JP2021523696A - Dnaメチル化の標的化された編集によるsnca発現の下方調節 - Google Patents

Dnaメチル化の標的化された編集によるsnca発現の下方調節 Download PDF

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JP2021523696A
JP2021523696A JP2020558973A JP2020558973A JP2021523696A JP 2021523696 A JP2021523696 A JP 2021523696A JP 2020558973 A JP2020558973 A JP 2020558973A JP 2020558973 A JP2020558973 A JP 2020558973A JP 2021523696 A JP2021523696 A JP 2021523696A
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snca
fusion protein
seq
grna
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オーニット チバ−ファレク
オーニット チバ−ファレク
ボリス カンター
ボリス カンター
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Duke University
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    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2020558973A 2018-04-23 2019-04-23 Dnaメチル化の標的化された編集によるsnca発現の下方調節 Pending JP2021523696A (ja)

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Application Number Priority Date Filing Date Title
JP2024019364A JP2024073420A (ja) 2018-04-23 2024-02-13 Dnaメチル化の標的化された編集によるsnca発現の下方調節

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862661134P 2018-04-23 2018-04-23
US62/661,134 2018-04-23
US201862676149P 2018-05-24 2018-05-24
US62/676,149 2018-05-24
US201962789932P 2019-01-08 2019-01-08
US62/789,932 2019-01-08
US201962824195P 2019-03-26 2019-03-26
US62/824,195 2019-03-26
PCT/US2019/028786 WO2019209869A2 (en) 2018-04-23 2019-04-23 Downregulation of snca expression by targeted editing of dna-methylation

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JP2021523696A true JP2021523696A (ja) 2021-09-09
JP2021523696A5 JP2021523696A5 (https=) 2022-05-06

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CN (1) CN112166194A (https=)
AU (1) AU2019261361A1 (https=)
CA (1) CA3097755A1 (https=)
WO (1) WO2019209869A2 (https=)

Families Citing this family (9)

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KR102922694B1 (ko) 2018-04-19 2026-02-03 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 유전자 편집을 위한 조성물 및 방법
SG11202102012SA (en) * 2018-08-28 2021-04-29 Pharos Vaccine Inc Improved lentiviral vector
SG11202111031RA (en) 2019-04-26 2021-11-29 Allogene Therapeutics Inc Rituximab-resistant chimeric antigen receptors and uses thereof
WO2021007457A1 (en) * 2019-07-09 2021-01-14 Urfer Buchwalder Anne Use of snca-mediated genes for diagnosis and treatment of parkinson's disease
GB202010075D0 (en) * 2020-07-01 2020-08-12 Imp College Innovations Ltd Therapeutic nucleic acids, peptides and uses
JP2024518553A (ja) * 2021-05-13 2024-05-01 フォージ バイオロジクス,インコーポレイテッド アデノウイルスヘルパープラスミド
CN114790463B (zh) * 2022-04-15 2023-10-13 中山大学 稳定转染CRISPR/dCas9系统的单克隆细胞株的构建方法及其应用
EP4342988A1 (en) 2022-09-20 2024-03-27 Eberhard Karls Universität Tübingen, Medizinische Fakultät Compositions and methods for modification of the expression of a snca gene
CN120230839A (zh) * 2025-04-30 2025-07-01 北京凯祥弘康生物科技有限公司 一种检测snca基因dna甲基化水平的引物探针组合物及其应用

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Rnaガイド遺伝子編集及び遺伝子調節
JP2016521995A (ja) * 2013-06-17 2016-07-28 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのCRISPR−Cas系および組成物の送達、使用および治療上の適用
WO2016205711A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016205749A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
JP2017501149A (ja) * 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのcrispr−cas系及び組成物の送達、使用及び治療適用
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
WO2018035495A1 (en) * 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016130600A2 (en) * 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
EP3091087A1 (en) * 2015-05-08 2016-11-09 Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts
EP3585807A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Rnaガイド遺伝子編集及び遺伝子調節
JP2016521995A (ja) * 2013-06-17 2016-07-28 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのCRISPR−Cas系および組成物の送達、使用および治療上の適用
JP2017501149A (ja) * 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのcrispr−cas系及び組成物の送達、使用及び治療適用
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
WO2016205711A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016205749A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2018035495A1 (en) * 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
AHMAD JOWAED ET AL., THE JOURNAL OF NEUROSCIENCE, vol. 30, no. 18, JPN6023018137, 2010, pages 6355 - 6359, ISSN: 0005342000 *
LUMINE MATSUMOTO ET AL., PLOS ONE, vol. Vol. 5, Issue 11, e15522, JPN6023018135, 2010, pages 1 - 9, ISSN: 0005052413 *
SUBHRANGSHU GUHATHAKURTA ET AL., PROGRESS IN NEUROBIOLOGY, vol. 154, JPN6023018136, 2017, pages 21 - 36, ISSN: 0005342001 *

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WO2019209869A2 (en) 2019-10-31
CN112166194A (zh) 2021-01-01
CA3097755A1 (en) 2019-10-31
US20210189361A1 (en) 2021-06-24
JP2024073420A (ja) 2024-05-29
AU2019261361A1 (en) 2020-11-19
EP3784785A4 (en) 2022-02-16
WO2019209869A3 (en) 2019-12-26
EP3784785A2 (en) 2021-03-03

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