CN114555084A - 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法 - Google Patents

用于治疗SCA1的转基因和内含子衍生miRNA联合疗法 Download PDF

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Publication number
CN114555084A
CN114555084A CN202080072237.4A CN202080072237A CN114555084A CN 114555084 A CN114555084 A CN 114555084A CN 202080072237 A CN202080072237 A CN 202080072237A CN 114555084 A CN114555084 A CN 114555084A
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aav
promoter
nucleic acid
sequence
acid molecule
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Chinese (zh)
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B·L·戴维森
E·卡雷尔
A·M·莫内斯
M·S·凯泽
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Children's Hospital of Philadelphia
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Children's Hospital of Philadelphia
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202080072237.4A 2019-08-15 2020-08-14 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法 Pending CN114555084A (zh)

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 (1)

Publication Number Publication Date
CN114555084A true CN114555084A (zh) 2022-05-27

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CN202080072237.4A Pending CN114555084A (zh) 2019-08-15 2020-08-14 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法

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

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Publication number Priority date Publication date Assignee Title
US10820847B1 (en) 2019-08-15 2020-11-03 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
CN120603600A (zh) * 2023-01-19 2025-09-05 优尼科生物制药有限公司 基因递送媒介物的药物配制品

Citations (3)

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US20170298350A1 (en) * 2014-10-03 2017-10-19 Hocuslocus, Llc Methods and compositions for use of non-coding rna in cell culturing and selection
US20170335318A1 (en) * 2002-08-05 2017-11-23 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
US20180169269A1 (en) * 2015-05-29 2018-06-21 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases

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US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
JP6091435B2 (ja) * 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
PT3628334T (pt) * 2014-03-21 2023-09-26 Genzyme Corp Terapia genética para retinopatia pigmentar
AU2015264263B2 (en) * 2014-05-20 2021-08-05 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
CA2977607A1 (en) * 2015-02-26 2016-09-01 Jacob Schneiderman Methods and compositions relating to leptin antagonists
IL302455A (en) * 2016-03-01 2023-06-01 Univ Florida Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
US20170335318A1 (en) * 2002-08-05 2017-11-23 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
US20170298350A1 (en) * 2014-10-03 2017-10-19 Hocuslocus, Llc Methods and compositions for use of non-coding rna in cell culturing and selection
US20180169269A1 (en) * 2015-05-29 2018-06-21 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases

Non-Patent Citations (1)

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MEGAN S. KEISER等: "RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1", 《NEUROBIOLOGY OF DISEASE》, vol. 56, pages 6 - 13, XP028560218, DOI: 10.1016/j.nbd.2013.04.003 *

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AU2020330108A1 (en) 2022-03-17
JP2022545378A (ja) 2022-10-27
CA3151122A1 (en) 2021-02-18
US20220288101A1 (en) 2022-09-15
WO2021030745A1 (en) 2021-02-18
BR112022002794A2 (pt) 2022-08-09
EP4013414A4 (en) 2023-09-27
EP4013414A1 (en) 2022-06-22
MX2022001984A (es) 2022-05-19

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