Search Results - gene+therapy

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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

Induced Pluripotent Stem Cells Derived from Patients with CEP290-associated Ciliopathies and Unaffected Family Members

Abstract: Approximately one-third of non-syndromic retinal dystrophies involve a defect in a ciliary protein. Non-syndromic retinal ciliopathies include retinitis pigmentosa, cone dystrophy, cone-rod dystrophy, macular dystrophy, and Leber-congenital amaurosis (LCA). Many CEP290-LCA patients also exhibit auditory and olfactory defects. Induced pluripotent...

Published: 4/8/2024 | Inventor(s): Anand Swaroop, Hiroko Shimada-Ishii, Yu Holly Chen, Milton English

Keywords(s): Anosmia, Cell therapy, Ciliopathies, Congenital Blindness, Drug Development, GENE THERAPY, hearing loss, iPS, National Eye Institute, NEI, Pluripotent Stem Cells, Primary Cilia, Retina, Retinal degeneration, Swaroop, VISION

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

Mouse Embryo Culture Chamber and Imaging System and Methods of Use

Abstract: The culture of mouse embryos ex utero and continuous monitoring and imaging of embryos as they develop have applications in drug testing, genetic studies, and basic research on embryonic development. However, the embryo culture systems currently available for post-implantation embryos include rolling bottle culture systems, which do not permit...

Published: 4/8/2024 | Inventor(s): Vijay Kalaskar

Keywords(s): Culture Chamber, Embryo Development, Embryonic, FETAL, GENE THERAPY, IMAGING, In Vitro Drug Toxicology, Kalaskar, National Eye Institute, NEI, Organ System Therapy

Category(s): Application > Non-Medical Devices, TherapeuticArea > Reproductive Health, Application > Medical Devices, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat

Recombinant Virus Vectors for the Treatment of Glycogen Storage Disease type Ib (GSD-Ib)

Abstract: Glycogen storage disease type Ib (GSD-Ib) is an autosomal recessive disorder caused by deficiencies in glucose-6-phosphate transporter (G6PT), a ubiquitously expressed endoplasmic reticulum (ER) protein that translocates G6P from the cytoplasm into the ER lumen. Inside the ER, G6P is hydrolyzed to glucose and phosphate by either the liver/kidney/intestine-restricted...

Published: 4/8/2024 | Inventor(s): Janice Chou

Keywords(s): autosomal recessive disorder, Chou, G6PT, GENE THERAPY, glucose homeostasis, Glycogen storage disease type Ib, GSD-Ib, hepatocellular adenoma, metabolic and immune disorders, myeloid dysfunction, NEUTROPENIA, NICHD

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

Methods and Compositions for Treating Genetically Linked Diseases of the Eye

Abstract: X-linked retinoschisis (XLRS) is an inherited, monogenetic ocular disease caused by mutations in the retinoschisin (RS1) gene, resulting in the development of cystic cavities throughout the retina and leading to juvenile macular degeneration. Approximately 1:15,000 males in the US are affected, classifying the condition as an orphan indication. The...

Published: 4/8/2024 | Inventor(s): Paul Sieving, Ronald Bush, Yong Zeng, Peter Colosi

Keywords(s): Clinical, EYE, GENE THERAPY, ophthalmological, Orphan Indication, Sieving, XLRS

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

RP2 and RPGR Vectors For Treating X-linked Retinitis Pigmentosa

Abstract: X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor dysfunction primarily caused by mutations in RPGR or RP2 gene. This technology is poised to advance RPGR or RP2 gene therapy to clinical stage using AAV8 or AAV9 vector carrying human full-length RPGR or RP2-coding...

Published: 4/8/2024 | Inventor(s): Zhijian Wu, Anand Swaroop, Suddhasil Mookherjee, Suja Hiriyanna, Tiansen Li

Keywords(s): GENE THERAPY, hereditary ocular disease, Pigmentosa, Retina, Wu

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

Use of Replicators in Gene Therapy

Abstract: Gene therapies offer promising prospects of treating a wide variety of human diseases. In one method, a gene therapy vector can be utilized to deliver an unmutated copy of a gene, called a transgene, to replace a mutated gene in order to treat the genetic disorder. However, lack of expression of a therapeutic transgene and uncontrolled gene...

Published: 4/8/2024 | Inventor(s): Mirit Aladjem, Cindy Tseng, Haiquing Fu, Lixin Wang

Keywords(s): Aladjem, delivery system, DNA replication initiation, GENE EXPRESSION, Gene Silencing Modulation, GENE THERAPY, Replicators, TRANSGENE

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

Rapid Methods for Human Artificial Chromosome (HAC) Formation

Abstract: Gene therapy is a promising strategy to treat a wide range of human diseases, and several gene therapy vectors have been developed to deliver these novel treatments. However, risks and challenges of using these vectors remain, such as: gene integration, potential infection, immune response and maintaining long term, stable gene expression....

Published: 4/8/2024 | Inventor(s): Vladimir Larionov, William Earnshaw, Reto Gassman, Stefanie Kandels-Lewis, Hiroshi Masumoto, Megumi Nakano, Vladimir Noskov, Natalay Kouprina, J. Carl Barrett

Keywords(s): Alphoid DNA arrays, Centromere Seeding, Engineered Centromeric Sequences, Gene expression/delivery system, GENE THERAPY, Human artificial chromosomes (HACs), Mammalian Artificial Chromosomes Larionov, RCA, Recombinational Amplification of Repeats RAR, Rolling-circle amplification, TAR-NV, Transformation-Associated Recombination targeting vector

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

Reporter Assay for Detection and Quantitation of Replication-Competent Gammaretrovirus

Abstract: Gammaretroviral vectors were the first viral gene-therapy vectors to enter clinical trials and remain in use. One potential hazard associated with the use of such vectors is the presence of replication-competent retroviruses (RCR) in the vector preparations – either as a result of: 1) recombination events between the plasmids used for...

Published: 4/8/2024 | Inventor(s): Alan Rein, Amanda (Declined Royalty) Aloia

Keywords(s): Cell line, Gammaretrovirus, Gaussia Luciferase, GENE THERAPY, PLASMID, RCR, Rein, Replication-Competent Retrovirus, Vector Preparation

Category(s): Application > Research Materials, Collaboration Sought > Licensing, TherapeuticArea > Immunology

Use of Interleukin (IL)-34 to Treat Retinal Inflammation and Neurodegeneration

Abstract: Interleukin (IL)-34 is a homodimer that is produced mainly by keratinocytes, neuronal cells and regulatory T cells (Tregs). It is believed to play important roles in chronic inflammation and the homeostasis of microglia. Currently, there is no effective treatment for many types of retinal degeneration. An improved treatment of autoimmune uveitis...

Published: 4/8/2024 | Inventor(s): Rachel Caspi, Mary Mattapallil, Zhijian Wu

Keywords(s): AAV Vector, Caspi, GENE THERAPY, IL-34, inflammatory disease, Inherited Retinal Dystrophies, Interleukin 34, National Eye Institute, NEI, NEURODEGENERATION, Photoreceptor Death, Retinal degeneration, Retinal Inflammation, uveitis

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

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