Review of gene therapies for age-related macular degeneration

Host immune responses after suprachoroidal delivery of AAV8 in nonhuman primate eyes.

(2021) Sook Hyun Chung et al.   HUMAN GENE THERAPY

Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells

(2021) Anna K. Dreismann et al.   GENE THERAPY

Long-Term Safety Evaluation of Continuous Intraocular Delivery of Aflibercept by the Intravitreal Gene Therapy Candidate ADVM-022 in Nonhuman Primates

(2021) Szilárd Kiss et al.   Translational Vision Science & Technology

Comprehensive Preclinical Assessment of ADVM-022, an Intravitreal Anti-VEGF Gene Therapy for the Treatment of Neovascular AMD and Diabetic Macular Edema

(2021) Claire M. Gelfman et al.   JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS

Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates

(2020) Glenn Yiu et al.   Molecular Therapy-Methods & Clinical Development

Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal Neovascularization

(2020) Sook Hyun Chung et al.   Molecular Therapy-Methods & Clinical Development

Suprachoroidal gene transfer with nonviral nanoparticles

(2020) Jikui Shen et al.   Science Advances

Analysis of Aflibercept Expression in NHPs following Intravitreal Administration of ADVM-022, a Potential Gene Therapy for nAMD

(2020) Szilárd Kiss et al.   Molecular Therapy-Methods & Clinical Development

AAVcis-regulatory sequences are correlated with ocular toxicity

(2019) Wenjun Xiong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

(2019) Albert M. Maguire et al.   OPHTHALMOLOGY

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression

(2019) Kun Ding et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2019) Andrew V. Anzalone et al.   NATURE

Choroidal Changes After Suprachoroidal Injection of Triamcinolone Acetonide in Eyes With Macular Edema Secondary to Retinal Vein Occlusion

(2018) Alex S. Willoughby et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Clustered Regularly Interspaced Short Palindromic Repeats-Based Genome Surgery for the Treatment of Autosomal Dominant Retinitis Pigmentosa

(2018) Yi-Ting Tsai et al.   OPHTHALMOLOGY

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Dosage thresholds and influence of transgene cassette in AAV-related toxicity

(2018) Hanen Khabou et al.   HUMAN GENE THERAPY

Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia

(2018) Kanmin Xue et al.   NATURE MEDICINE

Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration

(2018) Ruslan Grishanin et al.   MOLECULAR THERAPY

Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye

(2018) Gerard A. Rodrigues et al.   PHARMACEUTICAL RESEARCH

Nonintegrating Gene Therapy Vectors

(2017) Takis Athanasopoulos et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial

(2017) Céline Vandamme et al.   HUMAN GENE THERAPY

Adeno-associated Virus (AAV) Serotypes Have Distinctive Interactions with Domains of the Cellular AAV Receptor

(2017) Sirika Pillay et al.   JOURNAL OF VIROLOGY

Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial

(2017) Jeffrey S Heier et al.   LANCET

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

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

(2017) Guo-Xiang Ruan et al.   MOLECULAR THERAPY

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

(2017) Nicole M. Gaudelli et al.   NATURE

Subretinal Injection: A Review on the Novel Route of Therapeutic Delivery for Vitreoretinal Diseases

(2017) Yingqian Peng et al.   OPHTHALMIC RESEARCH

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

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

(2016) Alexis C. Komor et al.   NATURE

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

(2016) Richard G. Weleber et al.   OPHTHALMOLOGY

Protective responses to sublytic complement in the retinal pigment epithelium

(2016) Li Xuan Tan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina

(2016) Maria Carmela Latella et al.   Molecular Therapy-Nucleic Acids

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

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Review and update on the molecular basis of Leber congenital amaurosis

(2015) Oscar Francisco Chacon-Camacho   World Journal of Clinical Cases

Characterization of the Choroid-Scleral Junction and Suprachoroidal Layer in Healthy Individuals on Enhanced-Depth Imaging Optical Coherence Tomography

(2013) Glenn Yiu et al.   JAMA Ophthalmology

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy: Design and Prospects for Craniofacial Regeneration

(2009) E.L. Scheller et al.   JOURNAL OF DENTAL RESEARCH

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE