4.3 Article

Therapeutic Gene Editing in Inherited Retinal Disorders

Related references

Note: Only part of the references are listed.
Article Biotechnology & Applied Microbiology

CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa

Wen-Hsuan Wu et al.

Summary: This study presents a CRISPR-based gene modification therapy for autosomal dominant retinitis pigmentosa, which has been validated in a humanized mouse model, demonstrating the effectiveness of the treatment and its potential application in other genetic diseases.

MOLECULAR THERAPY (2022)

Article Biotechnology & Applied Microbiology

Dual-AAV delivering split prime editor system for in vivo genome editing

Shengyao Zhi et al.

Summary: Prime editor (PE) is a new genome editing tool that has the potential to correct the majority of known human genetic disease-related mutations. In this study, split-PEs were constructed and delivered using dual adenoassociated viruses (AAVs), successfully mediating gene editing in human cells and adult mouse retina.

MOLECULAR THERAPY (2022)

Article Multidisciplinary Sciences

In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration

Elliot H. Choi et al.

Summary: The study demonstrates that in vivo base editing can prolong the survival of cone photoreceptors and restore their function in a mouse model of Leber congenital amaurosis.

NATURE COMMUNICATIONS (2022)

Review Medicine, Research & Experimental

CRISPR/Cas therapeutic strategies for autosomal dominant disorders

Salvatore Marco Caruso et al.

Summary: This article details recent advancements in CRISPR therapeutics for treating a wide range of autosomal dominant disorders and discusses how these advancements are shaping the future of therapies.

JOURNAL OF CLINICAL INVESTIGATION (2022)

Article Engineering, Biomedical

Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing

Susie Suh et al.

Summary: Subretinal viral delivery of an adenine base editor and a single-guide RNA targeting a nonsense mutation in the Rpe65 gene restores near-normal levels of retinal and visual functions in mice.

NATURE BIOMEDICAL ENGINEERING (2021)

Article Biotechnology & Applied Microbiology

Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations

Kalyan Dulla et al.

Summary: Mutations in the USH2A gene are common causes of retinitis pigmentosa, with skipping of exon 13 showing promise as a treatment modality. Research has demonstrated that QR-421a-induced exon skipping has the potential to be a highly effective treatment option for RP caused by mutations in USH2A exon 13.

MOLECULAR THERAPY (2021)

Review Biotechnology & Applied Microbiology

Prime Editing for Inherited Retinal Diseases

Bruna Lopes da Costa et al.

Summary: Inherited retinal diseases (IRDs) are chronic, hereditary disorders that lead to progressive degeneration of the retina. While there is currently only one FDA-approved gene augmentation therapy available, new techniques such as prime editing (PE) offer promising implications for the future of IRD treatment.

FRONTIERS IN GENOME EDITING (2021)

Article Biotechnology & Applied Microbiology

CRISPR-Cas12-based detection of SARS-CoV-2

James P. Broughton et al.

NATURE BIOTECHNOLOGY (2020)

Review Biotechnology & Applied Microbiology

Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

Andrew V. Anzalone et al.

NATURE BIOTECHNOLOGY (2020)

Review Biotechnology & Applied Microbiology

Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa

Da Meng et al.

MOLECULAR THERAPY (2020)

Article Biochemistry & Molecular Biology

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

Morgan L. Maeder et al.

NATURE MEDICINE (2019)

Review Clinical Neurology

Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias

Ronald A. M. Buijsen et al.

NEUROTHERAPEUTICS (2019)

Article Ophthalmology

Ophthalmic features of spinocerebellar ataxia type 7

A. Campos-Romo et al.

Review Ophthalmology

Non-syndromic retinitis pigmentosa

Sanne K. Verbakel et al.

PROGRESS IN RETINAL AND EYE RESEARCH (2018)

Review Biochemistry & Molecular Biology

Seeing the Light after 25 Years of Retinal Gene Therapy

Ivana Trapani et al.

TRENDS IN MOLECULAR MEDICINE (2018)

Article Ophthalmology

Inherited Retinal Degenerations: Current Landscape and Knowledge Gaps

Jacque L. Duncan et al.

TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (2018)

Article Multidisciplinary Sciences

Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Artur V. Cideciyan et al.

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

Article Medicine, Research & Experimental

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

Kalyan Dulla et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Article Biochemistry & Molecular Biology

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

Lukas Villiger et al.

NATURE MEDICINE (2018)

Article Biochemistry & Molecular Biology

In utero CRISPR-mediated therapeutic editing of metabolic genes

Avery C. Rossidis et al.

NATURE MEDICINE (2018)

Review Ophthalmology

Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options

Preena Tanna et al.

BRITISH JOURNAL OF OPHTHALMOLOGY (2017)

Article Biotechnology & Applied Microbiology

CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10

Guo-Xiang Ruan et al.

MOLECULAR THERAPY (2017)

Article Multidisciplinary Sciences

RNA targeting with CRISPR-Cas13

Omar O. Abudayyeh et al.

NATURE (2017)

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)

Review Neurosciences

Polyglutamine spinocerebellar ataxias - from genes to potential treatments

Henry L. Paulson et al.

NATURE REVIEWS NEUROSCIENCE (2017)

Review Genetics & Heredity

Correction of Monogenic and Common Retinal Disorders with Gene Therapy

Jesse D. Sengillo et al.

GENES (2017)

Review Ophthalmology

CRISPR applications in ophthalmologic PINION genome surgery

Thiago Cabral et al.

CURRENT OPINION IN OPHTHALMOLOGY (2017)

Review Ophthalmology

A review of the mechanisms of cone degeneration in retinitis pigmentosa

Daniel S. Narayan et al.

ACTA OPHTHALMOLOGICA (2016)

Article Multidisciplinary Sciences

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

Alexis C. Komor et al.

NATURE (2016)

Article Medicine, General & Internal

Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis

J. W. B. Bainbridge et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

Review Medicine, Research & Experimental

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

Xiao-Hui Zhang et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2015)

Article Ophthalmology

Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration

Susan F. Murray et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2015)

Article Biotechnology & Applied Microbiology

Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7

Pavitra S. Ramachandran et al.

MOLECULAR THERAPY (2014)

Article Ophthalmology

Vector platforms for gene therapy of inherited retinopathies

Ivana Trapani et al.

PROGRESS IN RETINAL AND EYE RESEARCH (2014)

Article Multidisciplinary Sciences

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Artur V. Cideciyan et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)

Article Biotechnology & Applied Microbiology

Efficacy of Codelivery of Dual AAV2/5 Vectors in the Murine Retina and Hippocampus

Arpad Palfi et al.

HUMAN GENE THERAPY (2012)

Review Biochemistry & Molecular Biology

Playing the End Game: DNA Double-Strand Break Repair Pathway Choice

J. Ross Chapman et al.

MOLECULAR CELL (2012)

Article Multidisciplinary Sciences

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

Martin Jinek et al.

SCIENCE (2012)

Review Genetics & Heredity

Genetic therapies for RNA mis-splicing diseases

Suzan M. Hammond et al.

TRENDS IN GENETICS (2011)

Review Medicine, Research & Experimental

A look at autoimmunity and inflammation in the eye

Rachel R. Caspi

JOURNAL OF CLINICAL INVESTIGATION (2010)

Article Medicine, General & Internal

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Albert M. Maguire et al.

NEW ENGLAND JOURNAL OF MEDICINE (2008)

Article Medicine, General & Internal

Effect of gene therapy on visual function in Leber's congenital amaurosis

James W. B. Bainbridge et al.

NEW ENGLAND JOURNAL OF MEDICINE (2008)

Article Genetics & Heredity

RNA interference-mediated suppression and replacement of human rhodopsin in vivo

Mary O'Reilly et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2007)

Article Neurosciences

Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery

M. Gorbatyuk et al.

VISION RESEARCH (2007)

Review Cell Biology

An eye's view of T regulatory cells

Joan Stein-Streilein et al.

JOURNAL OF LEUKOCYTE BIOLOGY (2007)

Article Biochemistry & Molecular Biology

Effective gene therapy with nonintegrating lentiviral vectors

RJ Yáñez-Muñoz et al.

NATURE MEDICINE (2006)

Review Clinical Neurology

Molecular pathogenesis of spinocerebellar ataxias

Antoni Matilla Duenas et al.

BRAIN (2006)

Article Cell Biology

Spinocerebellar ataxia 7 (SCA7)

AS Lebre et al.

CYTOGENETIC AND GENOME RESEARCH (2003)