CN108779462A - 用于治疗血红蛋白病的组合物和方法 - Google Patents

用于治疗血红蛋白病的组合物和方法 Download PDF

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Publication number
CN108779462A
CN108779462A CN201680082815.6A CN201680082815A CN108779462A CN 108779462 A CN108779462 A CN 108779462A CN 201680082815 A CN201680082815 A CN 201680082815A CN 108779462 A CN108779462 A CN 108779462A
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seq
grna molecules
cell
tracr
molecules
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Chinese (zh)
Inventor
A·E·博伊塔诺
M·库克
L·B·科立克斯泰恩
R·莱斯卡博
C·S·米克宁
K·穆鲁巴
S·R·普利斯
J·斯尼德
S·C·史蒂文森
M·斯图尔特
Y·杨
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Internal Treatment Co
Novartis AG
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Internal Treatment Co
Novartis AG
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CN201680082815.6A 2015-12-28 2016-12-26 用于治疗血红蛋白病的组合物和方法 Pending CN108779462A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562271968P 2015-12-28 2015-12-28
US62/271,968 2015-12-28
US201662347484P 2016-06-08 2016-06-08
US62/347,484 2016-06-08
PCT/IB2016/058007 WO2017115268A1 (en) 2015-12-28 2016-12-26 Compositions and methods for the treatment of hemoglobinopathies

Publications (1)

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CN108779462A true CN108779462A (zh) 2018-11-09

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US (2) US20190010495A1 (enExample)
EP (2) EP3397767A1 (enExample)
JP (3) JP2019500043A (enExample)
KR (2) KR102860636B1 (enExample)
CN (1) CN108779462A (enExample)
AU (4) AU2016381313B2 (enExample)
CA (1) CA3009727A1 (enExample)
EA (1) EA201891532A1 (enExample)
HK (1) HK1258205A1 (enExample)
IL (2) IL308706A (enExample)
MX (2) MX2018007987A (enExample)
MY (1) MY192848A (enExample)
NZ (4) NZ744649A (enExample)
PH (1) PH12018501378A1 (enExample)
SG (1) SG11201805217XA (enExample)
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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020221291A1 (zh) * 2019-04-30 2020-11-05 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
CN111939271A (zh) * 2019-04-30 2020-11-17 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
WO2021037232A1 (zh) * 2019-08-28 2021-03-04 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
CN114149990A (zh) * 2020-09-08 2022-03-08 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
CN116445402A (zh) * 2023-05-09 2023-07-18 中国科学院生态环境研究中心 红系祖细胞的扩增培养基、扩增培养方法及应用

Families Citing this family (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016135558A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
WO2016182917A1 (en) * 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
US10808020B2 (en) * 2015-05-12 2020-10-20 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
EP3365356B1 (en) 2015-10-23 2023-06-28 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
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20190203230A1 (en) 2016-09-28 2019-07-04 Novartis Ag Porous membrane-based macromolecule delivery system
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
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
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
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
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US20210380937A1 (en) 2017-04-04 2021-12-09 The Board Of Trustees Of The Leland Stanford Junior University Preparation , expansion, and uses of adult pluripotent stem cells
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
US11788087B2 (en) * 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
WO2019003193A1 (en) 2017-06-30 2019-01-03 Novartis Ag METHODS FOR TREATING DISEASES USING 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
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
JP2020537541A (ja) * 2017-09-29 2020-12-24 インテリア セラピューティクス，インコーポレーテッド 脂質ナノ粒子を用いるインビトロでのｍｒｎａの送達方法
CN109706148A (zh) * 2017-09-30 2019-05-03 广东赤萌医疗科技有限公司 一种用于敲除BCL11A基因或者BCL11A基因增强子的gRNA、gRNA组合物以及电转方法
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
MA50849A (fr) * 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
WO2019098759A2 (ko) * 2017-11-16 2019-05-23 재단법인 목암생명과학연구소 형질전환된 인간세포 및 이의 용도
WO2019106522A1 (en) * 2017-11-28 2019-06-06 Novartis Ag Pooled crispr/cas9 screening in primary cells using guide swap technology
US12161674B2 (en) * 2017-12-05 2024-12-10 Vertex Pharmaceuticals Incorporated CRISPR-CAS9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof
BR112020009889A2 (pt) 2017-12-14 2020-11-03 Flodesign Sonics, Inc. acionador e controlador de transdutor acústico
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN111902411A (zh) * 2018-01-03 2020-11-06 美真达治疗公司 用于扩增造血干细胞和祖细胞以及治疗遗传性代谢紊乱的组合物和方法
WO2019147302A1 (en) * 2018-01-26 2019-08-01 Bauer Daniel E Targeting bcl11a distal regulatory elements with a cas9-cas9 fusion for fetal hemoglobin reinduction
US11566236B2 (en) 2018-02-05 2023-01-31 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749768A1 (en) * 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for 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
WO2019213273A1 (en) * 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
EP3787600A4 (en) 2018-05-02 2022-02-16 The Children's Medical Center Corporation ENHANCED BCL11A MICRORNAS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
CN112105732A (zh) * 2018-05-10 2020-12-18 先正达参股股份有限公司 用于多核苷酸的靶向编辑的方法和组合物
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CA3113255A1 (en) 2018-09-21 2020-03-26 APstem Therapeutics, Inc. Human pluripotent adult stem cells
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US20220047637A1 (en) * 2018-11-29 2022-02-17 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220380756A1 (en) * 2019-06-28 2022-12-01 Asc Therapeutics Inc. Methods and compositions for treating thalassemia or sickle cell disease
US20220380757A1 (en) * 2019-09-30 2022-12-01 The Children's Medical Center Corporation Bcl11a guide and base editor delivery
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
US12079386B2 (en) 2020-09-14 2024-09-03 Zebra Technologies Corporation Devices having protective screens selectively positionable to switch devices between user interface modes and methods of operating the same
US20240245725A1 (en) * 2021-05-13 2024-07-25 The Children's Medical Center Corporation Methods for stratifying subjects for fetal hemoglobin reinduction
CN113584167B (zh) * 2021-07-07 2023-10-03 武汉大学中南医院 一种用于检测FLT3-F691L突变的crRNA、等温扩增引物和试剂盒
EP4273242A1 (en) * 2022-05-03 2023-11-08 ETH Zurich Crispr-based modification of human hbd gene
WO2023214512A1 (ja) 2022-05-06 2023-11-09 学校法人常翔学園 人工ｒｎａ分子
EP4561639A2 (en) * 2022-07-25 2025-06-04 Celyntra Therapeutics SA Compositions and methods for genome editing
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

Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102203096A (zh) * 2008-10-30 2011-09-28 Irm责任有限公司 扩增造血干细胞的化合物
CN104144931A (zh) * 2012-01-27 2014-11-12 蒙特利尔大学 嘧啶并[4,5-b]吲哚衍生物及其在造血干细胞的扩增中的应用
CN104284669A (zh) * 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
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
EP3122880A2 (en) * 2014-03-26 2017-02-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
CN106794260A (zh) * 2014-04-25 2017-05-31 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
CN109706148A (zh) * 2017-09-30 2019-05-03 广东赤萌医疗科技有限公司 一种用于敲除BCL11A基因或者BCL11A基因增强子的gRNA、gRNA组合物以及电转方法
CN111278976A (zh) * 2017-10-27 2020-06-12 博雅辑因（北京）生物科技有限公司 一种提高胎儿血红蛋白表达的方法
CN112020558A (zh) * 2018-03-14 2020-12-01 爱迪塔斯医药公司 治疗血红蛋白病的系统和方法

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005060697A2 (en) 2003-12-19 2005-07-07 Chiron Corporation Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
SG181904A1 (en) 2009-12-23 2012-07-30 Novartis Ag Lipids, lipid compositions, and methods of using them
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
EP3542789A3 (en) 2010-08-31 2020-01-01 GlaxoSmithKline Biologicals SA Lipids suitable for liposomal delivery of protein-coding rna
EP4115875A1 (en) 2011-07-06 2023-01-11 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
BR112014009346B1 (pt) 2011-10-17 2020-09-15 Massachusetts Institute Of Technology Entrega intracelular
US9394547B2 (en) 2012-01-03 2016-07-19 City University Of Hong Kong Method and apparatus for delivery of molecules to cells
WO2013103467A1 (en) 2012-01-06 2013-07-11 Alcon Research, Ltd. Interfering rna delivery system and uses thereof
PT3502240T (pt) * 2012-11-27 2021-08-11 Childrens Medical Ct Corp Elementos reguladores distais de bcl11a como alvo para a reindução de hemoglobina fetal
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014136086A1 (en) 2013-03-08 2014-09-12 Novartis Ag Lipids and lipid compositions for the delivery of active agents
EP2970948B1 (en) 2013-03-15 2018-12-26 GlaxoSmithKline Biologicals SA Rna purification methods
KR102210319B1 (ko) * 2013-03-15 2021-02-01 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
CN105492611A (zh) * 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
KR102243597B1 (ko) 2013-08-16 2021-04-26 메사추세츠 인스티튜트 오브 테크놀로지 세포로의 선택적 물질 전달
PT3492593T (pt) * 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
WO2015079057A2 (en) * 2013-11-28 2015-06-04 Haplogen Genomics Gmbh Somatic haploid human cell line
US10426737B2 (en) 2013-12-19 2019-10-01 Novartis Ag Lipids and lipid compositions for the delivery of active agents
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
JP6695795B2 (ja) * 2014-03-31 2020-05-20 株式会社サイエンス・ラスター 造血細胞の増殖ペプチドおよびその用途
WO2015158860A1 (de) * 2014-04-16 2015-10-22 Sika Technology Ag Schnell härtende migrationsfreie zusammensetzung auf basis von silangruppen-haltigen polymeren
CN104480144B (zh) * 2014-12-12 2017-04-12 武汉大学 用于艾滋病基因治疗的CRISPR/Cas9重组慢病毒载体及其慢病毒
WO2016182917A1 (en) * 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102203096A (zh) * 2008-10-30 2011-09-28 Irm责任有限公司 扩增造血干细胞的化合物
CN104144931A (zh) * 2012-01-27 2014-11-12 蒙特利尔大学 嘧啶并[4,5-b]吲哚衍生物及其在造血干细胞的扩增中的应用
CN104284669A (zh) * 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
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
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
EP3122880A2 (en) * 2014-03-26 2017-02-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
CN106794260A (zh) * 2014-04-25 2017-05-31 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
CN109706148A (zh) * 2017-09-30 2019-05-03 广东赤萌医疗科技有限公司 一种用于敲除BCL11A基因或者BCL11A基因增强子的gRNA、gRNA组合物以及电转方法
CN111278976A (zh) * 2017-10-27 2020-06-12 博雅辑因（北京）生物科技有限公司 一种提高胎儿血红蛋白表达的方法
CN112020558A (zh) * 2018-03-14 2020-12-01 爱迪塔斯医药公司 治疗血红蛋白病的系统和方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CANVER, MATTHEW C1等: ""BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis."", 《NATURE》 *
高飏: ""CRISPR/Cas9编辑CD34+造血干细胞BCL11A基因提升HbF表达"", 《中国博士学位论文全文数据库（电子期刊）医药卫生科技辑》 *

Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020221291A1 (zh) * 2019-04-30 2020-11-05 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
CN111939271A (zh) * 2019-04-30 2020-11-17 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
CN113785059A (zh) * 2019-04-30 2021-12-10 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
WO2021037232A1 (zh) * 2019-08-28 2021-03-04 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
CN114364799A (zh) * 2019-08-28 2022-04-15 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
CN114364799B (zh) * 2019-08-28 2024-07-19 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
CN114149990A (zh) * 2020-09-08 2022-03-08 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
WO2022052909A1 (zh) * 2020-09-08 2022-03-17 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
CN116445402A (zh) * 2023-05-09 2023-07-18 中国科学院生态环境研究中心 红系祖细胞的扩增培养基、扩增培养方法及应用

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