MX2022001859A - Metodo para el tratamiento de la distrofia muscular al fijar como blanco el gen lama1. - Google Patents

Metodo para el tratamiento de la distrofia muscular al fijar como blanco el gen lama1.

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Publication number
MX2022001859A
MX2022001859A MX2022001859A MX2022001859A MX2022001859A MX 2022001859 A MX2022001859 A MX 2022001859A MX 2022001859 A MX2022001859 A MX 2022001859A MX 2022001859 A MX2022001859 A MX 2022001859A MX 2022001859 A MX2022001859 A MX 2022001859A
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MX
Mexico
Prior art keywords
seq
set forth
targeting
muscular dystrophy
guide rna
Prior art date
Application number
MX2022001859A
Other languages
English (en)
Inventor
Tetsuya Yamagata
Yuanbo Qin
Original Assignee
Modalis Therapeutics Corp
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Publication date
Application filed by Modalis Therapeutics Corp filed Critical Modalis Therapeutics Corp
Publication of MX2022001859A publication Critical patent/MX2022001859A/es

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    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
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Abstract

La presente invención tiene como objetivo proporcionar un enfoque terapéutico novedoso para la distrofia muscular humana (particularmente MDC1A). La presente invención proporciona un polinucleótido que comprende las siguientes secuencias de bases: (a) una secuencia de bases que codifica una proteína de fusión de una proteína efectora CRISPR deficiente en nucleasa y un activador de la transcripción, y (b) una secuencia de bases que codifica (i) un ARN guía que fija como diana una región continua establecida en SEQ ID NO: 15, 20, 25, 50, 56 o 61, (ii) un ARN guía que fija como diana una región continua establecida en SEQ ID NO: 124, o (iii) un ARN guía que fija como diana una región continua establecida en SEQ ID NO: 178, 193 o 195, en la región reguladora de la expresión del gen LAMA1 humano.
MX2022001859A 2019-08-16 2020-08-14 Metodo para el tratamiento de la distrofia muscular al fijar como blanco el gen lama1. MX2022001859A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962887863P 2019-08-16 2019-08-16
US202063008059P 2020-04-10 2020-04-10
PCT/JP2020/030864 WO2021033635A1 (en) 2019-08-16 2020-08-14 Method for treating muscular dystrophy by targeting lama1 gene

Publications (1)

Publication Number Publication Date
MX2022001859A true MX2022001859A (es) 2022-03-11

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Application Number Title Priority Date Filing Date
MX2022001859A MX2022001859A (es) 2019-08-16 2020-08-14 Metodo para el tratamiento de la distrofia muscular al fijar como blanco el gen lama1.

Country Status (12)

Country Link
US (1) US20230173036A1 (es)
EP (1) EP4013874A1 (es)
JP (1) JP2022544320A (es)
KR (1) KR20220045013A (es)
CN (1) CN114364802A (es)
AU (1) AU2020334462A1 (es)
BR (1) BR112022002889A2 (es)
CA (1) CA3146751A1 (es)
IL (1) IL290249A (es)
MX (1) MX2022001859A (es)
TW (1) TW202112797A (es)
WO (1) WO2021033635A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022176859A1 (en) * 2021-02-16 2022-08-25 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting lama1 gene
CN118660971A (zh) * 2021-12-30 2024-09-17 里格尔医疗股份有限公司 用于调节钠电压门控通道α亚基1表达的组合物及其用途

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2448120A1 (en) 2001-05-24 2002-11-28 Genzyme Corporation Muscle-specific expression vectors
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
US9015089B2 (en) * 2012-04-17 2015-04-21 The Mitre Corporation Identifying and forecasting shifts in the mood of social media users
AU2016237148A1 (en) 2015-03-24 2017-11-23 Kyowa Hakko Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticles
JP6926825B2 (ja) * 2017-08-25 2021-08-25 沖電気工業株式会社 通信装置、プログラムおよびオペレータ選択方法

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CA3146751A1 (en) 2021-02-25
CN114364802A (zh) 2022-04-15
WO2021033635A1 (en) 2021-02-25
KR20220045013A (ko) 2022-04-12
JP2022544320A (ja) 2022-10-17
EP4013874A1 (en) 2022-06-22
IL290249A (en) 2022-04-01
US20230173036A1 (en) 2023-06-08
AU2020334462A1 (en) 2022-02-24
BR112022002889A2 (pt) 2022-05-17
TW202112797A (zh) 2021-04-01

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