The Ocular Gene Delivery Landscape

Childhood-onset genetic cone-rod photoreceptor diseases and underlying pathobiology

(2021) Alexandra V. Garafalo et al.   EBioMedicine

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

(2021) Ying Kai Chan et al.   Science Translational Medicine

Emerging patent landscape for non-viral vectors used for gene therapy

(2020) Virginia Picanço-Castro et al.   NATURE BIOTECHNOLOGY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea

(2020) Amy Frederick et al.   HUMAN GENE THERAPY

Evaluation of Photoreceptor Transduction Efficacy of Capsid-Modified Adeno-Associated Viral Vectors Following Intravitreal and Subretinal Delivery in Sheep

(2020) Maya Ross et al.   HUMAN GENE THERAPY

Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays

(2020) Thilo Buck et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Polymeric Nanoparticles in Gene Therapy: New Avenues of Design and Optimization for Delivery Applications

(2019) Raj Rai et al.   Polymers

Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina

(2019) Trapani   Genes

AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression

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

Gene therapy and genome surgery in the retina

(2018) James E. DiCarlo et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene therapy clinical trials worldwide to 2017: An update

(2018) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

Non-viral ocular gene therapy, pEYS606, for the treatment of non-infectious uveitis: Preclinical evaluation of the medicinal product

(2018) Elodie Touchard et al.   JOURNAL OF CONTROLLED RELEASE

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina

(2017) D G Hickey et al.   GENE THERAPY

Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina

(2017) Pavitra S. Ramachandran et al.   HUMAN GENE THERAPY

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

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

(2017) M. Dominik Fischer et al.   MOLECULAR THERAPY

Targeted Multifunctional Lipid ECO Plasmid DNA Nanoparticles as Efficient Non-viral Gene Therapy for Leber’s Congenital Amaurosis

(2017) Da Sun et al.   Molecular Therapy-Nucleic Acids

Viral vectors for gene therapy: Current state and clinical perspectives

(2016) A. N. Lukashev et al.   BIOCHEMISTRY-MOSCOW

Extended ocular drug delivery systems for the anterior and posterior segments: biomaterial options and applications

(2016) Jennifer J. Kang-Mieler et al.   Expert Opinion on Drug Delivery

Production and clinical development of nanoparticles for gene delivery

(2016) Jie Chen et al.   Molecular Therapy-Methods & Clinical Development

Genomic DNA nanoparticles rescue rhodopsin-associated retinitis pigmentosa phenotype

(2015) Zongchao Han et al.   FASEB JOURNAL

Non-viral therapeutic approaches to ocular diseases: An overview and future directions

(2015) Rahel Zulliger et al.   JOURNAL OF CONTROLLED RELEASE

Adenovirus Vectors for Gene Therapy, Vaccination and Cancer Gene Therapy

(2014) William Wold et al.   CURRENT GENE THERAPY

Adenoviruses as gene/vaccine delivery vectors: promises and pitfalls

(2014) Mohan Babu Appaiahgari et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA

(2013) Adarsha Koirala et al.   HUMAN MOLECULAR GENETICS

Persistence of non-viral vector mediated RPE65 expression: Case for viability as a gene transfer therapy for RPE-based diseases

(2013) Adarsha Koirala et al.   JOURNAL OF CONTROLLED RELEASE

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

The ciliopathies: a transitional model into systems biology of human genetic disease

(2012) Erica E Davis et al.   CURRENT OPINION IN GENETICS & DEVELOPMENT

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

(2012) Zongchao Han et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

(2012) W. A. Beltran et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Ciliopathies

(2011) Friedhelm Hildebrandt et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Genomic, Biochemical, and Cellular Responses of the Retina in Inherited Photoreceptor Degenerations and Prospects for the Treatment of These Disorders

(2010) Alexa N. Bramall et al.   Annual Review of Neuroscience

Efficient Transgene Reconstitution with Hybrid Dual AAV Vectors Carrying the Minimized Bridging Sequences

(2010) Arkasubhra Ghosh et al.   HUMAN GENE THERAPY

Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait

(2010) Alan F. Wright et al.   NATURE REVIEWS GENETICS

Suprachoroidal Drug Delivery to the Back of the Eye Using Hollow Microneedles

(2010) Samirkumar R. Patel et al.   PHARMACEUTICAL RESEARCH

Long-term follow-up of a family with dominant X-linked retinitis pigmentosa

(2009) D M Wu et al.   EYE

Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency

(2009) James M. Wilson   MOLECULAR GENETICS AND METABOLISM

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

The evolution of early vertebrate photoreceptors

(2009) S. P. Collin et al.   PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES

Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector

(2008) O Argyros et al.   GENE THERAPY

Tailoring the AAV vector capsid for gene therapy

(2008) L H Vandenberghe et al.   GENE THERAPY

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY