Evaluation of AAV-DJ vector for retinal gene therapy

Gene therapy for RPE65-related retinal disease

(2018) Virginia Miraldi Utz et al.   OPHTHALMIC GENETICS

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

Heparan Sulfate Binding Promotes Accumulation of Intravitreally Delivered Adeno-associated Viral Vectors at the Retina for Enhanced Transduction but Weakly Influences Tropism

(2016) Kenton T. Woodard et al.   JOURNAL OF VIROLOGY

Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors

(2016) Sanford L. Boye et al.   JOURNAL OF VIROLOGY

A Mechanism for Cancer-Associated Membranous Nephropathy

(2016) Elion Hoxha et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models

(2015) Lucie P. Pellissier et al.   HUMAN MOLECULAR GENETICS

Promising and delivering gene therapies for vision loss

(2015) Livia S. Carvalho et al.   VISION RESEARCH

Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h−/− mouse

(2014) L C Byrne et al.   GENE THERAPY

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

(2014) Sandra P Melo et al.   MOLECULAR THERAPY

Specific tools for targeting and expression in Müller glial cells

(2014) Lucie P Pellissier et al.   Molecular Therapy-Methods & Clinical Development

Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors

(2013) Christine N. Kay et al.   PLoS One

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

Differential Proteomics and Functional Research following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis

(2012) Qinxiang Zheng et al.   PLoS One

Structure of AAV-DJ, a Retargeted Gene Therapy Vector: Cryo-Electron Microscopy at 4.5 Å Resolution

(2012) Thomas F. Lerch et al.   STRUCTURE

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Efficient production of Fah -null heterozygote pigs by chimeric adeno-associated virus-mediated gene knockout and somatic cell nuclear transfer

(2011) Raymond D. Hickey et al.   HEPATOLOGY

Resveratrol Prevents Light-Induced Retinal Degeneration via Suppressing Activator Protein-1 Activation

(2010) Shunsuke Kubota et al.   AMERICAN JOURNAL OF PATHOLOGY

Inner Limiting Membrane Barriers to AAV-mediated Retinal Transduction From the Vitreous

(2009) Deniz Dalkara et al.   MOLECULAR THERAPY

A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells

(2009) Ryan R. Klimczak et al.   PLoS One

Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization

(2008) P Pechan et al.   GENE THERAPY

Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection

(2008) M Hellström et al.   GENE THERAPY

Immunity to adeno-associated virus vectors in animals and humans: a continued challenge

(2008) A K Zaiss et al.   GENE THERAPY

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

Molecular Characterization of the Heparin-Dependent Transduction Domain on the Capsid of a Novel Adeno-Associated Virus Isolate, AAV(VR-942)

(2008) M. Schmidt et al.   JOURNAL OF VIROLOGY

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

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