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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    • C12N15/11—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
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    • 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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    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (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 (https=)
EP (1) EP4013414A4 (https=)
JP (1) JP2022545378A (https=)
CN (1) CN114555084A (https=)
AU (1) AU2020330108A1 (https=)
BR (1) BR112022002794A2 (https=)
CA (1) CA3151122A1 (https=)
MX (1) MX2022001984A (https=)
WO (1) WO2021030745A1 (https=)

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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
WO2024044469A1 (en) * 2022-08-26 2024-02-29 The Children's Hospital Of Philadelphia Mirnas targeting atnx2 for the treatment of als and sca2
EP4651883A1 (en) * 2023-01-19 2025-11-26 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
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
EP3146051B8 (en) * 2014-05-20 2019-11-27 University of Iowa Research Foundation Huntington's disease therapeutic compounds
EP3261582B1 (en) * 2015-02-26 2021-01-06 Remodeless CV Ltd. Methods and compositions relating to leptin antagonists
CN115896106A (zh) * 2016-03-01 2023-04-04 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的aav载体
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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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
JP2022545378A (ja) 2022-10-27
WO2021030745A1 (en) 2021-02-18
EP4013414A4 (en) 2023-09-27
EP4013414A1 (en) 2022-06-22
MX2022001984A (es) 2022-05-19
US20220288101A1 (en) 2022-09-15
BR112022002794A2 (pt) 2022-08-09
CA3151122A1 (en) 2021-02-18
AU2020330108A1 (en) 2022-03-17

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