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EP1486567A1   [0006] 
WO2010093784A2   [0006] 
WO2007089632A2   [0006] 
US20140050701A1   [0006] 
WO2010011404A2   [0006] 
US20070015238A   [0102] 
US20120322861A   [0102] 
US61947940B   [0177] 

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors   [0102] 
Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors   [0103] 
Identification of a Heparin-Binding Motif on Adeno-Associated Virus Type 2 Capsids   [0103] 
Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated Virus Vectors of Serotypes 1 to 6   [0103] 
A Conformational Change in the Adeno-Associated Virus Type 2 Capsid Leads to the Exposure of Hidden VP1 N Termini   [0103] 
International efforts for recombinant adeno-associated viral vector reference standards   [0103] 
Gene therapy restores vision in a canine model of childhood blindness   [0175] 
The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9   [0175] 
Gene transfer into the mouse retina mediated by an adeno-associated viral vector   [0175] 
Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors   [0175] 
Proteomics profiling of the cone photoreceptor cell line, 661W   [0175] 
Optimization of the capsid of recombinant 1 adeno-associated virus 2 (AAV2) vectors:The final threshold?   [0175] 
High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors   [0175] 
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model   [0175] 
Effect of gene therapy on visual function in Leber's congenital amaurosis   [0175] 
Directed evolution of novel adeno-associated viruses for therapeutic gene delivery   [0175] 
rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters   [0175] 
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina   [0175] 
The human thodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina   [0175] 
Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse   [0175] 
Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse   [0175] 
Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system   [0175] 
Distribution of heparan sulfate proteoglycans in embryonic chicken neural retina and isolated inner limiting membrane   [0175] 
In-frame deletion in a novel centrosomal/ciliary protein CEP290/NPHP6 perturbs its interaction with RPGR and results in early-onset retinal degeneration in the rd16 mouse   [0175] 
Expression pattern of glycoconjugates in rat retina as analysed by lectin histochemistry   [0175] 
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year   [0175] 
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous   [0175] 
Gene therapy using adeno-associated virus vectors   [0175] 
Bio-engineering of AAV-2 capsid at specific serine, threonine or lysine residues improves its transduction efficiency in vitro and in vivo   [0175] 
Identification of the gene and the mutation responsible for the mouse nob phenotype   [0175] 
Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1   [0175] 
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection   [0175] 
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years   [0175] 
Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity   [0175] 
MicroRNA-restricted transgene expression in the retina   [0175] 
miRNeye: a microRNA expression atlas of the mouse eye   [0175] 
AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter   [0175] 
A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Muller cells   [0175] 
Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles   [0175] 
Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina   [0175] 
Safety and efficacy of gene transfer for Leber's congenital amaurosis   [0175] 
Topographic variations in the rabbit and primate internal limiting membrane   [0175] 
AAV-mediated photoreceptor transduction of the pig cone-enriched retina   [0175] 
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa   [0175] 
AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia   [0175] 
A naturally occurring mouse model of X-linked congenital stationary night blindness   [0175] 
AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector   [0175] 
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina   [0175] 
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors   [0175] 
Peptide affinity reagents for AAV capsid recognition and purification   [0175] 
Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity   [0175] 
The threefold protrusions of adeno-associated virus type 8 are involved in cell surface targeting as well as postattachment processing   [0175] 
Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines   [0175] 
Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain   [0175] 
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions   [0175] 
Expression of cone-photoreceptor-specific antigens in a cell line derived from retinal tumors in transgenic mice   [0175] 
Cone and rod transduction with alternative AAV serotypes in the macula of non-human primates   [0175] 
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey   [0175] 
Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery   [0175] 
Rhodopsin-EGFP knock-ins for imaging quantal gene alterations   [0175] 
Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait   [0175] 
Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size   [0175] 
Intravitreal injection of AAV2 transduces macaque inner retina   [0175] 
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses   [0175] 
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors   [0175]