TW202329985A - 用於治療cag重複序列疾病之組合物及方法 - Google Patents

用於治療cag重複序列疾病之組合物及方法 Download PDF

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TW202329985A
TW202329985A TW111137904A TW111137904A TW202329985A TW 202329985 A TW202329985 A TW 202329985A TW 111137904 A TW111137904 A TW 111137904A TW 111137904 A TW111137904 A TW 111137904A TW 202329985 A TW202329985 A TW 202329985A
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seq
substitution
mismatch
double
strand
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TW111137904A
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Chinese (zh)
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菲利浦 詹西克
凱斯 加農
大衛 柯瑞
艾諾拉 柯勒克
梅根 布維特
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美商艾瑞斯醫藥公司
南伊利諾大學董事會
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TW111137904A 2021-10-06 2022-10-05 用於治療cag重複序列疾病之組合物及方法 TW202329985A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163253070P 2021-10-06 2021-10-06
US63/253,070 2021-10-06
US202263339363P 2022-05-06 2022-05-06
US63/339,363 2022-05-06

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TW202329985A true TW202329985A (zh) 2023-08-01

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US (1) US20240425856A1 (https=)
EP (1) EP4413137A4 (https=)
JP (1) JP2024543782A (https=)
AU (1) AU2022358732A1 (https=)
CA (1) CA3232737A1 (https=)
IL (1) IL311638A (https=)
MX (1) MX2024004119A (https=)
TW (1) TW202329985A (https=)
WO (1) WO2023060104A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024253981A1 (en) * 2023-04-05 2025-11-06 Board Of Trustees Of Southern Illinois University Compositions and methods for treating cag repeat diseases
US20240360443A1 (en) * 2023-04-09 2024-10-31 Dystrogen Gene Therapies Inc. Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
AU2024332679A1 (en) * 2023-08-31 2026-04-16 Iris Medicine, Inc. Methods and compositions for treating ggggcc repeat expansion diseases
WO2025137623A1 (en) * 2023-12-22 2025-06-26 Iris Medicine, Inc. Methods and compositions for treating ctg repeat expansion diseases
WO2025213123A1 (en) * 2024-04-05 2025-10-09 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL243776B1 (pl) * 2013-09-02 2023-10-09 Inst Chemii Bioorganicznej Polskiej Akademii Nauk Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG
EP3359667A1 (en) * 2015-10-05 2018-08-15 ProQR Therapeutics II B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
WO2018152523A1 (en) * 2017-02-20 2018-08-23 Northwestern University Use of trinucleotide repeat rnas to treat cancer

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MX2024004119A (es) 2024-12-06
EP4413137A1 (en) 2024-08-14
JP2024543782A (ja) 2024-11-26
US20240425856A1 (en) 2024-12-26
CA3232737A1 (en) 2023-04-13
IL311638A (en) 2024-05-01
WO2023060104A1 (en) 2023-04-13
AU2022358732A1 (en) 2024-04-11
EP4413137A4 (en) 2026-02-11

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