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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CN202080072237.4A
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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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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2750/00011—Details
    • 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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  • Health & Medical Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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

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)

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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

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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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
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
CA2977607A1 (en) * 2015-02-26 2016-09-01 Jacob Schneiderman Methods and compositions relating to leptin antagonists
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

Patent Citations (3)

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

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