JP7383478B2 - アルファ1アンチトリプシン欠乏症を治療するための組成物および方法 - Google Patents

アルファ1アンチトリプシン欠乏症を治療するための組成物および方法 Download PDF

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JP7383478B2
JP7383478B2 JP2019533577A JP2019533577A JP7383478B2 JP 7383478 B2 JP7383478 B2 JP 7383478B2 JP 2019533577 A JP2019533577 A JP 2019533577A JP 2019533577 A JP2019533577 A JP 2019533577A JP 7383478 B2 JP7383478 B2 JP 7383478B2
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ストラップス,ウォルター
マイケル レスカーボ,レイナルド
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インテリア セラピューティクス,インコーポレイテッド
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JP2019533577A 2016-12-22 2017-12-21 アルファ1アンチトリプシン欠乏症を治療するための組成物および方法 Active JP7383478B2 (ja)

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US201662438219P 2016-12-22 2016-12-22
US62/438,219 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

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Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI851534B (zh) * 2016-12-22 2024-08-11 美商英特利亞醫療公司 用於治療α-1抗胰蛋白酶缺乏症之組合物及方法
US20230140670A1 (en) * 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
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
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
AU2019360270B2 (en) 2018-10-18 2025-08-07 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
WO2020082047A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
US20200270617A1 (en) * 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
US20230416725A1 (en) * 2020-09-15 2023-12-28 Rutgers, The State University Of New Jersey Systems for gene editing and methods of use thereof
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20220160847A1 (en) * 2020-11-20 2022-05-26 Mark Egly Method of preventing and/or treating a plurality of diseases
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (26)

* 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
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する2′修飾オリゴヌクレオチド
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのpna プローブ
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
CN107879960B (zh) * 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
CN112301024A (zh) * 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
RU2716420C2 (ru) * 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
US9458457B2 (en) 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
CN120574876A (zh) * 2013-08-22 2025-09-02 纳幕尔杜邦公司 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法
CN106459995B (zh) * 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2935032C (en) 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
CA2969619A1 (en) * 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
KR102888521B1 (ko) * 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
EP4144844B1 (en) * 2015-10-12 2025-09-10 DuPont US Holding, LLC Protected dna templates for gene modification and increased homologous recombination in cells and methods of use
BR112018008971A2 (pt) * 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
AU2016365720B2 (en) * 2015-12-07 2020-11-26 Arc Bio, Llc Methods and compositions for the making and using of guide nucleic acids
US11512311B2 (en) * 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
LT3436077T (lt) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
KR20180134412A (ko) 2016-04-22 2018-12-18 인텔리아 테라퓨틱스, 인크. 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법
SG10202106058WA (en) * 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
TWI851534B (zh) * 2016-12-22 2024-08-11 美商英特利亞醫療公司 用於治療α-1抗胰蛋白酶缺乏症之組合物及方法
WO2020082047A1 (en) * 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Molecular Therapy,2015年,Vol.23, No.3,PP.570-577
Nature,2011年,Vol.478,PP.391-396

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