EA202192637A1 - Полинуклеотиды, композиции и способы для экспрессии полипептида - Google Patents

Полинуклеотиды, композиции и способы для экспрессии полипептида

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Publication number
EA202192637A1
EA202192637A1 EA202192637A EA202192637A EA202192637A1 EA 202192637 A1 EA202192637 A1 EA 202192637A1 EA 202192637 A EA202192637 A EA 202192637A EA 202192637 A EA202192637 A EA 202192637A EA 202192637 A1 EA202192637 A1 EA 202192637A1
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Eurasian Patent Office
Prior art keywords
methods
compositions
polynucleotides
polypeptide expression
editing
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Application number
EA202192637A
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English (en)
Inventor
Брэдли Эндрю Мюррей
Кристиан Домбровски
Сет С. Александер
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Интеллиа Терапьютикс, Инк.
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Application filed by Интеллиа Терапьютикс, Инк. filed Critical Интеллиа Терапьютикс, Инк.
Publication of EA202192637A1 publication Critical patent/EA202192637A1/ru

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Abstract

В данном изобретении предложены композиции и способы редактирования генов. В некоторых вариантах реализации предложен полинуклеотид, кодирующий Cas9, который может обеспечивать одно или более из улучшенной эффективности редактирования, пониженной иммуногенности или других преимуществ.
EA202192637A 2019-03-28 2020-03-27 Полинуклеотиды, композиции и способы для экспрессии полипептида EA202192637A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825656P 2019-03-28 2019-03-28
PCT/US2020/025372 WO2020198641A2 (en) 2019-03-28 2020-03-27 Polynucleotides, compositions, and methods for polypeptide expression

Publications (1)

Publication Number Publication Date
EA202192637A1 true EA202192637A1 (ru) 2022-03-18

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EA202192637A EA202192637A1 (ru) 2019-03-28 2020-03-27 Полинуклеотиды, композиции и способы для экспрессии полипептида

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US (1) US20230012687A1 (ru)
EP (1) EP3947670A2 (ru)
JP (1) JP2022527302A (ru)
KR (1) KR20220004649A (ru)
CN (1) CN113993994A (ru)
AU (1) AU2020248470A1 (ru)
BR (1) BR112021019224A2 (ru)
CA (1) CA3135172A1 (ru)
CO (1) CO2021014400A2 (ru)
EA (1) EA202192637A1 (ru)
IL (1) IL286579A (ru)
MA (1) MA55527A (ru)
MX (1) MX2021011757A (ru)
SG (1) SG11202110135YA (ru)
TW (1) TW202102529A (ru)
WO (1) WO2020198641A2 (ru)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7423521B2 (ja) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド
CA3237303A1 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
WO2023133525A1 (en) * 2022-01-07 2023-07-13 Precision Biosciences, Inc. Optimized polynucleotides for protein expression
WO2023154749A2 (en) * 2022-02-09 2023-08-17 The Regents Of The University Of California In vitro and in vivo protein translation via in situ circularized rnas
TW202423959A (zh) * 2022-08-24 2024-06-16 美商步行魚醫療公司 用於治療法布瑞氏症(fabry disease)之組成物及方法

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
WO1993013121A1 (en) 1991-12-24 1993-07-08 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
EP0760008A1 (en) 1994-05-19 1997-03-05 Dako A/S Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
WO2008000632A1 (en) * 2006-06-29 2008-01-03 Dsm Ip Assets B.V. A method for achieving improved polypeptide expression
US8993233B2 (en) 2012-12-12 2015-03-31 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
RU2699523C2 (ru) 2012-12-17 2019-09-05 Президент Энд Фэллоуз Оф Харвард Коллидж Рнк-направляемая инженерия генома человека
US10124065B2 (en) 2013-03-08 2018-11-13 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
US10342761B2 (en) 2014-07-16 2019-07-09 Novartis Ag Method of encapsulating a nucleic acid in a lipid nanoparticle host
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
HUE064227T2 (hu) 2015-09-21 2024-02-28 Trilink Biotechnologies Llc Módszer 5'-sapkával ellátott rns-ek szintetizálására
US20210206818A1 (en) * 2016-01-22 2021-07-08 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
KR102617874B1 (ko) 2016-03-30 2023-12-22 인텔리아 테라퓨틱스, 인크. Crispr/cas 성분을 위한 지질 나노입자 제제
US20200199599A1 (en) * 2016-09-23 2020-06-25 Dsm Ip Assets B.V. A guide-rna expression system for a host cell
WO2018067447A1 (en) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Improved methods for identifying double strand break sites
WO2019067910A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION

Also Published As

Publication number Publication date
KR20220004649A (ko) 2022-01-11
BR112021019224A2 (pt) 2021-11-30
MX2021011757A (es) 2021-12-10
MA55527A (fr) 2022-02-09
CN113993994A (zh) 2022-01-28
AU2020248470A1 (en) 2021-11-11
CA3135172A1 (en) 2020-10-01
JP2022527302A (ja) 2022-06-01
IL286579A (en) 2021-10-31
CO2021014400A2 (es) 2021-11-19
EP3947670A2 (en) 2022-02-09
US20230012687A1 (en) 2023-01-19
WO2020198641A2 (en) 2020-10-01
SG11202110135YA (en) 2021-10-28
WO2020198641A3 (en) 2020-11-05
TW202102529A (zh) 2021-01-16

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