IN2014DN07853A - - Google Patents

Info

Publication number
IN2014DN07853A
IN2014DN07853A IN7853DEN2014A IN2014DN07853A IN 2014DN07853 A IN2014DN07853 A IN 2014DN07853A IN 7853DEN2014 A IN7853DEN2014 A IN 7853DEN2014A IN 2014DN07853 A IN2014DN07853 A IN 2014DN07853A
Authority
IN
India
Prior art keywords
endonuclease
globin gene
disrupt
bell
fusion protein
Prior art date
Application number
Other languages
English (en)
Inventor
Michael A Bender
Mark T Groudine
Barry L Stoddard
Ryo Takeuchi
Original Assignee
Hutchinson Fred Cancer Res
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 Hutchinson Fred Cancer Res filed Critical Hutchinson Fred Cancer Res
Publication of IN2014DN07853A publication Critical patent/IN2014DN07853A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IN7853DEN2014 2012-02-24 2013-02-22 IN2014DN07853A (cs)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261603231P 2012-02-24 2012-02-24
PCT/US2013/027459 WO2013126794A1 (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
IN2014DN07853A true IN2014DN07853A (cs) 2015-04-24

Family

ID=49006265

Family Applications (1)

Application Number Title Priority Date Filing Date
IN7853DEN2014 IN2014DN07853A (cs) 2012-02-24 2013-02-22

Country Status (18)

Country Link
US (2) US20150166969A1 (cs)
EP (2) EP2836226B1 (cs)
JP (2) JP6170080B2 (cs)
KR (2) KR20180009383A (cs)
CN (1) CN104284669A (cs)
AU (2) AU2013222170B2 (cs)
BR (1) BR112014020625A2 (cs)
CA (1) CA2865129A1 (cs)
DK (1) DK2836226T3 (cs)
ES (1) ES2641840T3 (cs)
HK (1) HK1249861A1 (cs)
IL (2) IL234225B (cs)
IN (1) IN2014DN07853A (cs)
NZ (1) NZ630900A (cs)
RU (2) RU2650811C2 (cs)
SG (2) SG10201606959PA (cs)
WO (1) WO2013126794A1 (cs)
ZA (1) ZA201406187B (cs)

Families Citing this family (184)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
SG10201701800YA (en) 2012-05-25 2017-04-27 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
KR102218562B1 (ko) 2012-08-29 2021-02-19 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
AU2013335451C1 (en) * 2012-10-23 2024-07-04 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
CA2892860C (en) 2012-11-27 2023-01-03 Children's Medical Center Corporation Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction
EP3135765A1 (en) 2012-12-06 2017-03-01 Sigma-Aldrich Co. LLC Crispr-based genome modification and regulation
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
EP2971167B1 (en) 2013-03-14 2019-07-31 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
EP2971041B1 (en) 2013-03-15 2018-11-28 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
CN105518146B (zh) 2013-04-04 2022-07-15 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
SG10201913068PA (en) 2013-06-04 2020-02-27 Harvard College Rna-guided transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
EP3011031B1 (en) 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
EP3011032B1 (en) 2013-06-17 2019-10-16 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
CN114015726B (zh) 2013-06-17 2025-06-24 布罗德研究所有限公司 用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
EP3019595A4 (en) * 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
WO2015006294A2 (en) 2013-07-10 2015-01-15 President And Fellows Of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US11306328B2 (en) 2013-07-26 2022-04-19 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins 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
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
ES2721936T3 (es) 2013-11-13 2019-08-06 Childrens Medical Center Regulación de la expresión genética mediada por las nucleasas
EP3071695A2 (en) * 2013-11-18 2016-09-28 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
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
EP3089989B1 (en) 2013-12-31 2020-06-24 The Regents of The University of California Cas9 crystals and methods of use thereof
SG11201605550QA (en) * 2014-01-08 2016-08-30 Harvard College Rna-guided gene drives
WO2015122967A1 (en) 2014-02-13 2015-08-20 Clontech Laboratories, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
WO2015148716A1 (en) * 2014-03-26 2015-10-01 The Brigham And Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
EP3598984B1 (en) 2014-04-25 2024-04-10 The Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US11071289B2 (en) * 2014-08-14 2021-07-27 Biocytogen Boston Corp DNA knock-in system
EA201790532A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов против вирусного вектора для переноса
HUE055583T2 (hu) * 2014-09-16 2021-12-28 Sangamo Therapeutics Inc Eljárások és készítmények nukleáz által közvetített genommódosításhoz és -javításhoz hematopoetikus õssejtekben
CN107205353B (zh) 2014-09-26 2021-03-19 谱赛科美国股份有限公司 用于甜菊的单核苷酸多态性(snp)标志物
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
AU2015355546B2 (en) 2014-12-03 2021-10-14 Agilent Technologies, Inc. Guide RNA with chemical modifications
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3237615B2 (en) 2014-12-24 2023-07-26 The Broad Institute, Inc. Crispr having or associated with destabilization domains
US20180002379A1 (en) * 2015-01-21 2018-01-04 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
WO2016123100A1 (en) 2015-01-26 2016-08-04 Fate Therapeutics, Inc. Methods and compositions for inducing hematopoietic cell differentiation
WO2016135559A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
US10738305B2 (en) 2015-02-23 2020-08-11 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
US10155938B2 (en) * 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
CN104805118A (zh) * 2015-04-22 2015-07-29 扬州大学 一种苏禽黄鸡胚胎干细胞特定基因进行靶向敲除方法
US11845928B2 (en) * 2015-05-04 2023-12-19 Tsinghua University Methods and kits for fragmenting DNA
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
ES2835861T5 (en) * 2015-05-08 2025-02-18 Childrens Medical Ct Corp Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
JP7030522B2 (ja) * 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
EP3294866A4 (en) * 2015-05-12 2018-12-05 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2016276702B2 (en) 2015-06-09 2022-07-28 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for improving transplantation
JP2018518181A (ja) 2015-06-17 2018-07-12 ザ ユーエービー リサーチ ファンデーション 血液細胞系列の細胞に機能的ポリペプチドを導入するためのＣＲＩＳＰＲ／Ｃａｓ９複合体
WO2016205703A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for genomic editing
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
CN114875012B (zh) 2015-08-28 2025-02-18 通用医疗公司 工程化的CRISPR-Cas9核酸酶
US9897378B2 (en) 2015-10-08 2018-02-20 Nyc Designed Inspirations Llc Cosmetic makeup sponge/blender container
AU2016342380B2 (en) 2015-10-23 2022-04-07 President And Fellows Of Harvard College Nucleobase editors and uses thereof
DK3368052T3 (da) * 2015-10-27 2022-03-14 Childrens Hospital Med Ct Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation
JP6882282B2 (ja) 2015-11-03 2021-06-02 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 三次元核酸含有マトリックスの立体撮像のための方法と装置
AU2016348342B2 (en) 2015-11-04 2023-07-06 Fate Therapeutics, Inc. Methods and compositions for inducing hematopoietic cell differentiation
US12043843B2 (en) 2015-11-04 2024-07-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP4249074A3 (en) 2015-11-04 2024-01-10 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells
EP3397767A1 (en) * 2015-12-28 2018-11-07 Novartis AG Compositions and methods for the treatment of hemoglobinopathies
DK3408382T3 (da) 2016-01-27 2022-06-20 Oncorus Inc Onkolytiske virale vektorer og anvendelser deraf
US11530253B2 (en) 2016-02-25 2022-12-20 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by CRISPR/CAS9 technology
JP2019508051A (ja) * 2016-03-14 2019-03-28 エディタス・メディシン、インコーポレイテッド β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP4424829A3 (en) * 2016-04-18 2024-11-06 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN109415761B (zh) 2016-04-25 2022-09-20 哈佛学院董事及会员团体 用于原位分子检测的杂交链反应方法
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
AU2017301609A1 (en) * 2016-07-25 2019-02-21 Bluebird Bio, Inc. BCL11A homing endonuclease variants, compositions, and methods of use
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
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
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
CN106244555A (zh) * 2016-08-23 2016-12-21 广州医科大学附属第三医院 一种提高基因打靶的效率的方法及β‑球蛋白基因位点的碱基原位修复方法
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
CN109923211A (zh) * 2016-09-08 2019-06-21 蓝鸟生物公司 Pd-1归巢核酸内切酶变体、组合物及使用方法
JP7588390B2 (ja) 2016-10-14 2024-11-22 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 核酸塩基エディターのａａｖ送達
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
CA3050514A1 (en) 2017-01-27 2018-08-02 Children's Hospital Medical Center Methods of enhancing engraftment activity of hematopoietic stem cells
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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP7191388B2 (ja) 2017-03-23 2022-12-19 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 核酸によってプログラム可能なｄｎａ結合蛋白質を含む核酸塩基編集因子
CN111031790A (zh) 2017-04-20 2020-04-17 E开创生物技术股份有限公司 产生基因修改的动物的方法
CA3059956A1 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
EP3615664A4 (en) 2017-04-24 2021-01-27 Seattle Children's Hospital (DBA Seattle Children's Research Institute) HOMOLOGY-DIRECTED REPAIR COMPOSITIONS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
EP3622070A2 (en) 2017-05-10 2020-03-18 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
CA3064828A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Bipartite base editor (bbe) architectures and type-ii-c-cas9 zinc finger editing
WO2018218135A1 (en) 2017-05-25 2018-11-29 The Children's Medical Center Corporation Bcl11a guide delivery
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
BR112020001559A2 (pt) 2017-07-26 2020-08-11 Oncorus, Inc. vetores virais oncolíticos e usos dos mesmos
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019070974A1 (en) 2017-10-04 2019-04-11 Bluebird Bio, Inc. PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
CA3078705A1 (en) 2017-10-13 2019-04-18 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
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
WO2019081982A1 (en) * 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
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
KR20200106159A (ko) 2017-12-05 2020-09-11 버텍스 파마슈티칼스 인코포레이티드 크리스퍼-cas9 변형된 cd34+ 인간 조혈 줄기 및 전구 세포 및 그의 용도
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
EP3749768A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749767A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
KR20200139679A (ko) 2018-02-26 2020-12-14 안톨알엑스, 인크. 면역관용성 리포솜 및 그의 사용 방법
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
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
WO2019204226A1 (en) 2018-04-16 2019-10-24 University Of Massachusetts Compositions and methods for improved gene editing
WO2019210216A2 (en) * 2018-04-27 2019-10-31 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Talen-based and crispr/cas-based gene editing for bruton's tyrosine kinase
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
EP3799603A4 (en) 2018-05-11 2022-03-02 Beam Therapeutics, Inc. METHODS OF PATHOGENIC AMINO ACID SUBSTITUTION USING PROGRAMMABLE BASE EDITOR SYSTEMS
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
BR112020025439A2 (pt) 2018-06-14 2021-03-16 Bluebird Bio, Inc. Receptores de antígenos quiméricos cd79a
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
WO2020072699A1 (en) * 2018-10-02 2020-04-09 Exosome Therapeutics, Inc. Exosome loaded therapeutics for treating sickle cell disease
US12312584B2 (en) 2018-10-02 2025-05-27 Exosome Therapeutics, Inc. cGMP exosome loaded therapeutics for treating sickle cell disease
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
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
WO2020123371A2 (en) * 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
EP3924481A4 (en) 2019-02-13 2023-01-25 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
WO2020191153A2 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN110042124A (zh) * 2019-04-25 2019-07-23 国家卫生健康委科学技术研究所 基因组碱基编辑增加人红细胞中胎儿血红蛋白水平的试剂盒及应用
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
US20220193142A1 (en) * 2019-04-30 2022-06-23 Edigene Inc. Method for predicting effectiveness of treatment of hemoglobinopathy
KR20220015425A (ko) 2019-05-28 2022-02-08 셀렉타 바이오사이언시즈, 인크. 약화된 항바이러스 전달 벡터 면역 반응을 위한 방법 및 조성물
EP3990650A4 (en) * 2019-06-28 2024-03-20 ASC Therapeutics Inc. METHODS AND COMPOSITIONS FOR TREATING THALASSEMIA AND SICKLE CELL ANEMIA
WO2021067788A1 (en) 2019-10-03 2021-04-08 Artisan Development Labs, Inc. Crispr systems with engineered dual guide nucleic acids
CN115175991A (zh) 2019-10-16 2022-10-11 博德研究所 工程化肌肉靶向组合物
CA3160172A1 (en) * 2019-11-15 2021-05-20 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at alpha-globin locus in human hematopoietic stem and progenitor cells
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
JP2023519346A (ja) 2020-03-27 2023-05-10 メンドゥス・ベスローテン・フェンノートシャップ 養子細胞療法の有効性を増強するための白血病由来の改変細胞のエクスビボ（ｅｘ ｖｉｖｏ）使用
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US20230279438A1 (en) * 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
JP2023531384A (ja) * 2020-06-04 2023-07-24 エメンドバイオ・インコーポレイテッド 新規なｏｍｎｉ－５９、６１、６７、７６、７９、８０、８１及び８２クリスパーヌクレアーゼ
AU2021302958A1 (en) 2020-06-30 2023-02-16 Mendus B.V. Use of leukemia-derived cells in ovarian cancer vaccines
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
CA3202889A1 (en) * 2020-12-21 2022-06-30 Jordan JARJOUR Compositions and methods for site-directed mutagenesis
WO2022182801A1 (en) 2021-02-25 2022-09-01 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
AU2022235341A1 (en) 2021-03-12 2023-09-21 Mendus B.V. Methods of vaccination and use of cd47 blockade
US20250179481A1 (en) 2021-06-01 2025-06-05 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
US11884915B2 (en) 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023167882A1 (en) 2022-03-01 2023-09-07 Artisan Development Labs, Inc. Composition and methods for transgene insertion
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
CN114457119B (zh) * 2022-04-11 2022-08-12 中吉智药(南京)生物技术有限公司 慢病毒载体在制备治疗β-地中海贫血药物中的应用
EP4273242A1 (en) * 2022-05-03 2023-11-08 ETH Zurich Crispr-based modification of human hbd gene
CN117427169A (zh) * 2023-10-24 2024-01-23 中国医学科学院基础医学研究所 同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020182673A1 (en) * 1998-05-15 2002-12-05 Genentech, Inc. IL-17 homologous polypedies and therapeutic uses thereof
EP1590468A2 (en) 2003-01-28 2005-11-02 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
US20100086533A1 (en) 2007-02-19 2010-04-08 Cellectis Laglidadg homing endonuclease variants having novel substrate specificity and use thereof
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
BRPI0919017A2 (pt) * 2008-09-15 2017-05-30 Children's Medical Center Corp métodos para aumentar níveis de hemoglobina fetal em uma célula e em mamífero em necessidade do mesmo, e, para identificar um modulador de expressão ou atividade de bcl11a
US20140148361A1 (en) 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
EP2680877B1 (en) * 2011-02-28 2019-07-10 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drive high efficiency gene disruption
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
KR102218562B1 (ko) * 2012-08-29 2021-02-19 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물

Also Published As

Publication number Publication date
KR20180009383A (ko) 2018-01-26
US10619140B2 (en) 2020-04-14
IL266582A (en) 2019-07-31
CA2865129A1 (en) 2013-08-29
AU2017201235C1 (en) 2018-11-01
AU2017201235A1 (en) 2017-03-16
US20170298329A1 (en) 2017-10-19
ZA201406187B (en) 2018-05-30
EP3272356A1 (en) 2018-01-24
EP2836226B1 (en) 2017-06-28
DK2836226T3 (en) 2017-09-18
EP2836226A4 (en) 2016-04-13
IL234225B (en) 2019-05-30
JP2015516146A (ja) 2015-06-11
HK1249861A1 (en) 2018-11-16
RU2014138491A (ru) 2016-04-10
AU2013222170B2 (en) 2017-01-05
WO2013126794A1 (en) 2013-08-29
EP2836226A1 (en) 2015-02-18
RU2018109507A (ru) 2019-02-28
CN104284669A (zh) 2015-01-14
NZ630900A (en) 2016-10-28
KR101833589B1 (ko) 2018-03-02
AU2017201235B2 (en) 2018-08-02
AU2013222170A1 (en) 2014-10-02
RU2650811C2 (ru) 2018-04-17
BR112014020625A2 (pt) 2017-07-04
JP2017148056A (ja) 2017-08-31
SG10201606959PA (en) 2016-09-29
ES2641840T3 (es) 2017-11-14
SG11201405103SA (en) 2014-09-26
KR20140128453A (ko) 2014-11-05
US20150166969A1 (en) 2015-06-18
JP6170080B2 (ja) 2017-07-26

Similar Documents

Publication Publication Date Title
IN2014DN07853A (cs)
BR112017017812A2 (pt) materiais e métodos para tratamento de hemoglobinopatias
MX2018008733A (es) Metodos para extender la capacidad replicativa de las celulas somaticas durante un proceso de cultivo ex vivo.
MX2022000378A (es) Metodos y composiciones para la modificacion genetica dirigida mediante el uso de arn guia combinados.
CA2853731C (en) Humanized il-6 and il-6 receptor
MX2022015112A (es) Inductor haploide.
MX2015006528A (es) Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.
EP3839050A3 (en) Non-disruptive gene targeting
WO2015164750A3 (en) Compositions and methods to treating hemoglobinopathies
MX2018011791A (es) Compuestos para transduccion viral mejorada.
IN2014DN00288A (cs)
WO2012019168A3 (en) Engineered nucleic acids and methods of use thereof
WO2014143884A3 (en) Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma)
WO2014074852A8 (en) Compositions and methods for modulating an immune response
WO2012087676A3 (en) Production of fatty acids and fatty acid derivatives by recombinant microorganisms
AR092152A1 (es) Uso de una region no traducida de maiz para expresion transgenica en plantas
PH12016502170A1 (en) Humanized dipeptidyl peptidase iv (dpp4) animals
PH12014502038A1 (en) Genetic reduction of male fertility in plants
WO2009101149A3 (en) Anti-tumour effective paramyxovirus
MX350445B (es) Analogos del factor b del complemento y sus usos.
MX357166B (es) Anticuerpos que se unen a notum pectinacetilesterasa.
MX356317B (es) Tratamiento de síntomas asociados a gastroparesia femenina.
MX2016008753A (es) Gen rf3 restaurador tipo s de la esterilidad citoplasmatica masculina del maiz (cms).
MX367031B (es) Plantas para la produccion de proteinas terapeuticas.
MX2016015574A (es) Composiciones esterilizadas de las proteinas ivig y kh para modular los linfocitos y tratar el virus de la hepatitis b.