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公开(公告)号：US20210180023A1
公开(公告)日：2021-06-17
申请号：US16639523
申请日：2018-06-04
Inventor： Eric N. OLSON , Huanyu ZHOU , Rhonda BASSEL-DUBY
Abstract: The present disclosure involves the use of reprogramming factors including AKT1, GATA4, TBX5, MEF2C, HAND2 and either ZNF281 or AS-CL1 to reprogram adult non-cardiomyocytes, such as cardiac fibroblasts into cardiomyocytes, both in vitro and in vivo. Such methods find particular use in the treatment of patients post-myocardial infarction to prevent or limit scarring and to promote myocardial repair.
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公开(公告)号：US20200370042A1
公开(公告)日：2020-11-26
申请号：US16966274
申请日：2019-01-31
Inventor： Eric N. OLSON , Chengzu LONG
IPC: C12N15/11 , C12N9/22 , C12N7/00 , C12N15/86 , C12N5/10 , C12N5/077 , A61P21/00 , A61K35/34 , C12N5/074
Abstract: The disclosure provides a method for treating or preventing Duchene Muscular Dystrophy (DMD) in a subject in need thereof, the method comprising administering to the subject a Cas9 nuclease or a sequence encoding a Cas9 nuclease, and a gRNA or a sequence encoding a gRNA, wherein the gRNA targets a splice donor or splice acceptor site of the dystrophin gene. The administering restores dystrophin expression in at least a subset of the subjects cardiomyocytes, and may at least partially or fully restore cardiac contractility.
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公开(公告)号：US20240325454A1
公开(公告)日：2024-10-03
申请号：US18575499
申请日：2022-06-30
Inventor： Eric N. OLSON , Rhonda BASSEL-DUBY , Takahiko NISHIYAMA
CPC classification number: A61K35/34 , A61K48/005 , C12N9/22 , C12N15/11 , C12N15/86 , C12N2310/20 , C12N2750/14143
Abstract: Disclosures herein are directed to compositions comprising single guide RNA (sgRNA) designed for a CRISPR/Cas9 system and method of using thereof for preventing, ameliorating or treating one or more cardiomyopathies. For example, provided herein are composition and methods for the correction of dilated cardiomyopathy by precise genomic editing of RBM20 mutations in human cells and mice.
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公开(公告)号：US20230241249A1
公开(公告)日：2023-08-03
申请号：US18004522
申请日：2021-07-06
Inventor： Eric N. OLSON , Rhonda S. BASSEL-DUBY , Benjamin R. NELSON , Catherine A. MAKAREWICH
CPC classification number: A61K48/0058 , A61K48/0016 , A61P9/04
Abstract: Disclosed are methods of treating a subject, such as those having or at risk of cardiomyopathies, with an effective amount of a recombinant adeno-associated virus (rAAV) virion, the rAAV virion comprising an AAV capsid and an expression cassette comprising a polynucleotide encoding a DWORF polypeptide operatively linked to a promoter. Compositions and kits relating to the same are also disclosed.
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公开(公告)号：US20210380650A1
公开(公告)日：2021-12-09
申请号：US17392137
申请日：2021-08-02
Inventor： Eric N. OLSON , Rhonda S. BASSEL-DUBY , Catherine A. MAKAREWICH , Benjamin R. NELSON
Abstract: The present disclosure describes a new native peptide designated herein as Dwarf Open Reading Frame, or DWORF. This peptide enhances the apparent activity of the SERCA pump, is positively inotropic and lusitropic, and therefore is provided as a therapeutic agent for disorders characterized by cytosolic calcium overload.
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Patent Agency Ranking
公开(公告)号：US20200260698A1
公开(公告)日：2020-08-20
申请号：US16639828
申请日：2018-08-17
Inventor： Viktoriia KYRYCHENKO , Eric N. OLSON , Rhonda BASSEL-DUBY
IPC: A01K67/027 , C12N15/10 , C12N15/113 , A61P21/00
Abstract: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene and absence or deficiency of dystrophin protein in striated muscle. Provided herein are compositions and methods for treating DMD caused by mutations in the dystrophin Actin Binding Domain 1 (ABD-1). The compositions and method described herein can be used to remove mutant sequences in dystrophin ABD-1 to generate a corrected DMD protein that, while lacking one or more exons (e.g., exons 3-9), retains important functional properties.
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公开(公告)号：US20240165271A1
公开(公告)日：2024-05-23
申请号：US18552262
申请日：2022-03-25
Inventor： Eric N. OLSON , Francesco CHEMELLO , Rhonda BASSEL-DUBY
IPC: A61K48/00 , A61K31/7088 , A61K38/46 , A61P21/00 , C07K14/47 , C12N9/22 , C12N15/11 , C12N15/86 , C12N15/90
CPC classification number: A61K48/0066 , A61K31/7088 , A61K38/465 , A61K48/0033 , A61P21/00 , C07K14/4716 , C12N9/22 , C12N15/11 , C12N15/86 , C12N15/907 , C12N2310/20 , C12N2750/14143 , C12N2800/80
Abstract: Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. Provided herein are methods of using adenine base editor (ABE) to modify splice sites of the dystrophin gene, causing skipping or refraining of common DMD exon deletion mutations, restoring dystrophin expression. Also provided herein are methods of using prime editing to reframe the dystrophin open reading frame and restore dystrophin expression.
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公开(公告)号：US20220072156A1
公开(公告)日：2022-03-10
申请号：US17404915
申请日：2021-08-17
Inventor： Eric N. OLSON , Chengzu LONG , John R. McANALLY , John M. SHELTON , Rhonda BASSEL-DUBY
IPC: A61K48/00 , A61K38/46 , C12N15/113
Abstract: Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. The disclosure reports CRISPR/Cas9-mediated gene editing (Myo-editing) is effective at correcting the dystrophin gene mutation in the mdx mice, a model for DMD. Further, the disclosure reports optimization of germline editing of mdx mice by engineering the permanent skipping of mutant exon (exon 23) and extending exon skipping to also correct the disease by post-natal delivery of adeno-associate virus (AAV). AAV-mediated Myo-editing can efficiently rescue the reading frame of dystrophin in mdx mice in vivo. The disclosure reports means of Myo-editing-mediated exon skipping has been successfully advanced from somatic tissues in mice to human DMD patients-derived iPSCs (induced pluripotent stem cells). Custom Myo-editing was performed on iPSCs from patients with differing mutations and successfully restored dystrophin protein expression for all mutations in iPSCs-derived cardiomyocytes.
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9.发明公开公开(公告)号：US20240226334A1
公开(公告)日：2024-07-11
申请号：US18563790
申请日：2022-05-24
Inventor： Eric N. OLSON , Yu ZHANG , Rhonda BASSEL-DUBY
CPC classification number: A61K48/0058 , A61P21/00 , C12N9/22 , C12N15/111 , C12N15/86 , C12N2310/20 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2750/14143
Abstract: Provided herein are gene therapy methods, vectors and constructs for the treatment of Duchenne Muscular Dystrophy in a subject.
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公开(公告)号：US20190382732A1
公开(公告)日：2019-12-19
申请号：US16487937
申请日：2018-02-14
Inventor： Eric N. OLSON , Pengpeng BI
Abstract: The present disclosure describes the fusogenic promoting activity of the Myomixer protein. This polypeptide, when expressed in non-muscle cells with the Myomaker protein, is able to increase fusion of the cell with a muscle cell, but not with other non-muscle cells, as compared to cells only expression the Myomaker protein. The use of this protein and cells expressing it in the delivery of exogenous genetic material to muscle cells also is described.
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