JP2022545378A5 - - Google Patents

Info

Publication number
JP2022545378A5
JP2022545378A5 JP2022509153A JP2022509153A JP2022545378A5 JP 2022545378 A5 JP2022545378 A5 JP 2022545378A5 JP 2022509153 A JP2022509153 A JP 2022509153A JP 2022509153 A JP2022509153 A JP 2022509153A JP 2022545378 A5 JP2022545378 A5 JP 2022545378A5
Authority
JP
Japan
Prior art keywords
nucleic acid
acid molecule
promoter
expression cassette
molecule according
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2022509153A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022545378A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/046499 external-priority patent/WO2021030745A1/en
Publication of JP2022545378A publication Critical patent/JP2022545378A/ja
Publication of JP2022545378A5 publication Critical patent/JP2022545378A5/ja
Pending legal-status Critical Current

Links

JP2022509153A 2019-08-15 2020-08-14 SCA1の治療のための、導入遺伝子およびイントロン由来miRNAを組み合わせた治療法 Pending JP2022545378A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962887209P 2019-08-15 2019-08-15
US62/887,209 2019-08-15
PCT/US2020/046499 WO2021030745A1 (en) 2019-08-15 2020-08-14 Combined transgene and intron-derived mirna therapy for treatment of sca1

Publications (2)

Publication Number Publication Date
JP2022545378A JP2022545378A (ja) 2022-10-27
JP2022545378A5 true JP2022545378A5 (OSRAM) 2023-08-15

Family

ID=74570777

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022509153A Pending JP2022545378A (ja) 2019-08-15 2020-08-14 SCA1の治療のための、導入遺伝子およびイントロン由来miRNAを組み合わせた治療法

Country Status (9)

Country Link
US (1) US20220288101A1 (OSRAM)
EP (1) EP4013414A4 (OSRAM)
JP (1) JP2022545378A (OSRAM)
CN (1) CN114555084A (OSRAM)
AU (1) AU2020330108A1 (OSRAM)
BR (1) BR112022002794A2 (OSRAM)
CA (1) CA3151122A1 (OSRAM)
MX (1) MX2022001984A (OSRAM)
WO (1) WO2021030745A1 (OSRAM)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11008627B2 (en) 2019-08-15 2021-05-18 Talis Biomedical Corporation Diagnostic system
US20260043024A1 (en) * 2022-08-26 2026-02-12 The Children's Hospital Of Philadelphia Mirnas targeting atnx2 for the treatment of als and sca2
WO2024153769A1 (en) * 2023-01-19 2024-07-25 Uniqure Biopharma B.V. Pharmaceutical formulations of gene delivery vehicles

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7001733B1 (en) * 1998-05-12 2006-02-21 Rigel Pharmaceuticals, Inc. Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
WO2008150897A2 (en) * 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
DK3628334T5 (da) * 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
ES2759317T3 (es) * 2014-05-20 2020-05-08 Univ Iowa Res Found Compuestos terapéuticos para la enfermedad de Huntington
MA40782A (fr) * 2014-10-03 2017-08-08 Hocuslocus Llc Procédés et compositions utilisant un arn non codant en vue de la culture et de la sélection de cellules
CA2977607A1 (en) * 2015-02-26 2016-09-01 Jacob Schneiderman Methods and compositions relating to leptin antagonists
US11027024B2 (en) * 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
US11118185B2 (en) * 2016-03-01 2021-09-14 University Of Florida Research Foundation, Incorporated AAV vectors for treatment of dominant retinitis pigmentosa
CN111132699A (zh) * 2017-09-08 2020-05-08 世代生物公司 修饰的封闭端dna(cedna)
KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
KR20210042123A (ko) * 2018-08-07 2021-04-16 더 칠드런스 호스피탈 오브 필라델피아 유전자 발현의 선택적 스플라이싱 조절 및 치료 방법

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