BR112021023899A2 - Método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk - Google Patents

Método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk

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Publication number
BR112021023899A2
BR112021023899A2 BR112021023899A BR112021023899A BR112021023899A2 BR 112021023899 A2 BR112021023899 A2 BR 112021023899A2 BR 112021023899 A BR112021023899 A BR 112021023899A BR 112021023899 A BR112021023899 A BR 112021023899A BR 112021023899 A2 BR112021023899 A2 BR 112021023899A2
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BR
Brazil
Prior art keywords
seq
treatment
muscular dystrophy
targeting
dmpk gene
Prior art date
Application number
BR112021023899A
Other languages
English (en)
Inventor
Eiji Yoshimi
M Connolly Keith
Tetsuya Yamagata
Tomoya Oe
Original Assignee
Astellas Pharma Inc
Modalis Therapeutics Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Astellas Pharma Inc, Modalis Therapeutics Corp filed Critical Astellas Pharma Inc
Publication of BR112021023899A2 publication Critical patent/BR112021023899A2/pt

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Abstract

método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk. espera-se que os polinucleotídeos que compreendem as seguintes sequências de bases: (a) uma sequência de bases que codifica uma proteína de fusão de uma proteína efetora de crispr deficiente como nuclease e um repressor transcricional, e (b) uma sequência de bases que codifica um rna-guia direcionado a uma região contínua de 18 a 24 nucleotídeos de comprimento, em uma região mostrada na seq id no: 127, seq id no: 46, seq id no: 128, seq id no: 129, seq id no: 130, seq id no: 131, seq id no: 132, seq id no: 88, seq id no: 91, seq id no: 133, seq id no: 137, seq id no: 117 ou seq id no: 119, em uma região reguladora da expressão de um gene da dmpk humano, sejam úteis para o tratamento da distrofia muscular.
BR112021023899A 2019-05-28 2020-05-27 Método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk BR112021023899A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962853373P 2019-05-28 2019-05-28
US202063025417P 2020-05-15 2020-05-15
PCT/JP2020/021851 WO2020241903A1 (en) 2019-05-28 2020-05-27 Method for treating muscular dystrophy by targeting dmpk gene

Publications (1)

Publication Number Publication Date
BR112021023899A2 true BR112021023899A2 (pt) 2022-01-18

Family

ID=71787010

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021023899A BR112021023899A2 (pt) 2019-05-28 2020-05-27 Método para o tratamento da distrofia muscular por direcionamento ao gene da dmpk

Country Status (14)

Country Link
US (1) US20220233721A1 (pt)
EP (1) EP3976785A1 (pt)
JP (1) JP2022534627A (pt)
KR (1) KR20220012926A (pt)
CN (1) CN113785066B (pt)
AU (1) AU2020283372A1 (pt)
BR (1) BR112021023899A2 (pt)
CA (1) CA3135271A1 (pt)
CO (1) CO2021014746A2 (pt)
IL (1) IL287178A (pt)
MX (1) MX2021014478A (pt)
SG (1) SG11202111427RA (pt)
TW (1) TW202111122A (pt)
WO (1) WO2020241903A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022114243A1 (en) * 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
WO2022234519A1 (en) * 2021-05-05 2022-11-10 Crispr Therapeutics Ag Compositions and methods for using sacas9 scaffold sequences
WO2023018637A1 (en) * 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements

Family Cites Families (11)

* 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
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2292780T3 (en) 2003-09-30 2017-12-04 Univ Pennsylvania Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof
CN106434648A (zh) * 2010-07-19 2017-02-22 F·C·贝内特 肌强直性营养障碍蛋白激酶(dmpk)表达的调节
CN103189507A (zh) 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
SG10201700751XA (en) * 2012-09-25 2017-03-30 Genzyme Corp Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
TW201642837A (zh) 2015-03-24 2016-12-16 Kyowa Hakko Kirin Co Ltd 含有核酸之脂質奈米粒子
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
WO2018081806A2 (en) * 2016-10-31 2018-05-03 University Of Florida Research Foundation, Inc. Compositions and methods for impeding transcription of expanded microsatellite repeats
EP3805386A4 (en) 2018-06-08 2022-03-23 Modalis Therapeutics Corporation MODIFIED CAS9 PROTEIN AND USE THEREOF
SG11202104097TA (en) 2018-10-24 2021-05-28 Modalis Therapeutics Corp Modified cas9 protein, and use thereof

Also Published As

Publication number Publication date
EP3976785A1 (en) 2022-04-06
AU2020283372A1 (en) 2021-11-18
WO2020241903A1 (en) 2020-12-03
CN113785066A (zh) 2021-12-10
IL287178A (en) 2021-12-01
KR20220012926A (ko) 2022-02-04
JP2022534627A (ja) 2022-08-02
MX2021014478A (es) 2022-01-06
CO2021014746A2 (es) 2021-11-19
US20220233721A1 (en) 2022-07-28
CA3135271A1 (en) 2020-12-03
TW202111122A (zh) 2021-03-16
CN113785066B (zh) 2024-06-18
SG11202111427RA (en) 2021-11-29

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