MX2019007594A - Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina. - Google Patents

Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina.

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Publication number
MX2019007594A
MX2019007594A MX2019007594A MX2019007594A MX2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A MX 2019007594 A MX2019007594 A MX 2019007594A
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MX
Mexico
Prior art keywords
compositions
methods
antitrypsin deficiency
treating alpha
antitrypsin
Prior art date
Application number
MX2019007594A
Other languages
English (en)
Inventor
Strapps Walter
Odate Shobu
Michael Lescarbeau Reynald
Original Assignee
Intellia 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.)
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Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of MX2019007594A publication Critical patent/MX2019007594A/es

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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/5146Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
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    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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Abstract

Se proporcionan composiciones y métodos para introducir rupturas de doble cadena dentro del gen de SERPINA 1. Se proporcionan composiciones y métodos para reducir y eliminar las formas mutantes de a1-antitripsina (AAT), tal como se observa en los sujetos que tienen deficiencia de a1-antitripsina (AATD).
MX2019007594A 2016-12-22 2017-12-21 Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina. MX2019007594A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662438219P 2016-12-22 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

Publications (1)

Publication Number Publication Date
MX2019007594A true MX2019007594A (es) 2019-12-16

Family

ID=61054487

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019007594A MX2019007594A (es) 2016-12-22 2017-12-21 Composiciones y metodos para tratar la deficiencia de alfa-1 antitripsina.

Country Status (18)

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US (2) US11549107B2 (es)
EP (1) EP3559232A1 (es)
JP (2) JP7383478B2 (es)
KR (1) KR102551664B1 (es)
CN (2) CN110382697B (es)
AU (2) AU2017379073B2 (es)
BR (1) BR112019012825A2 (es)
CA (1) CA3047415A1 (es)
CO (1) CO2019007776A2 (es)
EA (1) EA201991455A1 (es)
IL (1) IL267448A (es)
MX (1) MX2019007594A (es)
NZ (1) NZ754739A (es)
PH (1) PH12019501457A1 (es)
SG (1) SG10202106412RA (es)
TW (1) TW201833331A (es)
WO (1) WO2018119182A1 (es)
ZA (1) ZA202007632B (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
CA3077413A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. Formulations
WO2020006708A1 (en) * 2018-07-04 2020-01-09 Yale University Compositions and methods for enhancement of homology-directed repair mediated precise gene editing by programming dna repair with a single rna-guided endonuclease
WO2020082047A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
JP7472121B2 (ja) * 2018-10-18 2024-04-22 インテリア セラピューティクス,インコーポレーテッド アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法
US20220160847A1 (en) * 2020-11-20 2022-05-26 Mark Egly Method of preventing and/or treating a plurality of diseases
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination

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DK1695979T3 (da) 1991-12-24 2011-10-10 Isis Pharmaceuticals Inc Gappede modificerede oligonukleotider
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
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KR20160102056A (ko) * 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 멀티플렉스 가이드 rna
JP7068821B2 (ja) * 2014-12-03 2022-05-17 アジレント・テクノロジーズ・インク 化学修飾を有するガイドrna
CN107787367B (zh) * 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna
AU2016338785B2 (en) * 2015-10-12 2022-07-14 E. I. Du Pont De Nemours And Company Protected DNA templates for gene modification and increased homologous recombination in cells and methods of use
MX2018005332A (es) * 2015-11-06 2018-11-09 Crispr Therapeutics Ag Materiales y metodos para tratamiento de la enfermedad de almacenamiento de glucogeno tipo 1a.
JP6932698B2 (ja) * 2015-12-01 2021-09-08 クリスパー・セラピューティクス・アクチェンゲゼルシャフトCRISPR Therapeutics AG アルファ1アンチトリプシン欠乏症の治療のための材料および方法
EP3433364A1 (en) * 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020082047A1 (en) * 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey

Also Published As

Publication number Publication date
NZ754739A (en) 2024-01-26
ZA202007632B (en) 2023-03-29
IL267448A (en) 2019-08-29
SG10202106412RA (en) 2021-07-29
JP2020501582A (ja) 2020-01-23
PH12019501457A1 (en) 2020-03-09
US11549107B2 (en) 2023-01-10
JP7383478B2 (ja) 2023-11-20
WO2018119182A1 (en) 2018-06-28
TW201833331A (zh) 2018-09-16
KR102551664B1 (ko) 2023-07-05
EA201991455A1 (ru) 2020-01-15
EP3559232A1 (en) 2019-10-30
CN117737062A (zh) 2024-03-22
CA3047415A1 (en) 2018-06-28
AU2024201464A1 (en) 2024-03-28
US20230212575A1 (en) 2023-07-06
KR20190100943A (ko) 2019-08-29
BR112019012825A2 (pt) 2019-11-26
AU2017379073B2 (en) 2023-12-14
US20190316129A1 (en) 2019-10-17
CO2019007776A2 (es) 2019-10-09
JP2023075164A (ja) 2023-05-30
AU2017379073A1 (en) 2019-07-11
CN110382697A (zh) 2019-10-25
CN110382697B (zh) 2023-12-29

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