF DERMATOLOGY (2020)
Context-Dependent Strategies for Enhanced Genome Editing of Genodermatoses
Oliver Patrick March et al.
CELLS (2020)
Footprint-free gene mutation correction in induced pluripotent stem cell (iPSC) derived from recessive dystrophic epidermolysis bullosa (RDEB) using the CRISPR/Cas9 and piggyBac transposon system
Munenari Itoh et al.
JOURNAL OF DERMATOLOGICAL SCIENCE (2020)
Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020
Cristina Has et al.
MOLECULAR DIAGNOSIS & THERAPY (2020)
Efficient Gene Reframing Therapy for Recessive Dystrophic Epidermolysis Bullosa with CRISPR/Cas9
Shota Takashima et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2019)
Talimogene laherparepvec: review of its mechanism of action and clinical efficacy and safety
Steven S. Raman et al.
IMMUNOTHERAPY (2019)
Physiological and pathological roles of kallikrein-related peptidases in the epidermis
Mari Kishibe
JOURNAL OF DERMATOLOGICAL SCIENCE (2019)
Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial
Wei-Li Di et al.
HUMAN GENE THERAPY (2019)
CRISPR Correction of Duchenne Muscular Dystrophy
Yi-Li Min et al.
ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)
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)
Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa
Su M. Lwin et al.
JCI INSIGHT (2019)
Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa
Shaundra Eichstadt et al.
JCI INSIGHT (2019)
Closure of a Large Chronic Wound through Transplantation of Gene-Corrected Epidermal Stem Cells
Johann W. Bauer et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2017)
Regeneration of the entire human epidermis using transgenic stem cells
Tobias Hirsch et al.
NATURE (2017)
Chimpanzee Adenovirus Vector Ebola Vaccine
Julie E. Ledgerwood et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
The retroviral vector family: something for everyone
Carina Elsner et al.
VIRUS GENES (2017)
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Michael F. Naso et al.
BIODRUGS (2017)
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)
Gene-Corrected Fibroblast Therapy for Recessive Dystrophic Epidermolysis Bullosa using a Self-Inactivating COL7A1 Retroviral Vector
Joanna Jackow et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2016)
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)
Adenoviral vector-based strategies against infectious disease and cancer
Chao Zhang et al.
HUMAN VACCINES & IMMUNOTHERAPEUTICS (2016)
Into the clinic: Talimogene laherparepvec (T-VEC), a first-in-class intratumoral oncolytic viral therapy
Hasan Rehman et al.
JOURNAL FOR IMMUNOTHERAPY OF CANCER (2016)
Retroviral integration: Site matters: Mechanisms and consequences of retroviral integration site selection
Jonas Demeulemeester et al.
BIOESSAYS (2015)
Long-Term Stability and Safety of Transgenic Cultured Epidermal Stem Cells in Gene Therapy of Junctional Epidermolysis Bullosa
Laura De Rosa et al.
STEM CELL REPORTS (2014)
Efficacy Trial of a DNA/rAd5 HIV-1 Preventive Vaccine
Scott M. Hammer et al.
NEW ENGLAND JOURNAL OF MEDICINE (2013)
LDL receptor and its family members serve as the cellular receptors for vesicular stomatitis virus
Danit Finkelshtein et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
Ultrastructure and molecular pathogenesis of epidermolysis bullosa
Satoru Shinkuma et al.
CLINICS IN DERMATOLOGY (2011)
Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting
Tomas Cermak et al.
NUCLEIC ACIDS RESEARCH (2011)
Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity
Chuanfeng Wu et al.
Frontiers of Medicine (2011)
Phase II Clinical Trial of Intratumoral Application of TG1042 (Adenovirus-interferon-γ) in Patients With Advanced Cutaneous T-cell Lymphomas and Multilesional Cutaneous B-cell Lymphomas
Reinhard Dummer et al.
MOLECULAR THERAPY (2010)
Genome editing with engineered zinc finger nucleases
Fyodor D. Urnov et al.
NATURE REVIEWS GENETICS (2010)
Intralesional adenovirus-mediated interleukin-2 gene transfer for advanced solid cancers and melanoma
Reinhard Dummer et al.
MOLECULAR THERAPY (2008)
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
Fulvio Mavilio et al.
NATURE MEDICINE (2006)
Bcl-2 antisense (oblimersen sodium) plus dacarbazine in patients with advanced melanoma: The oblimersen melanoma study group
Agop Y. Bedikian et al.
JOURNAL OF CLINICAL ONCOLOGY (2006)
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
FD Urnov et al.
NATURE (2005)
New recombinant serotypes of AAV vectors
GP Gao et al.
CURRENT GENE THERAPY (2005)
Adenovirus-mediated intralesional interferon-γ gene transfer induces tumor regressions in cutaneous lymphomas
R Dummer et al.
BLOOD (2004)
Current developments in the design of onco-retrovirus and lentivirus vector systems for hematopoietic cell gene therapy
S Brenner et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH (2003)
Side effects of retroviral gene transfer into hematopoietic stem cells
C Baum et al.
BLOOD (2003)
A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine Transcarbamylase deficiency
SE Raper et al.
HUMAN GENE THERAPY (2002)
Retroviral vectors
KM Kurian et al.
JOURNAL OF CLINICAL PATHOLOGY-MOLECULAR PATHOLOGY (2000)
Biological activity and safety of adenoviral vector-expressed wild-type p53 after intratumoral injection in melanoma and breast cancer patients with p53-overexpressing tumors
R Dummer et al.
CANCER GENE THERAPY (2000)
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
M Cavazzana-Calvo et al.
SCIENCE (2000)
分享论文
