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公开(公告)号：US20230032846A1
公开(公告)日：2023-02-02
申请号：US17782112
申请日：2020-12-03
Applicant: Duke University
Inventor： Charles A. Gersbach , Christopher Nelson
Abstract: The present invention provides DNA targeting systems and methods for delivery of gene editing machinery using lipid nanoparticles or microparticles.
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公开(公告)号：US20220195406A1
公开(公告)日：2022-06-23
申请号：US17603329
申请日：2020-04-14
Applicant: Duke University
Inventor： Charles A. Gersbach , Adrian Pickar Oliver
Abstract: Disclosed herein are CRISPR/Cas-based genome editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function.
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公开(公告)号：US20220177879A1
公开(公告)日：2022-06-09
申请号：US17603243
申请日：2020-04-12
Applicant: Duke University
Inventor： Charles A. Gersbach , Veronica Gough
IPC: C12N15/11 , C12N9/22 , A61K31/713 , C07K14/47
Abstract: Disclosed herein are CRISPR/Cas-based base editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function. In an aspect, the disclosure relates to a CRISPR/Cas-based base editing system for altering a RNA splice site encoded in the genomic DMA of a subject. In some embodiments, altering the RNA splice site encoded in the genomic DNA results in exclusion or inclusion of at least one exon sequence in an RNA transcript.
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公开(公告)号：US10745714B2
公开(公告)日：2020-08-18
申请号：US15991333
申请日：2018-05-29
Applicant: Duke University
Inventor： Charles A. Gersbach , Isaac B. Hilton , Pablo Perez-Pinera , Ami M. Kabadi , Pratiksha I. Thakore , David G. Ousterout , Joshua B. Black
Abstract: Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR/Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.
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公开(公告)号：US10704060B2
公开(公告)日：2020-07-07
申请号：US14895316
申请日：2014-06-05
Applicant: Duke University
Inventor： Charles A. Gersbach , Isaac B. Hilton , Pablo Perez-Pinera , Ami M. Kabadi , Pratiksha I. Thakore , David G. Ousterout , Joshua B. Black
Abstract: Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR/Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.
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Patent Agency Ranking
公开(公告)号：US10676726B2
公开(公告)日：2020-06-09
申请号：US15549842
申请日：2016-02-09
Applicant: Duke University
Inventor： Charles A. Gersbach , Isaac Hilton
Abstract: Disclosed herein are CRISPR/Cas9-based gene activation systems that include a fusion protein of a Cas9 protein and a protein having histone acetyltransferase activity, and methods of using said systems.
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17.发明申请公开(公告)号：US20180353615A1
公开(公告)日：2018-12-13
申请号：US15779633
申请日：2016-11-30
Applicant: Charles A. Gersbach , Jacqueline N. Robinson-Hamm , Duke University
Inventor： Charles A. Gersbach , Jacqueline N. Robinson-Hamm
IPC: A61K48/00 , C12Q1/68 , C12N15/85 , C12N15/10 , C12N15/113 , C12N15/80 , A61K31/7105 , A61K47/26 , A61K47/54
CPC classification number: A61K48/005 , A61K31/7105 , A61K47/26 , A61K47/549 , A61K48/0016 , C12N15/102 , C12N15/113 , C12N15/80 , C12N15/8509 , C12N2310/20 , C12Q1/68
Abstract: Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.
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18.发明公开公开(公告)号：US20240141341A1
公开(公告)日：2024-05-02
申请号：US18279996
申请日：2022-03-01
Applicant: Duke University
Inventor： Charles A. Gersbach , Alejandro Barrera , Tyler S. Klann , Maria ter Weele , Gregory E. Crawford , Timothy E. Reddy
IPC: C12N15/113 , A61K38/00 , C12N9/22
CPC classification number: C12N15/113 , C12N9/22 , A61K38/00 , C12N2310/20 , C12N2320/12
Abstract: Disclosed herein am compositions and methods for targeting a novel regulatory element of agene. The compositions may be used in methods of modifying growth of a cell, decreasing cell fitness, increasing cell fitness, and/or treating cancer such as leukemia.
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19.发明公开公开(公告)号：US20240058425A1
公开(公告)日：2024-02-22
申请号：US18180718
申请日：2023-03-08
Applicant: Duke University
Inventor： Charles A. Gersbach , Alejandro Barrera , Tyler S. Klann , Maria ter Weele , Gregory E. Crawford , Timothy E. Reddy
IPC: A61K38/46 , C12N15/11 , C12N9/22 , C12N15/90 , C12N15/86 , C12N15/10 , C12N5/09 , A61K31/7088 , A61P35/02
CPC classification number: A61K38/465 , C12N15/11 , C12N9/22 , C12N15/907 , C12N15/86 , C12N15/1082 , C12N5/0694 , A61K31/7088 , A61P35/02 , C12N2310/20 , C12N2800/80 , C12N2740/15043
Abstract: Disclosed herein are compositions and methods for targeting a novel regulatory element of a gene. The compositions may be used in methods of modifying growth of a cell, decreasing cell fitness, increasing cell fitness, and/or treating cancer such as leukemia.
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公开(公告)号：US20230383297A1
公开(公告)日：2023-11-30
申请号：US18030745
申请日：2021-10-08
Applicant: Duke University
Inventor： Charles A. Gersbach , Joshua B. Black
IPC: C12N15/113 , C12N15/11 , C12N9/22
CPC classification number: C12N15/1137 , C12N15/11 , C12N9/22 , C12N2310/20
Abstract: Disclosed herein are compositions and methods for inhibiting a gene selected from OGDH, LIPT1, SDHC, and DHRS7B. The inhibitors may be used to activate SNRPN, SPA1, SPA2, or SNORD118, or a combination thereof. The inhibitors may also be used to treat a subject having Prader Willi Syndrome (PWS) or a PWS-like disorder.
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