KR20240169111A - Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법 - Google Patents

Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법 Download PDF

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KR20240169111A
KR20240169111A KR1020247036731A KR20247036731A KR20240169111A KR 20240169111 A KR20240169111 A KR 20240169111A KR 1020247036731 A KR1020247036731 A KR 1020247036731A KR 20247036731 A KR20247036731 A KR 20247036731A KR 20240169111 A KR20240169111 A KR 20240169111A
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South Korea
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sequence
seq
nucleic acid
rnai
expression cassette
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Korean (ko)
Inventor
성 청
사라 멜리사 야코보
다카코 모리구치
캐서린 오리오던
궈샹 루안
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젠자임 코포레이션
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    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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KR1020247036731A 2022-04-06 2023-04-05 Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법 Pending KR20240169111A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263328241P 2022-04-06 2022-04-06
US63/328,241 2022-04-06
US202363483075P 2023-02-03 2023-02-03
US63/483,075 2023-02-03
PCT/US2023/065388 WO2023196862A1 (en) 2022-04-06 2023-04-05 Targeted gene therapy for dm-1 myotonic dystrophy

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KR20240169111A true KR20240169111A (ko) 2024-12-02

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KR1020247036731A Pending KR20240169111A (ko) 2022-04-06 2023-04-05 Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법

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US (1) US20230365968A1 (https=)
EP (1) EP4504936A1 (https=)
JP (1) JP2025513785A (https=)
KR (1) KR20240169111A (https=)
CN (1) CN119234040A (https=)
AU (1) AU2023248463A1 (https=)
CA (1) CA3247417A1 (https=)
CO (1) CO2024013743A2 (https=)
IL (1) IL316078A (https=)
MX (1) MX2024012308A (https=)
TW (1) TW202345914A (https=)
WO (1) WO2023196862A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025137378A1 (en) * 2023-12-20 2025-06-26 Aavantibio, Inc. Aav capsid engineering for cardiac and/or musculoskeletal gene therapy

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US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2152874A2 (en) 2007-04-26 2010-02-17 University of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
DK2164967T3 (en) 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
EP2875133B1 (en) 2012-07-17 2018-01-10 Université de Genève Nucleic acids for down-regulation of gene expression
WO2018178067A1 (en) * 2017-03-27 2018-10-04 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
JP2020518258A (ja) * 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
WO2018226638A1 (en) * 2017-06-05 2018-12-13 The Brigham And Women's Hospital, Inc. Vero cell lines stably expressing hsv icp0 protein
EP3765624A4 (en) 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
US20210363193A1 (en) 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
BR112020023298A2 (pt) * 2018-05-15 2021-03-09 University Of Washington composições e métodos para reduzir a spliceopatia e tratamento de distúrbios de dominância de rna
KR102931619B1 (ko) * 2018-08-03 2026-02-26 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
JP2021534755A (ja) * 2018-08-22 2021-12-16 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
CA3164335A1 (en) * 2020-01-10 2021-07-15 Fatih Ozsolak Viral vector for combination therapy

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EP4504936A1 (en) 2025-02-12
US20230365968A1 (en) 2023-11-16
CA3247417A1 (en) 2023-10-12
TW202345914A (zh) 2023-12-01
MX2024012308A (es) 2024-11-08
CO2024013743A2 (es) 2024-10-31
WO2023196862A1 (en) 2023-10-12
IL316078A (en) 2024-12-01
CN119234040A (zh) 2024-12-31
JP2025513785A (ja) 2025-04-30
AU2023248463A1 (en) 2024-11-28

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