Effective delivery of large genes to the retina by dual AAV vectors

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

(2013) V S Lopes et al.   GENE THERAPY

Oversized AAV Transductifon Is Mediated via a DNA-PKcs-independent, Rad51C-dependent Repair Pathway

(2013) Matthew L Hirsch et al.   MOLECULAR THERAPY

Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

(2013) Francesco Testa et al.   OPHTHALMOLOGY

AAV2/8 Vectors Purified from Culture Medium with a Simple and Rapid Protocol Transduce Murine Liver, Muscle, and Retina Efficiently

(2013) Monica Doria et al.   Human Gene Therapy Methods

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

(2012) Arpad Palfi et al.   HUMAN GENE THERAPY

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

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

Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

(2012) William E. Grose et al.   PLoS One

Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation

(2012) Yuan Wang et al.   Human Gene Therapy Methods

Increased cone sensitivity to ABCA4 deficiency provides insight into macular vision loss in Stargardt's dystrophy

(2011) Shannon M. Conley et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Novel adeno-associated viral vectors for retinal gene therapy

(2011) L H Vandenberghe et al.   GENE THERAPY

AAV-mediated photoreceptor transduction of the pig cone-enriched retina

(2011) C Mussolino et al.   GENE THERAPY

Fighting Blindness with Adeno-Associated Virus Serotype 8

(2011) Alberto Auricchio   HUMAN GENE THERAPY

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

(2011) L. H. Vandenberghe et al.   Science Translational Medicine

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

(2010) Arkasubhra Ghosh et al.   HUMAN GENE THERAPY

C20-D3-vitamin A Slows Lipofuscin Accumulation and Electrophysiological Retinal Degeneration in a Mouse Model of Stargardt Disease

(2010) Li Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus

(2010) Matthew L Hirsch et al.   MOLECULAR THERAPY

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

(2010) Paul E Monahan et al.   MOLECULAR THERAPY

Mass spectrometry provides accurate and sensitive quantitation of A2E

(2010) Danielle B. Gutierrez et al.   PHOTOCHEMICAL & PHOTOBIOLOGICAL SCIENCES

Establishment of an AAV Reverse Infection-Based Array

(2010) Xiaoyan Dong et al.   PLoS One

Defective lipid transport and biosynthesis in recessive and dominant Stargardt macular degeneration

(2010) Robert S. Molday et al.   PROGRESS IN LIPID RESEARCH

An Update on the Genetics of Usher Syndrome

(2010) José M. Millán et al.   Journal of Ophthalmology

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

AAV-mediated Tyrosinase Gene Transfer Restores Melanogenesis and Retinal Function in a Model of Oculo-cutaneous Albinism Type I (OCA1)

(2009) Annagiusi Gargiulo et al.   MOLECULAR THERAPY

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

(2009) Yi Lai et al.   MOLECULAR THERAPY

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

(2009) Biao Dong et al.   MOLECULAR THERAPY

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

(2009) Artur V. Cideciyan et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV Recombineering with Single Strand Oligonucleotides

(2009) Matthew L. Hirsch et al.   PLoS One

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

(2008) Mariacarmela Allocca et al.   JOURNAL OF CLINICAL INVESTIGATION

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

Ocular gene therapy: current progress and future prospects

(2008) Pasqualina Colella et al.   TRENDS IN MOLECULAR MEDICINE

A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY