JPWO2021188729A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2021188729A5
JPWO2021188729A5 JP2022556229A JP2022556229A JPWO2021188729A5 JP WO2021188729 A5 JPWO2021188729 A5 JP WO2021188729A5 JP 2022556229 A JP2022556229 A JP 2022556229A JP 2022556229 A JP2022556229 A JP 2022556229A JP WO2021188729 A5 JPWO2021188729 A5 JP WO2021188729A5
Authority
JP
Japan
Prior art keywords
sequence
seq
grna
protein
rna
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.)
Granted
Application number
JP2022556229A
Other languages
English (en)
Japanese (ja)
Other versions
JP7847853B2 (ja
JP2023518541A (ja
JP2023518541A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/022840 external-priority patent/WO2021188729A1/en
Publication of JP2023518541A publication Critical patent/JP2023518541A/ja
Publication of JP2023518541A5 publication Critical patent/JP2023518541A5/ja
Publication of JPWO2021188729A5 publication Critical patent/JPWO2021188729A5/ja
Application granted granted Critical
Publication of JP7847853B2 publication Critical patent/JP7847853B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022556229A 2020-03-18 2021-03-17 C9orf72の標的化のための組成物及び方法 Active JP7847853B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062991403P 2020-03-18 2020-03-18
US62/991,403 2020-03-18
PCT/US2021/022840 WO2021188729A1 (en) 2020-03-18 2021-03-17 Compositions and methods for the targeting of c9orf72

Publications (4)

Publication Number Publication Date
JP2023518541A JP2023518541A (ja) 2023-05-02
JP2023518541A5 JP2023518541A5 (https=) 2024-03-28
JPWO2021188729A5 true JPWO2021188729A5 (https=) 2024-03-28
JP7847853B2 JP7847853B2 (ja) 2026-04-20

Family

ID=75478206

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022556229A Active JP7847853B2 (ja) 2020-03-18 2021-03-17 C9orf72の標的化のための組成物及び方法

Country Status (12)

Country Link
US (1) US20240309344A1 (https=)
EP (1) EP4121535A1 (https=)
JP (1) JP7847853B2 (https=)
KR (1) KR20230002401A (https=)
CN (1) CN116096885A (https=)
AU (1) AU2021237633A1 (https=)
BR (1) BR112022018673A2 (https=)
CA (1) CA3172178A1 (https=)
CO (1) CO2022014598A2 (https=)
IL (1) IL296477A (https=)
MX (1) MX2022011460A (https=)
WO (1) WO2021188729A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
EP4288541A1 (en) 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
EP4423263A4 (en) * 2021-10-29 2026-03-04 Mammoth Biosciences Inc EFFECTOR PROTEINS, COMPOSITIONS, SYSTEMS, DEVICES, KITS AND METHODS FOR THERECOMMENDATION
US20250011766A1 (en) * 2021-11-09 2025-01-09 Prime Medicine, Inc. Genome Editing Compositions and Methods for Treatment of Amyotrophic Lateral Sclerosis
WO2023235725A2 (en) * 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240074A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2024173479A2 (en) * 2023-02-15 2024-08-22 The Trustees Of Columbia University In The City Of New York Compositions and methods for treating motor neuron diseases
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2025024285A1 (en) * 2023-07-21 2025-01-30 Mammoth Biosciences, Inc. Compositions for the modification of the human c9orf72 gene
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
BR112018012894A2 (en) * 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
CA3038960A1 (en) * 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
WO2018208972A1 (en) * 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
WO2019030306A1 (en) * 2017-08-08 2019-02-14 Depixus ISOLATION AND IN VITRO ENRICHMENT OF NUCLEIC ACIDS USING SITE-SPECIFIC NUCLEASES
US11578334B2 (en) * 2017-10-25 2023-02-14 Monsanto Technology Llc Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes
PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems

Similar Documents

Publication Publication Date Title
JPWO2021188729A5 (https=)
JP7832262B2 (ja) 肝特異的ウイルスプロモーター及びその使用方法
US12529051B2 (en) Methods of treating amyotrophic lateral sclerosis (ALS)
JP2024028643A5 (https=)
JP2025028917A (ja) 核酸構築物及び使用方法
US20230121437A1 (en) Rna editor-enhanced rna trans-splicing
JP2022173348A (ja) 高活性制御エレメント
CA3097857A1 (en) Fusion proteins and fusion ribonucleic acids for tracking and manipulating cellular rna
WO2020214609A1 (en) Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
JPWO2020247882A5 (https=)
US20230201375A1 (en) Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1)
EP3294891B1 (en) Polynucleotides, vectors and methods for insertion and expression of transgenes
US20240026381A1 (en) Split prime editing platforms
CN116949012B (zh) 一种融合蛋白及其应用
CA3218209A1 (en) Multiplex crispr/cas9-mediated target gene activation system
WO2024120528A1 (en) Improved system for producing rna-packaged aav particles
WO2024078645A2 (zh) Cas蛋白及其应用
CN115947796B (zh) 一种重组腺相关病毒载体及其在基因递送中的应用
JPWO2022120094A5 (https=)
WO2024050547A2 (en) Compact bidirectional promoters for gene expression
WO2023147558A2 (en) Crispr methods for correcting bag3 gene mutations in vivo
CA3218631A1 (en) Vector system
KR102640462B1 (ko) 신경병증성 통증의 치료를 위한 방법 및 조성물
US20240415984A1 (en) Precise Excisions of Portions of Exons for Treatment of Duchenne Muscular Dystrophy
US20250135032A1 (en) Crispr methods for correcting bag3 gene mutations in vivo