MX2021014460A - Composiciones y metodos para la regulacion genica selectiva. - Google Patents

Composiciones y metodos para la regulacion genica selectiva.

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Publication number
MX2021014460A
MX2021014460A MX2021014460A MX2021014460A MX2021014460A MX 2021014460 A MX2021014460 A MX 2021014460A MX 2021014460 A MX2021014460 A MX 2021014460A MX 2021014460 A MX2021014460 A MX 2021014460A MX 2021014460 A MX2021014460 A MX 2021014460A
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MX
Mexico
Prior art keywords
compositions
methods
gene regulation
selective gene
selective
Prior art date
Application number
MX2021014460A
Other languages
English (en)
Inventor
Kartik Ramamoorthi
Andrew Young
Stephanie Tagliatela
David Oberkofler
Anne Tanenhaus
Original Assignee
Encoded Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Encoded Therapeutics Inc filed Critical Encoded Therapeutics Inc
Publication of MX2021014460A publication Critical patent/MX2021014460A/es

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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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Abstract

En la presente se proporcionan factores de transcripción modificados para la regulación por aumento selectiva de SCN1a y usos de los mismos para tratar enfermedades y trastornos, tal como, síndrome de Dravet. También se proporcionan sitios de unión de microARN y usos de los mismos para la expresión selectiva en neuronas de parvalbúmina.
MX2021014460A 2019-05-29 2020-05-29 Composiciones y metodos para la regulacion genica selectiva. MX2021014460A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962854238P 2019-05-29 2019-05-29
US201962857727P 2019-06-05 2019-06-05
US202063008569P 2020-04-10 2020-04-10
PCT/US2020/035431 WO2020243651A1 (en) 2019-05-29 2020-05-29 Compositions and methods for selective gene regulation

Publications (1)

Publication Number Publication Date
MX2021014460A true MX2021014460A (es) 2022-02-11

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MX2021014460A MX2021014460A (es) 2019-05-29 2020-05-29 Composiciones y metodos para la regulacion genica selectiva.

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US (1) US20220136009A1 (es)
EP (1) EP3976798A4 (es)
JP (1) JP7432621B2 (es)
KR (1) KR20220066225A (es)
CN (1) CN114174520A (es)
AU (2) AU2020282352B2 (es)
BR (1) BR112021024055A2 (es)
CA (1) CA3141900C (es)
CL (1) CL2021003151A1 (es)
CO (1) CO2021017544A2 (es)
IL (1) IL288313A (es)
MX (1) MX2021014460A (es)
SG (1) SG11202113048SA (es)
TW (1) TW202113084A (es)
WO (1) WO2020243651A1 (es)

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AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
EP3952924A4 (en) * 2019-04-12 2023-05-24 Encoded Therapeutics, Inc. COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS
CN117980484A (zh) * 2021-09-16 2024-05-03 诺华股份有限公司 新颖的转录因子
EP4151736A1 (en) * 2021-09-21 2023-03-22 SVAR Life Science AB Novel system for the quantification of anti-aav neutralizing antibodies
WO2023129940A1 (en) * 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof
CN114507693A (zh) * 2022-02-09 2022-05-17 中国人民解放军陆军军医大学第一附属医院 重组腺相关病毒表达载体及其应用

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EP2046806A4 (en) * 2006-07-05 2009-10-21 Scripps Research Inst CHIMERIC RECOMBINASES WITH ZINC FINGERS OPTIMIZED FOR CATALYSIS BY DIRECT EVOLUTION
US9526784B2 (en) * 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2017180915A2 (en) * 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CN109890962A (zh) * 2016-09-07 2019-06-14 旗舰创业股份有限公司 用于调节基因表达的方法和组合物
JP2018088888A (ja) * 2016-12-06 2018-06-14 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法
SG11201909203WA (en) * 2017-04-03 2019-11-28 Encoded Therapeutics Inc Tissue selective transgene expression
JP7401432B2 (ja) * 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
WO2019224864A1 (ja) * 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法とそれによるドラベ症候群の治療法

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AU2020282352B2 (en) 2023-05-18
IL288313A (en) 2022-01-01
US20220136009A1 (en) 2022-05-05
AU2023204555A1 (en) 2023-08-03
CA3141900A1 (en) 2020-12-03
CN114174520A (zh) 2022-03-11
AU2020282352A1 (en) 2021-12-23
EP3976798A1 (en) 2022-04-06
BR112021024055A2 (pt) 2022-02-08
SG11202113048SA (en) 2021-12-30
CL2021003151A1 (es) 2022-07-29
JP2022535749A (ja) 2022-08-10
JP7432621B2 (ja) 2024-02-16
CA3141900C (en) 2023-06-06
EP3976798A4 (en) 2023-07-12
TW202113084A (zh) 2021-04-01
WO2020243651A1 (en) 2020-12-03
CO2021017544A2 (es) 2022-01-17
KR20220066225A (ko) 2022-05-24

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