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公开(公告)号：US20240216542A1
公开(公告)日：2024-07-04
申请号：US18557102
申请日：2022-04-18
Inventor： Huiya Yang , Guangping Gao , Kevin A. Strauss , Dan Wang
CPC classification number: A61K48/0058 , A61K48/0075 , A61P3/00 , A61P25/00 , C12N9/1081 , C12N15/86 , C12Y204/99009 , C12N2750/14143 , C12N2830/008
Abstract: Aspects of the disclosure relate to methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof by administering an rAAV expressing one or more Ganglioside GM3 synthase (GM3S) isoforms via intracerebroventricular (ICV) administration.
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公开(公告)号：US20240207370A1
公开(公告)日：2024-06-27
申请号：US18288211
申请日：2022-04-26
Inventor： Guangping Gao , Dan Wang , Jiaming Wang , Kevin A. Strauss
CPC classification number: A61K38/44 , A61K48/0033 , A61K48/0066 , A61P3/00 , C12N15/86 , C12Y102/04004 , C12N2750/14143
Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, and/or BCKDHB, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).
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公开(公告)号：US20220162571A1
公开(公告)日：2022-05-26
申请号：US17602348
申请日：2020-04-10
Applicant: University of Massachusetts , Clinic for Special Children
Inventor： Guangping Gao , Huiya Yang , Kevin A. Strauss , Dan Wang
Abstract: Aspects of the disclosure relate to compositions and methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof.
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公开(公告)号：US20210023241A1
公开(公告)日：2021-01-28
申请号：US16982640
申请日：2019-03-22
Applicant: University of Massachusetts
Inventor： Jae-Hyuck Shim , Guangqing Gao , Jun Xie , Jung Min Kim , Dan Wang , Yeon-Suk Yang
IPC: A61K48/00 , C07K14/635 , A61P19/10 , C12N15/86 , C07K14/005 , C12N15/113
Abstract: In some aspects, the disclosure relates to compositions and methods for modulating (e.g., increasing and/or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).
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公开(公告)号：US10584321B2
公开(公告)日：2020-03-10
申请号：US15550452
申请日：2016-02-12
Applicant: University of Massachusetts
Inventor： Guangping Gao , Phillip D. Zamore , Dan Wang
Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AAV capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
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Patent Agency Ranking
公开(公告)号：US11773410B2
公开(公告)日：2023-10-03
申请号：US16093575
申请日：2017-04-13
Applicant: University of Massachusetts
Inventor： Guangping Gao , Dan Wang
CPC classification number: C12N15/907 , C12N9/22 , C12N15/1024 , C12N15/11 , C12N15/86 , C12N2320/34 , C12N2750/14143 , C12N2800/80
Abstract: The disclosure in some aspects relates to methods and compositions for repairing mutations (e.g., compound heterozygous mutations) that are widely found in patients having certain diseases (e.g., monogenic recessive diseases). In some aspects, the disclosure provides a method for targeted allelic exchange using recombinant gene editing complex.
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公开(公告)号：US20230183711A1
公开(公告)日：2023-06-15
申请号：US17767962
申请日：2020-10-14
Applicant: University of Massachusetts
Inventor： Guangping Gao , Dan Wang , Jote Bulcha
IPC: C12N15/52 , C07K14/005
CPC classification number: C12N15/52 , C07K14/005 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152
Abstract: Aspects of the disclosure relate to manufacturing AAV using a stable cell line that expresses the rep gene by engineering tRNA to suppress a mutation introduced to a rep integrated into the genome of the cell line.
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公开(公告)号：US20230346978A1
公开(公告)日：2023-11-02
申请号：US17918714
申请日：2021-04-13
Applicant: University of Massachusetts
Inventor： Dan Wang , Guangping Gao , Jiaming Wang
CPC classification number: A61K48/0066 , C12N15/11 , C12N9/22 , C12N9/78 , C12Y305/04004 , C12N15/86 , A61P43/00 , C12N2310/20 , C12N2750/14143
Abstract: Aspects of the disclosure relate to compositions (e.g., isolated nucleic acids, rAAV vectors, rAAVs, etc.) and methods for gene editing. The disclosure is based, in part, on isolated nucleic acids encoding combinations of gene editing proteins (e.g., Cas proteins) and base editors (e.g., Adenosine Deaminase Acting on RNA deaminase domains) with certain regulatory sequences that are amenable to packaging in recombinant adeno-associated viruses (rAAVs). In some embodiments, compositions described by the disclosure are useful for treating certain diseases in a subject in need thereof.
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公开(公告)号：US20220162570A1
公开(公告)日：2022-05-26
申请号：US17602353
申请日：2020-04-10
Applicant: University of Massachusetts , Clinic for Special Children
Inventor： Guangping Gao , Dan Wang , Jiaming Wang , kevin A Strauss
Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHB protein, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA and BCKDHB proteins, in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).
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公开(公告)号：US20200231953A1
公开(公告)日：2020-07-23
申请号：US16775311
申请日：2020-01-29
Applicant: University of Massachusetts
Inventor： Guangping Gao , Phillip D. Zamore , Dan Wang
Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AA V capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
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