Search Results - aav

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Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies

Abstract: Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype...

Published: 4/8/2024 | Inventor(s): Anand Swaroop, Kamil Kruczek, Suja Hiriyanna, Zhijian Wu

Keywords(s): AAV, Adeno-associated Virus, Cone-rod Dystrophy, CRD, GENE THERAPY, LCA, Leber Congenital Amaurosis, Lentivirus, NEI, rare disease, Retinitis Pigmentosa, Retinopathies, RP, Swaroop

Category(s): TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat, Collaboration Sought > Licensing, Application > Therapeutics, Collaboration Sought > Collaboration

Novel Adeno-associated Viral (AAV) Vectors to Treat Hereditary Methylmalonic Acidemia (MMA) Caused by Methylmalonyl-coA Mutase (MMUT) Deficiency

Deficiency of the enzyme in methylmalonyl-CoA mutase (MMUT) results is a life-threatening disease, methylmalonic acidemia (MMA), that carries high rates of morbidity and mortality. NHGRI scientists have developed novel AAV vectors that combine the proprietary codon-optimized synMMUT alleles with either a liver-specific promoter from the human alpha-1...

Published: 7/25/2024 | Inventor(s): Randy Chandler, Charles Venditti

Keywords(s): AAV, Acidemia, Adeno-associated, CAUSED, DEFICIENCY, Hereditary, Methylmalonic, Methylmalonyl-CoA, MMA, Mmut, Mutase, Novel, TREAT, vectors, viral, VPXXXX, WIXXXX, WJXXXX, XEXXXX

Category(s): TherapeuticArea > Cardiology, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, Application > Research Materials

Capsid-Free AAV Vectors for Gene Delivery and Their Use for Gene Therapy

The invention concerns novel capsid-free AAV vectors that can be used for gene delivery and gene therapy applications. The invention provides for a linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest, and a second AAV ITR, wherein said nucleic acid...

Published: 7/25/2024 | Inventor(s): Lina Li, Luis Garcia, Cyriaque Beley, Thomas Voit, Robert Kotin

Keywords(s): AAV, Cell, Clonal, Containing, DNA, GB2AXX, Lines, Listed LPM Reichman as of 4/15/2015, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Rescueable, Sf9, STABLE, Vector, VPXXXX, WJXXXX, XEXXXX, YAXXXX, YBXXXX

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Dental, TherapeuticArea > Endocrinology, Application > Therapeutics, TherapeuticArea > Ophthalmology, TherapeuticArea > Oncology, TherapeuticArea > Cardiology, TherapeuticArea > Infectious Disease

AAV5 Vector and Uses Thereof

The invention described and claimed in this patent application provides for novel vectors and viral particles which comprise adeno-associated virus serotype 5 (AAV5). AAV5 is a single-stranded DNA virus of either plus or minus polarity which, like other AAV serotypes (e.g., AAV4, AAV2) requires a helper virus for replication. AAV type 2 has the interesting...

Published: 7/25/2024 | Inventor(s): Robert Kotin, John (Jay) Chiorini

Keywords(s): AAV, AAV5, control seq, Duke DNA Project, GB2A1X, GB2A2X, GB2AXX, GB2XXX, GBXXXX, GENE THERAPY, GXXXXX, Listed LPM Reichman as of 4/15/2015, Patent Category - Biotechnology, PCT/US99/11958; WO 99/61601 (12/02/99), Post LPM Assignment Set 20150420, Pre LPM working set 20150418, SACGHS DNA Patent Initial Set, viral vector

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Application > Therapeutics

AAV5 Vector for Transducing Brain Cells and Lung Cells

The invention described and claimed in this patent application is related to the delivery of heterologous nucleic acids or genes to particular target cells. In particular, the application relates to methods of delivering a heterologous nucleic acid or gene of interest to particular target cells using an Adeno-Associated Virus of serotype 5 (AAV5)....

Published: 7/25/2024 | Inventor(s): Beverly Davidson, Robert Kotin, Joseph Zabner, John (Jay) Chiorini

Keywords(s): AAV, AAV5, brain, CB2AXX, CBXXXX, CXXXXX, GB2A2X, GB2XXX, GENE THERAPY, IB4XXX, IBXXXX, IXXXXX, Listed LPM Reichman as of 4/15/2015, LUNG, NB1XXX, NBXXXX, NXXXXX, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, SACGHS DNA Patent Initial Set, USES, Vector, viral vector

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Pulmonology, TherapeuticArea > Oncology, TherapeuticArea > Neurology, Application > Research Materials, Application > Diagnostics, Application > Therapeutics

AAV4 Vector and Uses Thereof

The invention described and claimed in this patent application relates to the delivery of heterologous nucleic acids or genes to particular target cells. In particular, the application relates to methods of delivering a heterologous nucleic acid or gene of interest to particular target cells using Adeno-Associated Virus of serotype 4 (AAV4). The particular...

Published: 7/25/2024 | Inventor(s): Robert Kotin, Brian Safer, Beverly Davidson, John (Jay) Chiorini

Keywords(s): AAV, AAV4, brain, CB2AXX, CBXXXX, CXXXXX, ependymal cells, GB2A2X, GB2XXX, GENE THERAPY, Listed LPM Reichman as of 4/15/2015, NB1XXX, NBXXXX, NXXXXX, Patent Category - Biotechnology, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, SACGHS DNA Patent Initial Set, USES, viral vector

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Oncology, Application > Therapeutics, TherapeuticArea > Neurology

Production of Adeno-Associated Viruses in Insect Cells

Adeno-associated virus (AAV) is being developed for gene therapy applications. This virus type presents several advantages over alternate vectors for therapeutic gene delivery. AAV is not considered pathogenic and transduces stably dividing and non-dividing cells. AAV also shows good serotype specificity to various cell types for targeted gene delivery. The...

Published: 7/25/2024 | Inventor(s): Robert Kotin

Keywords(s): AAV, AAV1, AAV2, AAV5, Adeno-associated, CB2AXX, CB2XXX, CBXXXX, Cells, CXXXXX, Duke DNA Project, GB2A2X, GB2AXX, GB2XXX, GBXXXX, GENE THERAPY, GXXXXX, insect, production, virus

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Oncology, Application > Therapeutics

A Novel Adeno-Associated Virus for Gene Therapy

Scientists at the NIH disclosed a novel adeno-associated virus (AAV) termed "44-9." AAV44-9 based vectors have high gene transfer activity in a number of cell types, including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum). These vectors...

Published: 7/25/2024 | Inventor(s): Giovanni Di Pasquale, John (Jay) Chiorini

Keywords(s): AAV, CHARACTERIZATION, Gene, Generation, Listed LPM Hu as of 4/17/2015, Novel, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, THERAPY, USEFUL, VAXXXX, VCXXXX, VEXXXX, VFXXXX, VGXXXX, WJXXXX, XEXXXX, YBXXXX, YCXXXX

Category(s): TherapeuticArea > Neurology, TherapeuticArea > Immunology, TherapeuticArea > Endocrinology, TherapeuticArea > Oncology, TherapeuticArea > Geriatrics, Application > Therapeutics