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MX2018011114A - Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías. - Google Patents

Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.

Info

Publication number
MX2018011114A
MX2018011114A MX2018011114A MX2018011114A MX2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A MX 2018011114 A MX2018011114 A MX 2018011114A
Authority
MX
Mexico
Prior art keywords
crispr
cas
compositions
related methods
treating beta
Prior art date
Application number
MX2018011114A
Other languages
English (en)
Inventor
Leah Gori Jennifer
A Barrera Luis
Original Assignee
Editas Medicine 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 Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of MX2018011114A publication Critical patent/MX2018011114A/es

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Abstract

Se describen composiciones y métodos relacionados con CRISPR/CAS para el tratamiento de ß hemoglobinopatías.
MX2018011114A 2016-03-14 2017-03-14 Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías. MX2018011114A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662308190P 2016-03-14 2016-03-14
US201762456615P 2017-02-08 2017-02-08
PCT/US2017/022377 WO2017160890A1 (en) 2016-03-14 2017-03-14 Crispr/cas-related methods and compositions for treating beta hemoglobinopathies

Publications (1)

Publication Number Publication Date
MX2018011114A true MX2018011114A (es) 2019-02-20

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ID=58413206

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MX2018011114A MX2018011114A (es) 2016-03-14 2017-03-14 Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.
MX2024012899A MX2024012899A (es) 2016-03-14 2018-09-13 Metodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatias

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US (2) US20200255857A1 (es)
EP (1) EP3430142A1 (es)
JP (2) JP2019508051A (es)
KR (2) KR102532663B1 (es)
CN (4) CN117821458A (es)
AU (2) AU2017235333B2 (es)
CA (1) CA3017956A1 (es)
IL (2) IL313038A (es)
MX (2) MX2018011114A (es)
SG (1) SG11201807859WA (es)
WO (1) WO2017160890A1 (es)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
SG11201706767RA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US11390884B2 (en) 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
AU2016342380B2 (en) 2015-10-23 2022-04-07 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3371306B8 (en) 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
IL308426B1 (en) 2016-08-03 2025-11-01 Harvard College Adenosine nucleobase editors and uses thereof
JP7201153B2 (ja) 2016-08-09 2023-01-10 プレジデント アンド フェローズ オブ ハーバード カレッジ プログラム可能cas9-リコンビナーゼ融合タンパク質およびその使用
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018083071A1 (en) * 2016-11-02 2018-05-11 Universität Basel Immunologically discernible cell surface variants for use in cell therapy
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018200597A1 (en) * 2017-04-24 2018-11-01 Seattle Children's Hospital (dba Seattle Children's Research Institute) Homology directed repair compositions for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US20200140896A1 (en) * 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
KR20200121782A (ko) * 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3701029A1 (en) * 2017-10-26 2020-09-02 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN111556893A (zh) * 2017-11-06 2020-08-18 爱迪塔斯医药股份有限公司 免疫疗法t细胞中cblb的crispr-cas9编辑的方法、组合物和组分
MA51107A (fr) 2017-12-05 2020-10-14 Vertex Pharma Cellules souches et progénitrices hématopoïétiques humaines cd34+ modifiées par crispr-cas9 et utilisations associées
WO2019118516A1 (en) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019150203A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2019173654A2 (en) * 2018-03-07 2019-09-12 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP7706888B2 (ja) * 2018-03-14 2025-07-14 エディタス・メディシン、インコーポレイテッド 異常ヘモグロビン症の治療のためのシステム及び方法
CA3093701A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN112543650A (zh) * 2018-04-24 2021-03-23 利甘达尔股份有限公司 基因组编辑的方法和组合物
JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク. プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020053224A1 (en) * 2018-09-11 2020-03-19 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020113112A1 (en) * 2018-11-29 2020-06-04 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP7428712B2 (ja) * 2018-12-05 2024-02-06 フレッド ハッチンソン キャンサー センター 低/最小操作による遺伝子改変細胞の製造
CN111321171A (zh) * 2018-12-14 2020-06-23 江苏集萃药康生物科技有限公司 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN114096666B (zh) 2019-02-13 2025-03-18 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN112011576A (zh) * 2019-05-31 2020-12-01 华东师范大学 Crispr基因编辑技术在治疗地中海贫血中的应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN112979823B (zh) * 2019-12-18 2022-04-08 华东师范大学 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白
EP4146804A1 (en) 2020-05-08 2023-03-15 The Broad Institute Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
CN111876416B (zh) * 2020-07-01 2021-09-03 广州瑞风生物科技有限公司 激活γ-珠蛋白基因表达的方法和组合物
AU2022381552A1 (en) * 2021-11-02 2024-05-09 Incisive Genetics, Inc. Compositions and methods for preventing, ameliorating, or treating sickle cell disease
CN114848851A (zh) * 2022-04-29 2022-08-05 广州医科大学附属第三医院(广州重症孕产妇救治中心、广州柔济医院) 治疗β-地中海贫血的药物
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2025038164A1 (en) * 2023-08-15 2025-02-20 University Of Massachusetts Nick resection in cancer
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
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009013559A1 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
KR20180009383A (ko) * 2012-02-24 2018-01-26 프레드 헛친슨 켄서 리서치 센터 이상혈색소증 치료를 위한 조성물 및 방법
US9963715B2 (en) * 2012-08-29 2018-05-08 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
SG11201504038XA (en) * 2012-11-27 2015-06-29 Childrens Medical Center Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction
JP2016521975A (ja) * 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝的状態の処置のための方法および組成物
KR102307280B1 (ko) * 2013-06-05 2021-10-01 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
EP3066201B1 (en) * 2013-11-07 2018-03-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
EP3981876A1 (en) * 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
SG11201706767RA (en) * 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US11390884B2 (en) 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells

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