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公开(公告)号:US11617783B2
公开(公告)日:2023-04-04
申请号:US15776714
申请日:2016-11-15
IPC分类号: C12N5/00 , A61K38/39 , C12N9/22 , C12N15/90 , A61K48/00 , C07K14/47 , C12N15/88 , C12N15/113 , A61P21/00 , C12N15/11
摘要: The present application provides materials and methods for treating a patient with a titin-based myopathy, particularly a titin-based cardiomyopathy, and/or other titinopathy. In addition, the present application provides materials and methods for editing the titin gene in a cell by genome editing.
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公开(公告)号:US11566247B2
公开(公告)日:2023-01-31
申请号:US16519211
申请日:2019-07-23
IPC分类号: C07H21/04 , C12N15/113 , A61K31/7088 , A61K31/713
摘要: Compositions and methods for treating cancer in a subject in need thereof are described that includes administering a therapeutically effective amount of an oligonucleotide that inhibits the binding of splicing regulator SRSF1 or SRSF2 to MDM2 exon 4 or 11.
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公开(公告)号:US20230025574A1
公开(公告)日:2023-01-26
申请号:US17546428
申请日:2021-12-09
IPC分类号: C12N15/113 , A61K48/00 , C07K14/47 , C12N15/86 , C12N15/11 , C12N15/864 , C12N7/00
摘要: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
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公开(公告)号:US11534501B2
公开(公告)日:2022-12-27
申请号:US16757207
申请日:2018-03-16
摘要: The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
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公开(公告)号:US20220401482A1
公开(公告)日:2022-12-22
申请号:US17773755
申请日:2020-11-02
IPC分类号: A61K35/17 , C12N15/90 , C12N5/0783 , A61P35/00 , A61K39/395 , C07K16/28
摘要: Disclosed are genetically modified NK cells comprising a knockout of the cluster of differentiation 38 (CD38) gene and methods of using the same to treat a cancer including, but not limited to multiple myeloma, acute myeloid leukemia (AML), T-cell acute lymphoblastic leukemia (T-ALL), or Blastic plasmacytoid dendritic cell neoplasm (BPDCN).
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公开(公告)号:US20220389453A1
公开(公告)日:2022-12-08
申请号:US17769933
申请日:2020-10-19
IPC分类号: C12N15/86 , C12N15/113
摘要: The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of an Interferon regulatory factor 2 binding protein like (IRF2BPL) disorder.
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公开(公告)号:US20220333115A1
公开(公告)日:2022-10-20
申请号:US17231963
申请日:2021-04-15
申请人: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL , THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
IPC分类号: C12N15/113 , C12N15/86 , A61K31/713
摘要: RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 variants are provided. Delivery vehicles such as recombinant adeno-associated viruses deliver DNAs encoding RNAs that inhibit the expression of the dynamin-1 variants. The methods treat, for example, developmental and epileptic encephalopathies.
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公开(公告)号:US20220323498A1
公开(公告)日:2022-10-13
申请号:US17641473
申请日:2020-09-10
摘要: Disclosed are compositions and methods for measuring the likelihood of recovery of a recently thawed immune cell. Methods include assaying the level of an ADAM-17-cleaved surface receptor expressed on the immune cells, wherein the level of the surface receptor directly correlates with the likelihood of immune cell recovery (i.e., the greater the increase of the expression level an ADAM-17-cleaved surface receptor relative to a control, the greater the likelihood of recovery). The method can be used to determine if immune cells have sufficient viability to be used in immunotherapy before use.
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公开(公告)号:US20220282205A1
公开(公告)日:2022-09-08
申请号:US17668123
申请日:2022-02-09
发明人: Brian K. Kaspar
摘要: The present invention is directed to methods and materials for producing recombinant viruses. In particular, methods and materials are provided for producing recombinant viruses in eukaryotic microalgae such as Chlamydomonas reinhardtii. Recombinant adeno-associated viruses are examples of recombinant viruses produced according to the invention.
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公开(公告)号:US20220251156A1
公开(公告)日:2022-08-11
申请号:US17580228
申请日:2022-01-20
摘要: Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
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