CN112805382A - 针对α-突触核蛋白的变体RNAi - Google Patents

针对α-突触核蛋白的变体RNAi Download PDF

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Publication number
CN112805382A
CN112805382A CN201980063972.6A CN201980063972A CN112805382A CN 112805382 A CN112805382 A CN 112805382A CN 201980063972 A CN201980063972 A CN 201980063972A CN 112805382 A CN112805382 A CN 112805382A
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nucleic acid
aav
rnai
promoter
sequence
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Pending
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CN201980063972.6A
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English (en)
Chinese (zh)
Inventor
B·埃尔默
B·理查兹
M·拉塔·马希欧
M·G·奥比努
V·陶平
V·布兰查德
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Genzyme Corp
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Genzyme Corp
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    • 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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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase

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  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201980063972.6A 2018-08-03 2019-08-02 针对α-突触核蛋白的变体RNAi Pending CN112805382A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862714616P 2018-08-03 2018-08-03
US62/714,616 2018-08-03
PCT/US2019/044924 WO2020028816A1 (en) 2018-08-03 2019-08-02 Variant rnai against alpha-synuclein

Publications (1)

Publication Number Publication Date
CN112805382A true CN112805382A (zh) 2021-05-14

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CN201980063972.6A Pending CN112805382A (zh) 2018-08-03 2019-08-02 针对α-突触核蛋白的变体RNAi

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US (1) US12365895B2 (https=)
EP (1) EP3830265A1 (https=)
JP (2) JP2021534814A (https=)
KR (1) KR102931619B1 (https=)
CN (1) CN112805382A (https=)
AR (1) AR114540A1 (https=)
AU (1) AU2019314529B2 (https=)
BR (1) BR112021001980A2 (https=)
CA (1) CA3108526A1 (https=)
CO (1) CO2021002637A2 (https=)
IL (1) IL280581A (https=)
MX (1) MX2021001395A (https=)
PH (1) PH12021550254A1 (https=)
SG (1) SG11202101072UA (https=)
TW (2) TW202509215A (https=)
WO (1) WO2020028816A1 (https=)

Cited By (1)

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CN111356763A (zh) * 2017-09-22 2020-06-30 建新公司 变体RNAi

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KR102931619B1 (ko) 2018-08-03 2026-02-26 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
US20210363524A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for snca modulation
EP4221838A4 (en) * 2020-10-01 2025-04-16 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
WO2022268835A1 (en) * 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
WO2023196862A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
US20260103703A1 (en) * 2022-09-29 2026-04-16 Atalanta Therapeutics, Inc. siRNA COMPOSITIONS AND METHODS TARGETING ALPHA-SYNUCLEIN NUCLEIC ACIDS
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025162214A1 (en) * 2024-01-30 2025-08-07 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of synuclein alpha (snca) gene

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US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
WO2010029303A1 (en) * 2008-09-12 2010-03-18 Isis Innovation Limited Gene silencing
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
WO2012068405A2 (en) * 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN107438671A (zh) * 2015-02-10 2017-12-05 建新公司 变体RNAi

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US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
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US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
WO2010029303A1 (en) * 2008-09-12 2010-03-18 Isis Innovation Limited Gene silencing
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
WO2012068405A2 (en) * 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN107438671A (zh) * 2015-02-10 2017-12-05 建新公司 变体RNAi

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111356763A (zh) * 2017-09-22 2020-06-30 建新公司 变体RNAi
CN111356763B (zh) * 2017-09-22 2024-03-12 建新公司 变体RNAi
US12331296B2 (en) 2017-09-22 2025-06-17 Genzyme Corporation Variant RNAi

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AU2019314529B2 (en) 2025-09-11
WO2020028816A1 (en) 2020-02-06
EP3830265A1 (en) 2021-06-09
TW202022116A (zh) 2020-06-16
JP2021534814A (ja) 2021-12-16
KR102931619B1 (ko) 2026-02-26
PH12021550254A1 (en) 2023-02-06
IL280581A (en) 2021-03-25
JP2024156715A (ja) 2024-11-06
AU2019314529A1 (en) 2021-03-25
BR112021001980A2 (pt) 2021-05-04
AR114540A1 (es) 2020-09-16
CO2021002637A2 (es) 2021-07-30
US12365895B2 (en) 2025-07-22
CA3108526A1 (en) 2020-02-06
KR20210062627A (ko) 2021-05-31
TW202509215A (zh) 2025-03-01
US20210309999A1 (en) 2021-10-07
MX2021001395A (es) 2021-08-11
SG11202101072UA (en) 2021-03-30

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