JP2025515503A5 - - Google Patents

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Publication number
JP2025515503A5
JP2025515503A5 JP2024563706A JP2024563706A JP2025515503A5 JP 2025515503 A5 JP2025515503 A5 JP 2025515503A5 JP 2024563706 A JP2024563706 A JP 2024563706A JP 2024563706 A JP2024563706 A JP 2024563706A JP 2025515503 A5 JP2025515503 A5 JP 2025515503A5
Authority
JP
Japan
Prior art keywords
nucleic acid
acid molecule
promoter
domain
molecule according
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024563706A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025515503A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2023/066389 external-priority patent/WO2023212715A1/en
Publication of JP2025515503A publication Critical patent/JP2025515503A/ja
Publication of JP2025515503A5 publication Critical patent/JP2025515503A5/ja
Pending legal-status Critical Current

Links

JP2024563706A 2022-04-28 2023-04-28 塩基エディタをコードするaavベクターおよびその使用 Pending JP2025515503A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263336064P 2022-04-28 2022-04-28
US63/336,064 2022-04-28
US202263389796P 2022-07-15 2022-07-15
US63/389,796 2022-07-15
PCT/US2023/066389 WO2023212715A1 (en) 2022-04-28 2023-04-28 Aav vectors encoding base editors and uses thereof

Publications (2)

Publication Number Publication Date
JP2025515503A JP2025515503A (ja) 2025-05-15
JP2025515503A5 true JP2025515503A5 (enExample) 2026-05-12

Family

ID=86497459

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024563706A Pending JP2025515503A (ja) 2022-04-28 2023-04-28 塩基エディタをコードするaavベクターおよびその使用

Country Status (7)

Country Link
US (1) US20250064981A1 (enExample)
EP (1) EP4514401A1 (enExample)
JP (1) JP2025515503A (enExample)
CN (1) CN120456933A (enExample)
AU (1) AU2023259642A1 (enExample)
CA (1) CA3250697A1 (enExample)
WO (1) WO2023212715A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2025231247A1 (en) * 2024-05-02 2025-11-06 University Of Massachusetts Editing of aatd-related genes using a nme2 base editor
WO2026072421A2 (en) * 2024-09-27 2026-04-02 The Broad Institute, Inc. Base editing methods and compositions for editing prnp in the treatment of prion disease
CN121278103B (zh) * 2025-12-10 2026-03-17 成都理工大学 基于自然语言处理的媒体画像生成方法及系统

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US8394604B2 (en) 2008-04-30 2013-03-12 Paul Xiang-Qin Liu Protein splicing using short terminal split inteins
JP5723774B2 (ja) 2008-09-05 2015-05-27 プレジデント アンド フェローズ オブ ハーバード カレッジ タンパク質および核酸の連続的指向性進化
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
CA2825370A1 (en) 2010-12-22 2012-06-28 President And Fellows Of Harvard College Continuous directed evolution
CN114634950A (zh) 2012-12-12 2022-06-17 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
EP3097196B1 (en) 2014-01-20 2019-09-11 President and Fellows of Harvard College Negative selection and stringency modulation in continuous evolution systems
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US11299729B2 (en) 2015-04-17 2022-04-12 President And Fellows Of Harvard College Vector-based mutagenesis system
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
CA3048479A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Gene editing of pcsk9
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US11117812B2 (en) 2018-05-24 2021-09-14 Aqua-Aerobic Systems, Inc. System and method of solids conditioning in a filtration system
US20230021641A1 (en) 2018-08-23 2023-01-26 The Broad Institute, Inc. Cas9 variants having non-canonical pam specificities and uses thereof
US20240173430A1 (en) 2018-09-05 2024-05-30 The Broad Institute, Inc. Base editing for treating hutchinson-gilford progeria syndrome
US20220380740A1 (en) 2018-10-24 2022-12-01 The Broad Institute, Inc. Constructs for improved hdr-dependent genomic editing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020102659A1 (en) 2018-11-15 2020-05-22 The Broad Institute, Inc. G-to-t base editors and uses thereof
AU2020221366A1 (en) 2019-02-13 2021-08-26 Beam Therapeutics Inc. Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence
WO2020181180A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to c:g base editors and uses thereof
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3973054A1 (en) 2019-05-20 2022-03-30 The Broad Institute Inc. Aav delivery of nucleobase editors
EP4028026A4 (en) 2019-09-09 2023-09-06 Beam Therapeutics, Inc. NOVEL NUCLEOBASE EDITORS AND METHODS OF USING THE SAME
US20230086199A1 (en) 2019-11-26 2023-03-23 The Broad Institute, Inc. Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
EP4100519A2 (en) 2020-02-05 2022-12-14 The Broad Institute, Inc. Adenine base editors and uses thereof

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