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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CN201980063972.6A
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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
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P25/00—Drugs for disorders of the nervous system
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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
    • C12N2310/10—Type of nucleic acid
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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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  • Psychiatry (AREA)
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  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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 (enExample)
EP (1) EP3830265A1 (enExample)
JP (2) JP2021534814A (enExample)
KR (1) KR20210062627A (enExample)
CN (1) CN112805382A (enExample)
AR (1) AR114540A1 (enExample)
AU (1) AU2019314529B2 (enExample)
BR (1) BR112021001980A2 (enExample)
CA (1) CA3108526A1 (enExample)
CO (1) CO2021002637A2 (enExample)
IL (1) IL280581A (enExample)
MX (1) MX2021001395A (enExample)
PH (1) PH12021550254A1 (enExample)
SG (1) SG11202101072UA (enExample)
TW (2) TW202509215A (enExample)
WO (1) WO2020028816A1 (enExample)

Cited By (1)

* 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

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PH12021550254A1 (en) 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein
CN115777021A (zh) * 2020-03-18 2023-03-10 马萨诸塞大学 用于snca调节的寡核苷酸
WO2022072447A1 (en) * 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
EP4359525A1 (en) * 2021-06-21 2024-05-01 uniQure biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
AU2023248463A1 (en) * 2022-04-06 2024-11-28 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
JP2025531512A (ja) * 2022-09-29 2025-09-19 アタランタ セラピューティクス，インコーポレーテッド アルファシヌクレイン核酸を標的化するｓｉＲＮＡ組成物及び方法
WO2024211480A2 (en) * 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
TW202529779A (zh) * 2024-01-30 2025-08-01 大陸商上海舶望製藥有限公司 用於抑制突觸核蛋白α（SNCA）基因表達的組合物和方法

Citations (5)

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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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EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
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CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
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BR112014021104B1 (pt) 2012-02-29 2023-03-28 Sangamo Biosciences, Inc Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington
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Publication number Priority date Publication date Assignee Title
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
SG11202101072UA (en) 2021-03-30
MX2021001395A (es) 2021-08-11
US20210309999A1 (en) 2021-10-07
PH12021550254A1 (en) 2023-02-06
CO2021002637A2 (es) 2021-07-30
WO2020028816A1 (en) 2020-02-06
BR112021001980A2 (pt) 2021-05-04
JP2021534814A (ja) 2021-12-16
JP2024156715A (ja) 2024-11-06
US12365895B2 (en) 2025-07-22
AU2019314529A1 (en) 2021-03-25
AR114540A1 (es) 2020-09-16
IL280581A (en) 2021-03-25
TW202022116A (zh) 2020-06-16
KR20210062627A (ko) 2021-05-31
CA3108526A1 (en) 2020-02-06
TW202509215A (zh) 2025-03-01
EP3830265A1 (en) 2021-06-09

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