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公开(公告)号：US20240026352A1
公开(公告)日：2024-01-25
申请号：US18036862
申请日：2021-11-12
Applicant: Duke University
Inventor： Charles A. Gersbach , Sean McCutcheon
CPC classification number: C12N15/11 , C12N9/22 , C07K14/4702 , C12N15/907 , C12N2310/20 , C12N2800/80 , C07K2319/80
Abstract: Disclosed herein are CRISPR/Cas systems comprising a fusion protein and at least one gRNA targeting a gene or a regulatory element thereof in a cell such as an immune cell, and vector compositions encoding the same. The systems and compositions may be used in methods of modulating expression of a gene in a cell such as an immune cell, as well as in methods of treating a disease such as cancer, autoimmune diseases, or viral infections.
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公开(公告)号：US20220396790A1
公开(公告)日：2022-12-15
申请号：US17633467
申请日：2020-08-08
Applicant: Duke University
Inventor： Charles A. Gersbach , Matthew Gemberling , Tyler Klann
Abstract: Disclosed herein are methods of identifying or engineering a polynucleotide sequence for directing tissue-specific gene expression. The methods may further include creating a regulatory element fragment library. Further disclosed are vectors comprising a tissue-specific regulatory element identified by the methods.
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公开(公告)号：US20220307015A1
公开(公告)日：2022-09-29
申请号：US17636750
申请日：2020-08-19
Applicant: Duke University
Inventor： Charles A. Gersbach , Joshua B. Black , Jennifer Kwon , Shaunak Adkar
IPC: C12N15/10 , C07K14/47 , C12N15/11 , C12N9/22 , C12N5/0793
Abstract: Disclosed herein are compositions, methods, and systems for selecting a polynucleotide for activity as a neuronal-specific transcription factor. The system may include a polynucleotide encoding a reporter protein and a pan-neuronal marker, a Gas protein, and a library of guide RNAs (gRNAs) targeting putative transcription factors. Further provided are methods of screening for a neuronal-specific transcription factor.
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公开(公告)号：US11427817B2
公开(公告)日：2022-08-30
申请号：US15754861
申请日：2016-08-25
Applicant: Duke University
Inventor： Eric Josephs , Dewran Kocak , Piotr Marszalek , Charles A. Gersbach
Abstract: Disclosed herein are optimized guide RNAs (gRNAs) that have increased target binding specificity and reduced off-target binding. Further disclosed herein are methods of designing and using the optimized gRNAs.
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公开(公告)号：US20200347105A1
公开(公告)日：2020-11-05
申请号：US16963034
申请日：2019-01-19
Applicant: Duke University , North Carolina State University
Inventor： Charles A. Gersbach , Adrian Pickar Oliver , Rodolphe Barrangou
IPC: C07K14/47 , C12N15/11 , C12N9/22 , C12N15/90 , C07K14/245
Abstract: Disclosed herein are Type I Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) programmable systems and methods of using said Type I CRISPR/Cas programmable systems for activating gene expression, repressing gene expression, and gene editing. The disclosure relates to compositions that include Type I CRISPR-Cas fusion proteins designed for transcriptional activation, transcriptional repression, and/or gene editing of target genes in eukaryotic cells. The disclosure relates to Type I Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) transcriptional activation system related compositions and methods of using said Type I CRISPR/Cas transcriptional activation system related compositions for activating gene expression. The disclosure relates to compositions that include a Type I CRISPR-Cas fusion protein designed for transcriptional activation of target genes in eukaryotic cells.
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Patent Agency Ranking
6.发明申请公开(公告)号：US20190194633A1
公开(公告)日：2019-06-27
申请号：US16322234
申请日：2017-08-10
Applicant: Duke University
Inventor： Charles A. Gersbach , Joseph J. Bellucci
IPC: C12N9/22 , C12N15/11 , C12N15/10 , C12N5/0783 , C12N9/10
CPC classification number: C12N9/22 , C07K14/4702 , C07K19/00 , C07K2319/00 , C12N5/0636 , C12N5/0637 , C12N9/1029 , C12N9/24 , C12N15/1093 , C12N15/11 , C12N15/113 , C12N2310/20 , C12N2310/3513 , C12N2320/32 , C12N2501/04 , C12N2501/73 , C12N2501/998 , C12N2506/11 , C12N2510/00 , C12N2800/80 , C12Y203/01048
Abstract: Disclosed herein are compositions and methods for programming immune cell function though targeted gene regulation.
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公开(公告)号：US20190127713A1
公开(公告)日：2019-05-02
申请号：US16093272
申请日：2017-04-13
Applicant: Duke University
Inventor： Charles A. Gersbach , Pratiksha I. Thakore
Abstract: The present disclosure provides Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
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公开(公告)号：US20180201951A1
公开(公告)日：2018-07-19
申请号：US15528296
申请日：2015-11-20
Applicant: Duke University
Inventor： Farshid Guilak , Jonathan M. Brunger , Charles A. Gersbach
CPC classification number: C12N15/85 , A61K35/28 , A61K38/1793 , A61K48/005 , A61P25/28 , C12N15/111 , C12N2310/20 , C12N2320/30
Abstract: Disclosed herein are compositions and methods for cell therapy comprising an engineered cell. The present invention is directed to a composition for treating a subject having or suspected of having a disease, the composition comprising a modified cell comprising a modified endogenous gene, wherein an endogenous gene or fragment thereof is replaced with a transgene using a CRISPR/Cas9 system to generate the modified endogenous gene, the modified cell having an altered response to a cell signal or stimulus.
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公开(公告)号：US12215345B2
公开(公告)日：2025-02-04
申请号：US15789348
申请日：2017-10-20
Applicant: Duke University
Inventor： Pablo Perez-Pinera , Charles A. Gersbach
Abstract: Disclosed herein are compositions of transcription activator-like effectors transcription factors and methods of using the compositions for inducing gene expression of mammalian genes.
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公开(公告)号：US12214056B2
公开(公告)日：2025-02-04
申请号：US16318745
申请日：2017-07-19
Applicant: Duke University
Inventor： Charles A. Gersbach , Sarina Madhavan , Christopher Nelson
IPC: A61K48/00 , A61K31/7105 , A61K38/46 , A61P21/00 , C07K14/47 , C12N9/22 , C12N15/11 , C12N15/113 , C12N15/90
Abstract: Disclosed herein are therapeutic applications of CRISPR/Cpf1-based genome editing.
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