CN116096885A - 用于靶向C9orf72的组合物和方法 - Google Patents

用于靶向C9orf72的组合物和方法 Download PDF

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CN116096885A
CN116096885A CN202180034169.7A CN202180034169A CN116096885A CN 116096885 A CN116096885 A CN 116096885A CN 202180034169 A CN202180034169 A CN 202180034169A CN 116096885 A CN116096885 A CN 116096885A
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seq
sequence
casx
gna
protein
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CN202180034169.7A
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Chinese (zh)
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B·奥克斯
H·施平纳
S·丹尼
B·T·斯特尔
K·泰勒
K·巴尼
I·科林
M·阿迪尔
C·乌尔内斯
S·希金斯
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Scribe Therapy
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Scribe Therapy
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    • A01K2227/105—Murine
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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CN202180034169.7A 2020-03-18 2021-03-17 用于靶向C9orf72的组合物和方法 Pending CN116096885A (zh)

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

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CN116096885A true CN116096885A (zh) 2023-05-09

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CN (1) CN116096885A (enExample)
AU (1) AU2021237633A1 (enExample)
BR (1) BR112022018673A2 (enExample)
CA (1) CA3172178A1 (enExample)
CO (1) CO2022014598A2 (enExample)
IL (1) IL296477A (enExample)
MX (1) MX2022011460A (enExample)
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SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
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GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
AU2022216614B2 (en) 2021-02-05 2026-03-05 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
US20250313819A1 (en) * 2022-05-12 2025-10-09 The Regents Of The University Of California Therapeutic crispr/cas9 gene editing approaches to the c9orf72 repeat expansion mutation in ipscs
WO2023235725A2 (en) * 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
EP4536286A1 (en) * 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
EP4665848A2 (en) * 2023-02-15 2025-12-24 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
EP4771154A2 (en) 2023-09-01 2026-07-08 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

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JP2019532644A (ja) * 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ誘導型核酸修飾酵素及びその使用方法
US11773409B2 (en) 2017-04-21 2023-10-03 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
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AU2021237633A1 (en) 2022-10-06
JP2023518541A (ja) 2023-05-02
CA3172178A1 (en) 2021-09-23
IL296477A (en) 2022-11-01
WO2021188729A1 (en) 2021-09-23
MX2022011460A (es) 2022-12-15
KR20230002401A (ko) 2023-01-05
BR112022018673A2 (pt) 2022-12-27
JP7847853B2 (ja) 2026-04-20
US20240309344A1 (en) 2024-09-19
EP4121535A1 (en) 2023-01-25
CO2022014598A2 (es) 2022-10-31

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