CN112020558A - 治疗血红蛋白病的系统和方法 - Google Patents

治疗血红蛋白病的系统和方法 Download PDF

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CN112020558A
CN112020558A CN201980025285.5A CN201980025285A CN112020558A CN 112020558 A CN112020558 A CN 112020558A CN 201980025285 A CN201980025285 A CN 201980025285A CN 112020558 A CN112020558 A CN 112020558A
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certain embodiments
grna
rna
cells
rnp
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J·L·戈里
E·奥佩潘·德·拉莫特-德赫兹
J·希斯
J·A·左瑞斯
K·常
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Editas Medicine Inc
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CN201980025285.5A 2018-03-14 2019-03-14 治疗血红蛋白病的系统和方法 Pending CN112020558A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862643168P 2018-03-14 2018-03-14
US62/643,168 2018-03-14
US201862767488P 2018-11-14 2018-11-14
US62/767,488 2018-11-14
US201862773073P 2018-11-29 2018-11-29
US62/773,073 2018-11-29
PCT/US2019/022374 WO2019178426A1 (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies

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US (2) US12031132B2 (https=)
EP (1) EP3765614A1 (https=)
JP (2) JP7706888B2 (https=)
KR (2) KR20260006713A (https=)
CN (1) CN112020558A (https=)
AU (2) AU2019236209B2 (https=)
BR (1) BR112020018364A2 (https=)
CA (1) CA3093702A1 (https=)
IL (2) IL277234B2 (https=)
MX (1) MX2020009487A (https=)
SG (1) SG11202008956XA (https=)
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108779462A (zh) * 2015-12-28 2018-11-09 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法
WO2023046086A1 (zh) * 2021-09-24 2023-03-30 华东师范大学 一种碱基编辑系统及其应用
WO2025067403A1 (zh) * 2023-09-28 2025-04-03 上海天泽云泰生物医药有限公司 用于治疗血红蛋白疾病的组合物和方法

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US12139720B2 (en) * 2017-06-02 2024-11-12 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant lentiviral vector for stem cell- based gene therapy of sickle cell disorder
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
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
IL325857A (en) * 2018-08-08 2026-03-01 Integrated Dna Tech Inc Novel mutations that enhance DNA cleavage activity of ACIDAMINOCOCCUS SP. CPF1
US20220047637A1 (en) * 2018-11-29 2022-02-17 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3164055A1 (en) * 2019-12-08 2021-06-17 Editas Medicine, Inc. Modified cells and methods for the treatment of hemoglobinopathies
EP4069829A1 (en) * 2019-12-08 2022-10-12 Editas Medicine, Inc. Modified cells and methods for the treatment of hemoglobinopathies
EP4076479A4 (en) * 2019-12-18 2024-05-22 Editas Medicine, Inc. GENETICALLY MODIFIED CELLS FOR THERAPY
CA3170326A1 (en) * 2020-02-13 2021-08-19 Beam Therapeutics Inc. Compositions and methods for engraftment of base edited cells
WO2021228944A1 (en) * 2020-05-13 2021-11-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
AU2022220203A1 (en) * 2021-02-11 2023-08-24 Koninklijke Nederlandse Akademie Van Wetenschappen Curing disease by transcription regulatory gene editing
EP4047095A1 (en) * 2021-02-22 2022-08-24 Eberhard Karls Universität Tübingen Medizinische Fakultät Method and composition for a targeted gene knockout
JP2024528967A (ja) * 2021-08-02 2024-08-01 エディタス・メディシン、インコーポレイテッド ヘモグロビン異常症の治療のためのシステム及び方法
KR102853682B1 (ko) 2022-03-07 2025-09-03 광주과학기술원 신속 DNA 진단을 위한 Cpf1 변이 단백질
CN121241136A (zh) * 2023-04-07 2025-12-30 基因泰克公司 经修饰的指导rna
AU2024272060A1 (en) * 2023-05-17 2026-01-08 Renagade Therapeutics Management Inc. Gene editing systems and compositions for treatment of hemoglobinopathies and methods of using the same
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 (118)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US6203986B1 (en) 1998-10-22 2001-03-20 Robert H. Singer Visualization of RNA in living cells
ATE283013T1 (de) 1999-10-21 2004-12-15 Alcon Inc Medikamentenzuführvorrichtung
WO2002089767A1 (en) 2001-05-03 2002-11-14 Massachusetts Eye And Ear Infirmary Implantable drug delivery device and use thereof
WO2003072788A1 (en) 2002-02-21 2003-09-04 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
US20070020627A1 (en) 2002-06-11 2007-01-25 The Scripps Research Institute Artificial transcription factors
WO2007014181A2 (en) 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007106690A2 (en) 2006-03-15 2007-09-20 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
AU2008223544B2 (en) 2007-03-02 2014-06-05 Dupont Nutrition Biosciences Aps Cultures with improved phage resistance
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010037001A2 (en) 2008-09-26 2010-04-01 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
SG185481A1 (en) 2010-05-10 2012-12-28 Univ California Endoribonuclease compositions and methods of use thereof
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
GB201013153D0 (en) 2010-08-04 2010-09-22 Touchlight Genetics Ltd Primer for production of closed linear DNA
US9499592B2 (en) 2011-01-26 2016-11-22 President And Fellows Of Harvard College Transcription activator-like effectors
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
CA2841710C (en) 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
RU2650811C2 (ru) 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
CN108285491B (zh) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
KR102530118B1 (ko) 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
HK1207111A1 (en) 2012-08-03 2016-01-22 加利福尼亚大学董事会 Methods and compositions for controlling gene expression by rna processing
SI2890780T1 (sl) 2012-08-29 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje genetskega stanja
EP3789405A1 (en) 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
EP4357457B1 (en) 2012-10-23 2024-10-16 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
SG11201504038XA (en) 2012-11-27 2015-06-29 Childrens Medical Center Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2894684A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
WO2014093718A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CA2895155C (en) 2012-12-17 2021-07-06 President And Fellows Of Harvard College Rna-guided human genome engineering
AU2014214719B2 (en) 2013-02-07 2020-02-13 The General Hospital Corporation Tale transcriptional activators
MX374090B (es) 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
AU2014227653B2 (en) 2013-03-15 2017-04-20 The General Hospital Corporation Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
EP4596565A3 (en) 2013-06-04 2025-11-05 President And Fellows Of Harvard College Rna-guided transcriptional regulation
EP3004370B1 (en) 2013-06-05 2024-08-21 Duke University Rna-guided gene editing and gene regulation
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
JP7120717B2 (ja) 2013-07-09 2022-08-17 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 多重ｒｎａ誘導型ゲノム編集
WO2015006294A2 (en) 2013-07-10 2015-01-15 President And Fellows Of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
HUE044540T2 (hu) 2013-11-13 2019-10-28 Childrens Medical Center Nukleáz közvetítette génexpresszió-szabályozás
EP3760719A1 (en) 2013-11-18 2021-01-06 CRISPR Therapeutics AG Crispr-cas system materials and methods
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
CA2935032C (en) 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
WO2015148860A1 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
DK3155101T3 (da) 2014-06-16 2020-05-04 Univ Johns Hopkins Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren
ES3047792T3 (en) 2014-07-14 2025-12-04 Univ California Crispr/cas transcriptional modulation
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CA2963820A1 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US10900034B2 (en) * 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
AU2016225178B2 (en) 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3851530A1 (en) 2015-03-26 2021-07-21 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
US20160324987A1 (en) 2015-04-15 2016-11-10 Cedars-Sinai Medical Center Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
WO2016186772A2 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
JP6424768B2 (ja) * 2015-08-04 2018-11-21 アイシン精機株式会社 操作入力検知装置
EP3341727B1 (en) 2015-08-25 2022-08-10 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
WO2017040511A1 (en) 2015-08-31 2017-03-09 Agilent Technologies, Inc. Compounds and methods for crispr/cas-based genome editing by homologous recombination
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
WO2017106657A1 (en) * 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
KR102860636B1 (ko) 2015-12-28 2025-09-17 노파르티스 아게 혈색소병증의 치료를 위한 조성물 및 방법
MX2018011114A (es) * 2016-03-14 2019-02-20 Editas Medicine Inc Métodos y composiciones relacionadas con crispr/cas para el tratamiento de beta hemoglobinopatías.
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
CN106244591A (zh) * 2016-08-23 2016-12-21 苏州吉玛基因股份有限公司 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2018017754A1 (en) * 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
AU2017388753A1 (en) 2016-12-30 2019-07-11 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
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
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
CN120290559A (zh) * 2017-10-02 2025-07-11 基因编辑有限公司 修饰的cpf1引导rna
WO2019118516A1 (en) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
US12275951B2 (en) * 2018-02-06 2025-04-15 Ohio State Innovation Foundation Engineered guide RNA and uses thereof
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
AU2019234922A1 (en) 2018-03-14 2020-10-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP4069829A1 (en) 2019-12-08 2022-10-12 Editas Medicine, Inc. Modified cells and methods for the treatment of hemoglobinopathies

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108779462A (zh) * 2015-12-28 2018-11-09 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法
WO2023046086A1 (zh) * 2021-09-24 2023-03-30 华东师范大学 一种碱基编辑系统及其应用
WO2025067403A1 (zh) * 2023-09-28 2025-04-03 上海天泽云泰生物医药有限公司 用于治疗血红蛋白疾病的组合物和方法

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