Search Results - aav

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CHST6 Mini Gene - CHST6 Expression Construct With Native Promoter (UCLA Case No. 2023-301)
UCLA researchers from the Department of Human Genetics and Department of Ophthalmology have developed a mini gene encoding a protein which is mutated in macular corneal dystrophy, leading to a potential gene therapy for this disorder. BACKGROUND: Macular corneal dystrophy (MCD) is a rare genetic disease caused by mutations in the CHST6 gene, which...
Published: 11/22/2024   |   Inventor(s): Wenlin Zhang, Anthony Aldave
Keywords(s): AAV, Adeno-associated viruses (AAV), Gene Editing Systems, Gene Therapy, genetics, keratinocytes, macular corneal dystrophy, mini gene, mutation, native promoter, Ophthalmology
Category(s): Therapeutics > Ophthalmology, Therapeutics > Gene Therapy And Editing
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: 12/6/2024   |   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): Collaboration Sought > Licensing, Application > Therapeutics, Collaboration Sought > Collaboration, TherapeuticArea > Ear, Nose, & Throat, TherapeuticArea > Ophthalmology
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: 10/28/2024   |   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: 10/28/2024   |   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: 10/28/2024   |   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: 10/28/2024   |   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: 10/28/2024   |   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: 10/28/2024   |   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: 12/26/2024   |   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
Improved Large Scale Production of Adeno-Associated Viruses
Enables a More Efficient and More Potent Production of AAV VectorsThis production of highly concentrated and potent Adeno-Associated Virus (AAV) vectors has significant advantage over available protocol and allows for large-scale manufacturing of the virus. More than 6 percent of all gene therapy clinical trials worldwide use AAV vectors due to their...
Published: 6/27/2021   |   Inventor(s): Barry Byrne, Nathalie Clement, Laura Adamson
Keywords(s): AAV, Gene Therapy, HEK293, HSV
Category(s): Technology Classifications > Human Health Care > Gene Therapy
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