SA519402400B1 - تركيبات وطرق لعلاج أمراض الهيموجلوبين - Google Patents

تركيبات وطرق لعلاج أمراض الهيموجلوبين

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Publication number
SA519402400B1
SA519402400B1 SA519402400A SA519402400A SA519402400B1 SA 519402400 B1 SA519402400 B1 SA 519402400B1 SA 519402400 A SA519402400 A SA 519402400A SA 519402400 A SA519402400 A SA 519402400A SA 519402400 B1 SA519402400 B1 SA 519402400B1
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SA
Saudi Arabia
Prior art keywords
hemoglobinopathies
treatment
methods
compositions
reagents
Prior art date
Application number
SA519402400A
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English (en)
Inventor
سنيد جينفير
سي. ستيفنسون سوزان
ستيفن ميكانين كريج
شــميدت كريستــيان
يانغ يي
Original Assignee
إنتيليا ثيرابيوتيكس ، إنك.
نوفارتيس أيه جي
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Publication date
Application filed by إنتيليا ثيرابيوتيكس ، إنك., نوفارتيس أيه جي filed Critical إنتيليا ثيرابيوتيكس ، إنك.
Publication of SA519402400B1 publication Critical patent/SA519402400B1/ar

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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 RNAses, DNAses
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)

Abstract

يتعلق الاختراع الحالي بأنظمة تعديل الجينوم، عوامل كاشفة وطرق لعلاج أمراض الهيموجلوبين. الشكل 1.
SA519402400A 2017-02-06 2019-08-06 تركيبات وطرق لعلاج أمراض الهيموجلوبين SA519402400B1 (ar)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762455464P 2017-02-06 2017-02-06

Publications (1)

Publication Number Publication Date
SA519402400B1 true SA519402400B1 (ar) 2023-11-20

Family

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Family Applications (1)

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SA519402400A SA519402400B1 (ar) 2017-02-06 2019-08-06 تركيبات وطرق لعلاج أمراض الهيموجلوبين

Country Status (18)

Country Link
US (2) US11466271B2 (ar)
EP (1) EP3577223A1 (ar)
JP (2) JP2020505934A (ar)
KR (2) KR102630217B1 (ar)
CN (1) CN110546262B (ar)
AR (1) AR110962A1 (ar)
AU (3) AU2018215726B2 (ar)
BR (1) BR112019016205A2 (ar)
CA (1) CA3052275A1 (ar)
EA (1) EA201991862A1 (ar)
IL (2) IL311222A (ar)
MX (1) MX2019009361A (ar)
PH (1) PH12019501812A1 (ar)
RU (1) RU2019127919A (ar)
SA (1) SA519402400B1 (ar)
SG (1) SG11201907056XA (ar)
TW (1) TW201839136A (ar)
WO (1) WO2018142364A1 (ar)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9725710B2 (en) 2014-01-08 2017-08-08 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11390884B2 (en) 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells
KR20180031671A (ko) 2015-06-09 2018-03-28 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and their uses
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
CA3032699A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018071868A1 (en) 2016-10-14 2018-04-19 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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
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
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US20200140896A1 (en) * 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
KR102439221B1 (ko) 2017-12-14 2022-09-01 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
AU2019236209A1 (en) 2018-03-14 2020-10-01 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2020113112A1 (en) * 2018-11-29 2020-06-04 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2020112195A1 (en) * 2018-11-30 2020-06-04 Yale University Compositions, technologies and methods of using plerixafor to enhance gene editing
CN114096666A (zh) 2019-02-13 2022-02-25 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
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
CA3138029A1 (en) * 2019-04-30 2020-11-05 Edigene Inc. Method for predicting effectiveness of treatment of hemoglobinopathy
CN110106203B (zh) * 2019-05-24 2023-08-11 中国医学科学院血液病医院（血液学研究所） 一种新型hbb过表达载体及其设计方法和应用
AU2020380812A1 (en) * 2019-11-06 2022-06-09 Syros Pharmaceuticals, Inc. Compositions and methods for treating sickle cell disease
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
CN111575306B (zh) * 2020-06-11 2022-08-05 南方医科大学 一种激活红细胞中γ珠蛋白基因表达的方法
WO2022036306A1 (en) * 2020-08-14 2022-02-17 Navan Technologies, Inc. Methods and apparatuses for biomolecule delivery to primary human hematopoietic stem cells using nanostraws
WO2023164636A1 (en) * 2022-02-25 2023-08-31 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
WO2024163679A1 (en) * 2023-02-01 2024-08-08 Prime Medicine, Inc. Genome editing compositions and methods for treatment of cystic fibrosis

Family Cites Families (236)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5800815A (en) 1903-05-05 1998-09-01 Cytel Corporation Antibodies to P-selectin and their uses
US6309639B1 (en) 1991-02-05 2001-10-30 The Board Of Regents Of The University Of Oklahoma Method for inhibiting an inflammatory response using antibodies to P-selectin glycoprotein ligand
US5213796A (en) 1991-05-06 1993-05-25 Dana Farber Cancer Institute Assay for polyomavirus in humans and uses thereof
US5726014A (en) 1991-06-27 1998-03-10 Genelabs Technologies, Inc. Screening assay for the detection of DNA-binding molecules
US5578444A (en) 1991-06-27 1996-11-26 Genelabs Technologies, Inc. Sequence-directed DNA-binding molecules compositions and methods
DK0642356T3 (da) 1992-05-05 2003-08-04 Aeres Biomedical Ltd Antistoffer mod P-selectin og deres anvendelser
US7910624B1 (en) 1995-03-03 2011-03-22 The Trustees Of Boston University Compositions for the treatment of blood disorders
JP3971454B2 (ja) 1993-10-29 2007-09-05 ザ トラスティーズ オブ ボストン ユニバーシティ 抗新生物剤としての酪酸、酪酸塩、および誘導体の生理学的に安定な組成物
US5622701A (en) 1994-06-14 1997-04-22 Protein Design Labs, Inc. Cross-reacting monoclonal antibodies specific for E- and P-selectin
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6780590B2 (en) 1999-09-14 2004-08-24 Sangamo Biosciences, Inc. Gene identification
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
DE60023936T2 (de) 1999-12-06 2006-05-24 Sangamo Biosciences Inc., Richmond Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
IT1320168B1 (it) 2000-03-13 2003-11-18 Univ Ferrara Oligonucleotidi sintetici in grado di indurre il differenziamentoeritroide.
WO2001085765A2 (en) 2000-05-12 2001-11-15 Merck Patent Gmbh Identification of mfq-110, a human c2h2-type zinc finger protein
US20050054836A1 (en) 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
AU2002219992A1 (en) 2000-12-01 2002-06-11 Sangamo Biosciences, Inc. Targeted regulation of gene expression
JP2005527180A (ja) 2001-04-18 2005-09-15 プロテイン デザイン ラブス， インコーポレイテッド 肺がんの診断方法、肺がんの修飾因子の組成及びスクリーニングの方法
US8735153B2 (en) 2001-09-24 2014-05-27 Sangamo Biosciences, Inc. Modulation of stem cells using zinc finger proteins
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
WO2004054512A2 (en) 2002-12-13 2004-07-01 Genetix Pharmaceuticals, Inc. Therapeutic retroviral vectors for gene therapy
US20050025753A1 (en) 2003-04-30 2005-02-03 Wei Han Methods for production of non-disease causing hemoglobin by ex vivo oligonucleotide gene editing of human stem/progenitor cells
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
CA2534296C (en) 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2004305111B2 (en) 2003-12-19 2011-04-28 Novartis Vaccines And Diagnostics, Inc. Cell transfecting formulations of small interfering RNA, related compositions and methods of making and use
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP2357201B1 (en) 2004-04-13 2017-08-30 F. Hoffmann-La Roche AG Anti-P-selectin antibodies
WO2007014181A2 (en) 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
US20110212096A1 (en) 2006-12-01 2011-09-01 Scott Rollins Anti-p-selectin antibodies and methods of their use and identification
US8945565B2 (en) 2006-12-01 2015-02-03 Selexys Pharmaceuticals Corporation Methods of treating inflammatory or thrombotic conditions with anti-P-selectin antibodies
EP2097105A4 (en) 2006-12-01 2010-09-15 Selexys Pharmaceuticals Corp ANTI-P-SELECTINE ANTIBODIES AND METHODS FOR USE IN THE TREATMENT OF INFLAMMATORY DISEASES
GB0713183D0 (en) 2007-07-06 2007-08-15 King S College London Method
ES2738980T3 (es) 2008-09-15 2020-01-28 Childrens Medical Ct Corp Modulación de BCL11A para el tratamiento de hemoglobinopatías
PE20100362A1 (es) 2008-10-30 2010-05-27 Irm Llc Derivados de purina que expanden las celulas madre hematopoyeticas
BR112012015755B1 (pt) 2009-12-23 2021-06-22 Novartis Ag Lipídeo furtivo, e composição
WO2011103215A1 (en) 2010-02-16 2011-08-25 Mount Sinai School Of Medicine Embedded chimeric peptide nucleic acids and use thereof
WO2012075027A1 (en) 2010-11-29 2012-06-07 Mount Sinai School Of Medicine Embedded chimeric peptide nucleic acids for generation of induced pluripotent stem cells
WO2012006378A1 (en) 2010-07-06 2012-01-12 Novartis Ag Liposomes with lipids having an advantageous pka- value for rna delivery
US20140127814A1 (en) 2010-08-10 2014-05-08 Srinivasan Chandrasegaran Generation and use of pluripotent stem cells
AU2011295938B2 (en) 2010-08-31 2016-01-14 Glaxosmithkline Biologicals S.A. Lipids suitable for liposomal delivery of protein-coding RNA
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
WO2012112232A1 (en) 2011-02-18 2012-08-23 Academia Sinica METHODS AND COMPOSITIONS FOR TREATING β-THALASSEMIA AND SICKLE CELL DISEASE
DE202011050611U1 (de) 2011-07-01 2012-10-09 Makita Corporation Anordnung zum Bereitstellen eines Kraftstoff-Luftgemischs für eine Brennkraftmaschine
EP4014966A1 (en) 2011-07-06 2022-06-22 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
WO2013019745A1 (en) 2011-07-29 2013-02-07 Georgia Health Sciences University Methods and compositions for genetically modifiying cells
US20140271657A1 (en) 2011-10-12 2014-09-18 Children's Medical Center Corporation Combinatorial compositions and methods of treating hemoglobinopathies
LT2768942T (lt) 2011-10-17 2020-04-10 Massachusetts Institute Of Technology Pristatymas į ląstelės vidų
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
AU2013212457B2 (en) 2012-01-27 2017-02-16 Universite De Montreal Pyrimido[4,5-b]indole derivatives and use thereof in the expansion of hematopoietic stem cells
IN2014DN07853A (ar) 2012-02-24 2015-04-24 Hutchinson Fred Cancer Res
EP2839013B1 (en) 2012-04-18 2020-08-26 The Board of Trustees of the Leland Stanford Junior University Non-disruptive gene targeting
DE202013012241U1 (de) 2012-05-25 2016-01-18 Emmanuelle Charpentier Zusammensetzungen für die durch RNA gesteuerte Modifikation einer Ziel-DNA und für die durch RNA gesteuerte Modulation der Transkription
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
CN104718284A (zh) 2012-05-25 2015-06-17 塞勒克提斯公司 工程化用于免疫疗法的异体和免疫抑制耐受性t细胞的方法
KR20150023670A (ko) 2012-06-12 2015-03-05 제넨테크, 인크. 조건적 녹아웃 대립유전자의 생성 방법 및 이를 위한 조성물
EP2872154B1 (en) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Methods and compositions for delivery of biologics
ES2757623T3 (es) 2012-07-25 2020-04-29 Broad Inst Inc Proteínas de unión a ADN inducibles y herramientas de perturbación genómica y aplicaciones de las mismas
KR102474010B1 (ko) 2012-08-29 2022-12-02 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
EP2893004B1 (en) 2012-09-04 2018-10-24 Cellectis Multi-chain chimeric antigen receptor and uses thereof
ES2926021T3 (es) 2012-10-23 2022-10-21 Toolgen Inc Composición para escindir un ADN objetivo que comprende un ARN guía específico para el ADN objetivo y ácido nucleico codificador de proteína Cas o proteína Cas, y uso de la misma
SG10202110062SA (en) 2012-11-27 2021-11-29 Childrens Medical Center Targeting Bcl11a Distal Regulatory Elements for Fetal Hemoglobin Reinduction
PL2928496T3 (pl) 2012-12-06 2020-04-30 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu w oparciu o CRISPR
MX2015007549A (es) 2012-12-12 2017-01-20 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
PL2896697T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Projektowanie systemów, sposoby i optymalizowane kompozycje kierujące do manipulacji sekwencją
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP2931892B1 (en) 2012-12-12 2018-09-12 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
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
PL2898075T3 (pl) 2012-12-12 2016-09-30 PROJEKTOWANIE i OPTYMALIZACJA ULEPSZONYCH SYSTEMÓW, SPOSOBY I KOMPOZYCJE ENZYMÓW DO MANIPULACJI SEKWENCJĄ
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
EP2825654B1 (en) 2012-12-12 2017-04-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
US8993233B2 (en) 2012-12-12 2015-03-31 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
RU2699523C2 (ru) 2012-12-17 2019-09-05 Президент Энд Фэллоуз Оф Харвард Коллидж Рнк-направляемая инженерия генома человека
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
ES2750550T3 (es) 2013-03-01 2020-03-26 Univ Minnesota Corrección de gen a base de TALEN
US10124065B2 (en) 2013-03-08 2018-11-13 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
KR102210322B1 (ko) 2013-03-15 2021-02-01 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
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
ES2923018T3 (es) 2013-03-15 2022-09-22 Glaxosmithkline Biologicals Sa Métodos de purificación de ARN
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
CA2902709A1 (en) 2013-03-15 2014-09-25 Global Blood Therapeutics, Inc. Compositions and methods for the modulation of hemoglobin (s)
ES2747833T3 (es) 2013-04-04 2020-03-11 Dartmouth College Composiciones y procedimientos para la escisión in vivo del ADN proviral del vih-1
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
MX2015015662A (es) 2013-05-13 2016-09-16 Cellectis Receptor quimérico de antígeno especifico para cd19 y sus usos.
MX2015015638A (es) 2013-05-13 2016-10-28 Cellectis Metodos para diseñar celulas t altamente activas para inmunoterapia.
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
KR20160013110A (ko) 2013-05-24 2016-02-03 로슈 이노베이션 센터 코펜하겐 에이/에스 B-세포 cll/림프종 11a(bcl11a)의 올리고뉴클레오티드 조절제 및 이의 용도
EP3004337B1 (en) 2013-05-29 2017-08-02 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
EP3003392B1 (en) 2013-06-04 2019-10-23 President and Fellows of Harvard College Rna-guideded transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
AU2014274840B2 (en) 2013-06-05 2020-03-12 Duke University RNA-guided gene editing and gene regulation
WO2014200659A1 (en) 2013-06-11 2014-12-18 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使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
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
KR20160034901A (ko) 2013-06-17 2016-03-30 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작에 최적화된 ｃｒｉｓｐｒ-ｃａｓ 이중 닉카아제 시스템, 방법 및 조성물
CN104239331B (zh) 2013-06-19 2018-10-09 阿里巴巴集团控股有限公司 一种用于实现评论搜索引擎排序的方法和装置
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
RU2748433C2 (ru) 2013-07-10 2021-05-25 Президент Энд Фэллоуз Оф Харвард Коллидж ОРТОГОНАЛЬНЫЕ БЕЛКИ Cas9 ДЛЯ РНК-НАПРАВЛЯЕМОЙ РЕГУЛЯЦИИ И РЕДАКТИРОВАНИЯ ГЕНОВ
JP7019233B2 (ja) 2013-07-11 2022-02-15 モデルナティエックス インコーポレイテッド ＣＲＩＳＰＲ関連タンパク質をコードする合成ポリヌクレオチドおよび合成ｓｇＲＮＡを含む組成物ならびに使用方法
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
KR102304167B1 (ko) 2013-08-16 2021-09-24 메사추세츠 인스티튜트 오브 테크놀로지 세포로의 선택적 물질 전달
EP3036334A1 (en) 2013-08-22 2016-06-29 E. I. du Pont de Nemours and Company A soybean u6 polymerase iii promoter and methods of use
GB201315321D0 (en) 2013-08-28 2013-10-09 Koninklijke Nederlandse Akademie Van Wetenschappen Transduction Buffer
EP3041498B1 (en) 2013-09-05 2022-02-16 Massachusetts Institute of Technology Tuning microbial populations with programmable nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
DE202014010413U1 (de) 2013-09-18 2015-12-08 Kymab Limited Zellen und Organismen
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
JP5774657B2 (ja) 2013-10-04 2015-09-09 国立大学法人京都大学 エレクトロポレーションを利用した哺乳類の遺伝子改変方法
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
WO2015054507A1 (en) 2013-10-10 2015-04-16 Pronutria, Inc. Nutritive polypeptide production systems, and methods of manufacture and use thereof
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
US9850497B2 (en) 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物
PT3492593T (pt) 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
EP3071695A2 (en) 2013-11-18 2016-09-28 Crispr Therapeutics AG Crispr-cas system materials and methods
BR112016011169A2 (pt) 2013-11-22 2017-09-19 Cellectis Método para a produção de células imunes ex vivo que são resistentes a um fármaco análogo de purina, célula t isolada resistente a um análogo de purina, composição farmacêutica e uso das mesmas
US10450586B2 (en) 2013-11-28 2019-10-22 Horizon Discovery Limited Somatic haploid human cell line
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
KR20160089527A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
CA2932472A1 (en) 2013-12-12 2015-06-18 Massachusetts Institute Of Technology Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
AU2014361784A1 (en) 2013-12-12 2016-06-23 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
EP3083579B1 (en) 2013-12-19 2022-01-26 Novartis AG Lipids and lipid compositions for the delivery of active agents
CN106029886B (zh) 2013-12-19 2021-02-05 阿迈瑞斯公司 基因组整合的方法
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
EP3985124A1 (en) 2013-12-26 2022-04-20 The General Hospital Corporation Multiplex guide rnas
EP3089989B1 (en) 2013-12-31 2020-06-24 The Regents of The University of California Cas9 crystals and methods of use thereof
AU2015204784B2 (en) 2014-01-08 2021-01-28 President And Fellows Of Harvard College RNA-guided gene drives
CA2936646C (en) 2014-01-24 2024-04-30 North Carolina State University Methods and compositions for sequences guiding cas9 targeting
EP3102673B1 (en) 2014-02-03 2020-04-15 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
US10287590B2 (en) 2014-02-12 2019-05-14 Dna2.0, Inc. Methods for generating libraries with co-varying regions of polynuleotides for genome modification
CA2939621C (en) 2014-02-13 2019-10-15 Takara Bio Usa, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
SG11201606819QA (en) 2014-02-18 2016-09-29 Univ Duke Compositions for the inactivation of virus replication and methods of making and using the same
EP3971283A1 (en) 2014-02-27 2022-03-23 Monsanto Technology LLC Compositions and methods for site directed genomic modification
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
WO2015139139A1 (en) 2014-03-20 2015-09-24 UNIVERSITé LAVAL Crispr-based methods and products for increasing frataxin levels and uses thereof
EP3916007A1 (en) 2014-03-24 2021-12-01 Immco Diagnostics, Inc. Improved anti-nuclear antibody detection and diagnostics for systemic and non-systemic autoimmune disorders
CA2943622A1 (en) 2014-03-25 2015-10-01 Editas Medicine Inc. Crispr/cas-related methods and compositions for treating hiv infection and aids
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
WO2015152305A1 (ja) 2014-03-31 2015-10-08 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
EP4123024A1 (en) 2014-04-01 2023-01-25 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
WO2015153791A1 (en) 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 2 (hsv-2)
US20170029850A1 (en) 2014-04-02 2017-02-02 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
JP2017509350A (ja) 2014-04-03 2017-04-06 マサチューセッツ インスティテュート オブ テクノロジー ガイドｒｎａの生成のための方法および組成物
EP3129485B2 (en) 2014-04-09 2022-12-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
PL3132034T3 (pl) 2014-04-14 2021-04-06 Nemesis Bioscience Ltd. Środek terapeutyczny
SG10201912171PA (en) 2014-04-18 2020-02-27 Editas Medicine Inc Crispr-cas-related methods, compositions and components for cancer immunotherapy
SG10201809290SA (en) 2014-04-25 2019-01-30 Childrens Medical Ct Corp Compositions and Methods to Treating Hemoglobinopathies
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
WO2015168800A1 (en) 2014-05-09 2015-11-12 UNIVERSITé LAVAL Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
WO2015173436A1 (en) 2014-05-16 2015-11-19 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
US20170175143A1 (en) 2014-05-20 2017-06-22 Regents Of The University Of Minnesota Method for editing a genetic sequence
WO2015188056A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
KR102374379B1 (ko) 2014-06-06 2022-03-17 리제너론 파마슈티칼스 인코포레이티드 표적화된 좌를 변형시키는 방법 및 조성물
EP3155116A4 (en) 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
WO2015191911A2 (en) 2014-06-12 2015-12-17 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
EP3354732B1 (en) 2014-06-23 2020-01-08 Regeneron Pharmaceuticals, Inc. Nuclease-mediated dna assembly
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
US20150376587A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
SG11201610633QA (en) 2014-06-26 2017-01-27 Regeneron Pharma Methods and compositions for targeted genetic modifications and methods of use
US10017770B2 (en) 2014-07-03 2018-07-10 Ut-Battelle, Llc TNT cloning system
WO2016007948A1 (en) 2014-07-11 2016-01-14 Pioneer Hi-Bred International, Inc. Agronomic trait modification using guide rna/cas endonuclease systems and methods of use
US11254933B2 (en) 2014-07-14 2022-02-22 The Regents Of The University Of California CRISPR/Cas transcriptional modulation
US10975406B2 (en) 2014-07-18 2021-04-13 Massachusetts Institute Of Technology Directed endonucleases for repeatable nucleic acid cleavage
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CN107208093B (zh) 2014-09-04 2022-03-01 纪念斯隆-凯特琳癌症中心 用于治疗血红蛋白病的球蛋白基因治疗
PL3194570T3 (pl) 2014-09-16 2021-12-20 Sangamo Therapeutics, Inc. Sposoby i kompozycje do inżynierii genomowej w której pośredniczy nukleaza i korekty hematopoetycznych komórek macierzystych
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
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)
AU2015369725A1 (en) 2014-12-24 2017-06-29 Massachusetts Institute Of Technology CRISPR having or associated with destabilization domains
AU2015370435A1 (en) 2014-12-24 2017-06-15 Dana-Farber Cancer Institute, Inc. Systems and methods for genome modification and regulation
CN107429263A (zh) 2015-01-15 2017-12-01 斯坦福大学托管董事会 调控基因组编辑的方法
EP3247808B1 (en) 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
EP3247366A4 (en) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
EP3838296A1 (en) 2015-01-21 2021-06-23 Cornell University Viral vectors for prophylaxis and therapy of hemoglobinopathies
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
BR112017017810A2 (pt) 2015-02-23 2018-04-10 Crispr Therapeutics Ag materiais e métodos para tratamento de hemoglobinopatias
US20180271891A1 (en) 2015-03-11 2018-09-27 The Broad Institute Inc. Selective treatment of prmt5 dependent cancer
EP3274454B1 (en) 2015-03-25 2021-08-25 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
US20160281111A1 (en) 2015-03-26 2016-09-29 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
CN108012545B (zh) 2015-03-31 2022-08-30 葛莱高托普有限公司 包含珠蛋白基因簇的调控元件的真核表达载体
CA2981715A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
US11572543B2 (en) 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
US11390884B2 (en) 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells
MX2017014446A (es) 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
WO2016201272A1 (en) 2015-06-12 2016-12-15 King Abdulaziz City For Science And Technology Method of diagnosing patients with conditions caused by mendelian mutations
US11643668B2 (en) 2015-06-17 2023-05-09 The Uab Research Foundation CRISPR/Cas9 complex for genomic editing
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
WO2017059241A1 (en) 2015-10-02 2017-04-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Lentiviral protein delivery system for rna-guided genome editing
EP3371306B8 (en) 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP4053277A1 (en) 2015-12-28 2022-09-07 Novartis AG Compositions and methods for the treatment of hemoglobinopathies
IL313038A (en) * 2016-03-14 2024-07-01 Editas Medicine Inc Methods and preparations related to CRISPR/CAS - for the treatment of diseases in the hemoglobin cell
KR102351329B1 (ko) 2016-04-18 2022-01-18 크리스퍼 테라퓨틱스 아게 혈색소병증의 치료를 위한 물질 및 방법
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US20190345450A1 (en) 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
US20190316154A1 (en) 2016-07-07 2019-10-17 St. Jude Children's Research Hospital Erythroid-specific promoter and method of use thereof
EP3487994A4 (en) 2016-07-25 2020-01-29 Bluebird Bio, Inc. BCL11A HOMING ENDONUCLEASE VERSIONS, COMPOSITIONS AND METHODS OF USE
US20190169597A1 (en) 2016-08-19 2019-06-06 Bluebird Bio, Inc. Genome editing enhancers
JP7203014B2 (ja) 2016-08-24 2023-01-12 サンガモ セラピューティクス， インコーポレイテッド 工学操作されたヌクレアーゼを使用した遺伝子発現の調節
CN110418841A (zh) 2016-08-24 2019-11-05 桑格摩生物治疗股份有限公司 工程化的靶特异性核酸酶
JP2019532672A (ja) 2016-09-28 2019-11-14 ノバルティス アーゲー 多孔質膜系巨大分子送達システム

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