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

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion 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 (pt)

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 (pt) 2015-04-24

Family

ID=49006265

Family Applications (1)

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

Country Status (18)

Country Link
US (2) US20150166969A1 (pt)
EP (2) EP3272356A1 (pt)
JP (2) JP6170080B2 (pt)
KR (2) KR20180009383A (pt)
CN (1) CN104284669A (pt)
AU (2) AU2013222170B2 (pt)
BR (1) BR112014020625A2 (pt)
CA (1) CA2865129A1 (pt)
DK (1) DK2836226T3 (pt)
ES (1) ES2641840T3 (pt)
HK (1) HK1249861A1 (pt)
IL (2) IL234225B (pt)
IN (1) IN2014DN07853A (pt)
NZ (1) NZ630900A (pt)
RU (2) RU2018109507A (pt)
SG (2) SG11201405103SA (pt)
WO (1) WO2013126794A1 (pt)
ZA (1) ZA201406187B (pt)

Families Citing this family (157)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2012333134B2 (en) 2011-07-22 2017-05-25 John Paul Guilinger Evaluation and improvement of nuclease cleavage specificity
US11021737B2 (en) 2011-12-22 2021-06-01 President And Fellows Of Harvard College Compositions and methods for analyte detection
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
IN2014DN07853A (pt) 2012-02-24 2015-04-24 Hutchinson Fred Cancer Res
NZ728024A (en) 2012-05-25 2019-05-31 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP2890780B8 (en) 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
BR112015011995B1 (pt) 2012-11-27 2023-02-07 Children's Medical Center Corporation Método para a produção de uma célula progenitora hematopoiética tendo bcl11a diminuído ou para aumentar seus níveis de hemoglobina fetal, composição e uso das mesmas
WO2014089290A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
IL293526A (en) 2012-12-12 2022-08-01 Harvard College Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014099750A2 (en) 2012-12-17 2014-06-26 President And Fellows Of Harvard College Rna-guided human genome engineering
US10138509B2 (en) 2013-03-12 2018-11-27 President And Fellows Of Harvard College Method for generating a three-dimensional nucleic acid containing matrix
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
KR102405549B1 (ko) 2013-03-15 2022-06-08 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
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
JP2016522679A (ja) 2013-04-04 2016-08-04 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系を用いたゲノム編集の治療的使用
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
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
KR102282990B1 (ko) 2013-06-04 2021-07-28 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Rna-가이드된 전사 조절
AU2014281031B2 (en) 2013-06-17 2020-05-21 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
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
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
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
SG10201800213VA (en) 2013-07-10 2018-02-27 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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9834791B2 (en) 2013-11-07 2017-12-05 Editas Medicine, Inc. CRISPR-related methods and compositions with governing gRNAS
LT3492593T (lt) * 2013-11-13 2021-11-25 Children's Medical Center Corporation Nukleazės sąlygojama genų raiškos reguliacija
US20160298096A1 (en) * 2013-11-18 2016-10-13 Crispr Therapeutics Ag Crispr-cas system materials and methods
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
EP3079725B1 (en) 2013-12-12 2019-10-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
WO2015103153A1 (en) * 2013-12-31 2015-07-09 The Regents Of The University Of California Cas9 crystals and methods of use thereof
SG10201805815YA (en) 2014-01-08 2018-08-30 Harvard College Rna-guided gene drives
CN113215219A (zh) 2014-02-13 2021-08-06 宝生物工程(美国) 有限公司 从核酸的初始集合中耗尽靶分子的方法、以及用于实践其的组合物和试剂盒
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
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)
EP3777534A1 (en) * 2014-03-26 2021-02-17 The Brigham & Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
WO2015148863A2 (en) * 2014-03-26 2015-10-01 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
BR112016024565A2 (pt) 2014-04-25 2018-01-23 Children's Medical Center Corporation composições e métodos de tratar hemoglobinopatias
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016025759A1 (en) * 2014-08-14 2016-02-18 Shen Yuelei Dna knock-in system
CA2957802A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses
LT3194570T (lt) * 2014-09-16 2021-09-27 Sangamo Therapeutics, Inc. Būdai ir kompozicijos, skirti kraujodaros kamieninių ląstelių genomo inžinerijai ir korekcijai, kuriai tarpininkauja nukleazė
WO2016049531A1 (en) 2014-09-26 2016-03-31 Purecircle Usa Inc. Single nucleotide polymorphism (snp) markers for stevia
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
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)
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
EP3247366A4 (en) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
SG10202101358XA (en) 2015-01-26 2021-03-30 Fate Therapeutics Inc Methods and compositions for inducing hematopoietic cell differentiation
SG11201706767RA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3262173A2 (en) * 2015-02-23 2018-01-03 Crispr Therapeutics AG Materials and methods for treatment of human genetic diseases including hemoglobinopathies
AU2016246450B2 (en) 2015-04-06 2022-03-17 Agilent Technologies, Inc. Chemically modified guide RNAs for CRISPR/Cas-mediated gene regulation
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
CN108026566A (zh) * 2015-05-04 2018-05-11 特拉维夫大学拉莫特有限公司 用于使dna片段化的方法和试剂盒
EP3294873B1 (en) * 2015-05-08 2020-08-19 The Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
WO2016182959A1 (en) * 2015-05-11 2016-11-17 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
AU2016278982A1 (en) * 2015-06-17 2018-01-18 The Uab Research Foundation CRISPR/Cas9 complex for genomic editing
AU2016278959A1 (en) 2015-06-17 2018-01-18 The Uab Research Foundation CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
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
US9897378B2 (en) 2015-10-08 2018-02-20 Nyc Designed Inspirations Llc Cosmetic makeup sponge/blender container
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
US20180325947A1 (en) * 2015-10-27 2018-11-15 Children's Hospital Medical Center Use of mapk inhibitors to reduce loss of hematopoietic stem cells during ex vivo culture and/or genetic manipulation
CA3004285A1 (en) 2015-11-03 2017-05-11 President And Fellows Of Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
EP3371306B8 (en) * 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN115927199A (zh) * 2015-11-04 2023-04-07 菲特治疗公司 用于诱导造血细胞分化的方法和组合物
ES2953925T3 (es) 2015-11-04 2023-11-17 Fate Therapeutics Inc Ingeniería genómica de células pluripotentes
SG11201805217XA (en) * 2015-12-28 2018-07-30 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
CN116904404A (zh) 2016-01-27 2023-10-20 昂克诺斯公司 溶瘤病毒载体及其用途
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
EP3430142A1 (en) * 2016-03-14 2019-01-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta hemoglobinopathies
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
KR102351329B1 (ko) * 2016-04-18 2022-01-18 크리스퍼 테라퓨틱스 아게 혈색소병증의 치료를 위한 물질 및 방법
CA3022290A1 (en) 2016-04-25 2017-11-02 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
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
CA3031785A1 (en) * 2016-07-25 2018-02-01 Bluebird Bio, Inc. Bcl11a homing endonuclease variants, compositions, and methods of use
SG11201900907YA (en) 2016-08-03 2019-02-27 Harvard College Adenosine nucleobase editors and uses thereof
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
WO2018031683A1 (en) 2016-08-09 2018-02-15 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
US11542509B2 (en) 2016-08-24 2023-01-03 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
IL265045B2 (en) 2016-09-08 2023-09-01 Bluebird Bio Inc Variants of endonuclease pd-1, compositions and methods of use
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
EP3574088A4 (en) 2017-01-27 2020-09-23 Children's Hospital Medical Center METHOD FOR INCREASING THE CLOSURE ACTIVITY OF HEMATOPOETIC 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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
US20210222201A1 (en) * 2017-04-24 2021-07-22 Seattie 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
WO2018218135A1 (en) 2017-05-25 2018-11-29 The Children's Medical Center Corporation Bcl11a guide delivery
EP3630198A4 (en) 2017-05-25 2021-04-21 The General Hospital Corporation USE OF CLIVED DESAMINASES TO LIMIT UNWANTED OUT-OF-TARGET DESAMINATION FROM BASE EDITING
EP3635120A1 (en) * 2017-06-02 2020-04-15 Institut National de la Santé et de la Recherche Médicale (INSERM) Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
JP7249323B2 (ja) 2017-07-26 2023-03-30 オンコラス, インコーポレイテッド 腫瘍溶解性ウイルスベクター及びその使用
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
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
EP3694543A1 (en) 2017-10-13 2020-08-19 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP3697906A1 (en) 2017-10-16 2020-08-26 The Broad Institute, Inc. Uses of adenosine base editors
WO2019081982A1 (en) * 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
CN109722415B (zh) * 2017-10-27 2021-01-26 博雅辑因(北京)生物科技有限公司 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
WO2019113149A1 (en) * 2017-12-05 2019-06-13 Crispr Therapeutics Ag Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
MA51788A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes pour traiter des hémoglobinopathies
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス,インコーポレーテッド 寛容原性リポソーム及びその使用方法
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
EP3781677A4 (en) 2018-04-16 2022-01-19 University of Massachusetts COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING
JP2021521838A (ja) * 2018-04-27 2021-08-30 シアトル チルドレンズ ホスピタル (ディービーエイ シアトル チルドレンズ リサーチ インスティテュート) ブルトン型チロシンキナーゼに対するtalenベースのおよびcrispr/casベースのゲノム編集
WO2020072699A1 (en) * 2018-10-02 2020-04-09 Exosome Therapeutics, Inc. Exosome loaded therapeutics for treating sickle cell disease
KR20210102925A (ko) * 2018-12-10 2021-08-20 블루버드 바이오, 인코포레이티드. 호밍 엔도뉴클레아제 변이체
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
AU2020223060B2 (en) * 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
EP3942042A1 (en) 2019-03-19 2022-01-26 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
EP3964579A4 (en) * 2019-04-30 2023-07-26 EdiGene (GuangZhou) Inc. METHOD OF PREDICTING THE EFFECTIVENESS OF THE TREATMENT OF HEMOGLOBINOPATHY
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
AU2020284555A1 (en) 2019-05-28 2021-12-23 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
US20220380756A1 (en) * 2019-06-28 2022-12-01 Asc Therapeutics Inc. Methods and compositions for treating thalassemia or sickle cell disease
EP4038190A1 (en) 2019-10-03 2022-08-10 Artisan Development Labs, Inc. Crispr systems with engineered dual guide nucleic acids
JP2023502626A (ja) * 2019-11-15 2023-01-25 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー ヒト造血幹前駆細胞におけるアルファ-グロビン遺伝子座での標的化組み込み
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
KR20230019843A (ko) 2020-05-08 2023-02-09 더 브로드 인스티튜트, 인코퍼레이티드 표적 이중 가닥 뉴클레오티드 서열의 두 가닥의 동시 편집을 위한 방법 및 조성물
EP4150081A1 (en) * 2020-05-13 2023-03-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
AU2021410632A1 (en) * 2020-12-21 2023-08-03 2Seventy Bio, Inc. Compositions and methods for site-directed mutagenesis
KR20230157388A (ko) 2021-03-12 2023-11-16 멘두스 비.브이. 백신접종의 방법 및 cd47 차단의 용도
WO2022256448A2 (en) 2021-06-01 2022-12-08 Artisan Development Labs, Inc. 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
CA3230927A1 (en) 2021-09-10 2023-03-16 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

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
JP4966006B2 (ja) 2003-01-28 2012-07-04 セレクティス カスタムメイドメガヌクレアーゼおよびその使用
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
CA2678526A1 (en) 2007-02-19 2008-08-28 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
ES2738980T3 (es) * 2008-09-15 2020-01-28 Childrens Medical Ct Corp Modulación de BCL11A para el tratamiento de hemoglobinopatías
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
ES2906059T3 (es) * 2011-02-28 2022-04-13 Seattle Childrens Res Institute Endonucleasas de acoplamiento con enzimas de procesamiento de extremos que impulsan la interrupción génica de alta eficiencia
IN2014DN07853A (pt) 2012-02-24 2015-04-24 Hutchinson Fred Cancer Res
EP2890780B8 (en) * 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition

Also Published As

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

Similar Documents

Publication Publication Date Title
IN2014DN07853A (pt)
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.
MX2016013832A (es) Composiciones y metodos para tratar hemoglobinopatias.
MX2015006528A (es) Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.
AU2016277883A8 (en) PD-1-CD28 fusion proteins and their use in medicine
MY172726A (en) Humanized il-6 and il-6 receptor
MX2018011791A (es) Compuestos para transduccion viral mejorada.
MX2017008283A (es) Inductor haploide.
MX2016001204A (es) Vectores virales adeno-asociados (aav) utiles para transducir tejido adiposo.
WO2014074852A8 (en) Compositions and methods for modulating an immune response
WO2014143884A3 (en) Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma)
MX354516B (es) Vectores que codifican el factor de viabilidad de conos derivado de bastones.
WO2012087676A3 (en) Production of fatty acids and fatty acid derivatives by recombinant microorganisms
PH12014502038A1 (en) Genetic reduction of male fertility in plants
MX2013012791A (es) Analogos del factor b del complemento y sus usos.
AR092152A1 (es) Uso de una region no traducida de maiz para expresion transgenica en plantas
MX2010008942A (es) Paramyxovirus eficaz como antitumoral.
MX357166B (es) Anticuerpos que se unen a notum pectinacetilesterasa.
MX356317B (es) Tratamiento de síntomas asociados a gastroparesia femenina.
MX2016015574A (es) Composiciones esterilizadas de las proteinas ivig y kh para modular los linfocitos y tratar el virus de la hepatitis b.
MX2015000777A (es) Terapia basada en baclofeno y acamprosato de trastornos de degeneración macular..
MX367031B (es) Plantas para la produccion de proteinas terapeuticas.
PH12015501109A1 (en) Novel compounds as diacylglycerol acyltransferase inhibitors