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公开(公告)号:US20200181651A1
公开(公告)日:2020-06-11
申请号:US16093575
申请日:2017-04-13
Applicant: University of Massachusetts
Inventor: Guangping Gao , Dan Wang
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:US11920168B2
公开(公告)日:2024-03-05
申请号:US16775311
申请日:2020-01-29
Applicant: University of Massachusetts
Inventor: Guangping Gao , Phillip D. Zamore , Dan Wang
CPC classification number: C12N9/22 , A61K48/005 , C07K14/005 , C12N7/00 , C12N15/11 , C12N15/907 , A61K38/00 , C07K2319/735 , C12N2310/20 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143
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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公开(公告)号:US20230416779A1
公开(公告)日:2023-12-28
申请号:US18311367
申请日:2023-05-03
Applicant: University of Massachusetts
Inventor: Miguel Sena Esteves , Guangping Gao , Michael R. Green , Dan Wang , Tessa Mercedes Simone
CPC classification number: C12N15/86 , C07K14/4702 , C12N2750/14143 , C12N2310/141 , C12N2330/10 , C07K2319/41
Abstract: In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.
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公开(公告)号:US12194108B2
公开(公告)日:2025-01-14
申请号:US16982640
申请日:2019-03-22
Applicant: University of Massachusetts
Inventor: Jae-Hyuck Shim , Guangping Gao , Jun Xie , Jung Min Kim , Dan Wang , Yeon-Suk Yang
IPC: A61K48/00 , A61P19/10 , C07K14/005 , C07K14/635 , C12N15/113 , C12N15/86
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:US11680275B2
公开(公告)日:2023-06-20
申请号:US16619733
申请日:2018-06-06
Applicant: UNIVERSITY OF MASSACHUSETTS
Inventor: Miguel Sena Esteves , Guangping Gao , Michael R. Green , Dan Wang , Tessa Mercedes Simone
CPC classification number: C12N15/86 , C07K14/4702 , C07K2319/41 , C12N2310/141 , C12N2330/10 , C12N2750/14143
Abstract: In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.
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公开(公告)号:US20230151359A1
公开(公告)日:2023-05-18
申请号:US17910574
申请日:2021-03-08
Applicant: University of Massachusetts
Inventor: Dan Wang , Guangping Gao , Ailing Du
CPC classification number: C12N15/11 , C12N15/86 , A61P43/00 , C12N2750/14143 , C12N2800/30
Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional Forkhead box G1 (FOXG1) protein in a subject. In some embodiments, the disclosure provides methods of treating a subject having FOXG1 deficiency.
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公开(公告)号:US20230121437A1
公开(公告)日:2023-04-20
申请号:US17768305
申请日:2020-10-14
Applicant: University of Massachusetts
Inventor: Guangping Gao , Dan Wang , Jiaming Wang
Abstract: Aspects of the disclosure relate to compositions and methods for exon replacement in a cell or a subject. In some embodiments, the disclosure relates to isolated nucleic acids (and vectors, such as rAAV vectors) encoding one or more guideRNAs (gRNAs) that target an intron-exon boundary; an intronic sequence having a splice signal; and a donor sequence encoding a gene product of a gene of interest, or portion thereof. In some embodiments, compositions described herein are useful for replacing mutant exons associated with certain diseases, for example Duchen's muscular dystrophy (DMD), cystic fibrosis (CF), spinal muscular atrophy (SMA), Rett syndrome, and mucopolysaccharidosis (MPS).
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公开(公告)号:US20230089490A1
公开(公告)日:2023-03-23
申请号:US17767727
申请日:2020-10-09
Applicant: University of Massachusetts
Inventor: Guangping Gao , Craig Mendonca , Dan Wang
Abstract: Aspects of the disclosure relate to compositions and methods for treating certain diseases associated with the presence of one or more premature stop codons in a gene, for example dominantly inherited diseases or recessively inherited diseases. In some embodiments, compositions comprise a vector (e.g., a viral vector, such as an rAAV vector) encoding one or more synthetic suppressor transfer RNAs (tRNAs) configured to read-through certain stop codons (e.g., premature stop codons). In some embodiments, the disclosure relates to methods for treating Hurler syndrome comprising administering such vectors to a subject.
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公开(公告)号:US20180179501A9
公开(公告)日:2018-06-28
申请号:US15550452
申请日:2016-02-12
Applicant: University of Massachusetts
Inventor: Guangping Gao , Phillip D. Zamore , Dan Wang
CPC classification number: C12N9/22 , A61K38/00 , A61K48/005 , C07K14/005 , C07K2319/735 , C12N7/00 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143
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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公开(公告)号:US20180037877A1
公开(公告)日:2018-02-08
申请号:US15550452
申请日:2016-02-12
Applicant: University of Massachusetts
Inventor: Guangping Gao , Phillip D. Zamore , Dan Wang
CPC classification number: C12N9/22 , A61K38/00 , A61K48/005 , C07K14/005 , C07K2319/735 , C12N7/00 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143
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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