MX375592B - Composiciones y su uso para tratar hemoglobinopatias. - Google Patents

Composiciones y su uso para tratar hemoglobinopatias.

Info

Publication number
MX375592B
MX375592B MX2016013832A MX2016013832A MX375592B MX 375592 B MX375592 B MX 375592B MX 2016013832 A MX2016013832 A MX 2016013832A MX 2016013832 A MX2016013832 A MX 2016013832A MX 375592 B MX375592 B MX 375592B
Authority
MX
Mexico
Prior art keywords
compositions
treat hemoglobinopathies
expression
hemoglobinopathies
treat
Prior art date
Application number
MX2016013832A
Other languages
English (en)
Other versions
MX2016013832A (es
Inventor
David A Williams
Michael Milsom
Richard Gregory
Original Assignee
The Children´S Medical Center Corp
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 The Children´S Medical Center Corp filed Critical The Children´S Medical Center Corp
Publication of MX2016013832A publication Critical patent/MX2016013832A/es
Publication of MX375592B publication Critical patent/MX375592B/es

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
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/11—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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Las modalidades en la presente proporcionan microRNAs con dirección a BCL11A sintéticos, especialmente diseñados para la expresión de RNA polimerasa II, y métodos de uso para tratar hemoglobinopatías tal como la enfermedad de células falciformes o talasemia al incrementar los niveles de expresión de los niveles de hemoglobina fetal.
MX2016013832A 2014-04-25 2015-04-24 Composiciones y su uso para tratar hemoglobinopatias. MX375592B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461984247P 2014-04-25 2014-04-25
US201462066783P 2014-10-21 2014-10-21
PCT/US2015/027527 WO2015164750A2 (en) 2014-04-25 2015-04-24 Compositions and methods to treating hemoglobinopathies

Publications (2)

Publication Number Publication Date
MX2016013832A MX2016013832A (es) 2017-03-09
MX375592B true MX375592B (es) 2025-03-06

Family

ID=54333430

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016013832A MX375592B (es) 2014-04-25 2015-04-24 Composiciones y su uso para tratar hemoglobinopatias.

Country Status (17)

Country Link
US (4) US10287588B2 (es)
EP (2) EP3598984B1 (es)
JP (2) JP6514717B2 (es)
KR (1) KR102390629B1 (es)
CN (2) CN106794260B (es)
AU (1) AU2015249381B2 (es)
BR (1) BR112016024565A2 (es)
CA (1) CA2946309C (es)
DK (1) DK3134130T3 (es)
ES (1) ES2744186T3 (es)
IL (1) IL248469B (es)
MX (1) MX375592B (es)
RU (1) RU2707540C2 (es)
SA (1) SA516380143B1 (es)
SG (2) SG11201608482UA (es)
WO (1) WO2015164750A2 (es)
ZA (1) ZA201607540B (es)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109554350B (zh) 2012-11-27 2022-09-23 儿童医疗中心有限公司 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件
MX375592B (es) 2014-04-25 2025-03-06 The Children´S Medical Center Corp Composiciones y su uso para tratar hemoglobinopatias.
US11572543B2 (en) 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
MX2018007987A (es) * 2015-12-28 2019-01-10 Novartis Ag Composiciones y metodos para el tratamiento de hemoglobinopatias.
EP3426781B1 (en) * 2016-03-07 2024-06-26 The United States of America, as represented by The Secretary, Department of Health and Human Services Micrornas and methods of their use
US11957713B2 (en) 2016-10-14 2024-04-16 Children's Medical Center Corporation Compositions and methods for treating diseases and disorders of the central nervous system
CA3050690A1 (en) 2017-01-17 2018-07-26 Dana-Farber Cancer Institute, Inc. Compositions and methods for diagnosing and treating peroxisomal diseases
AU2018210853B2 (en) 2017-01-17 2023-09-28 Children's Medical Center Corporation Compositions and methods for treating lysosomal storage diseases and disorders
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
JP7228523B2 (ja) * 2017-03-29 2023-02-24 ブルーバード バイオ， インコーポレイテッド ヘモグロビン異常症を治療するためのベクターおよび組成物
US11261441B2 (en) * 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
CN118581154A (zh) * 2017-06-02 2024-09-03 国家健康与医学研究院 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法
US10648728B2 (en) 2017-09-29 2020-05-12 Nxp Usa, Inc. Multifunctional radio frequency systems and methods for UV sterilization, air purification, and defrost operations
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
AU2018378479B2 (en) * 2017-12-05 2025-03-13 Vertex Pharmaceuticals Incorporated CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof
WO2019213273A1 (en) 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
WO2019213013A1 (en) 2018-05-02 2019-11-07 The Children's Medical Center Corporation Improved bcl11a micrornas for treating hemoglobinopathies
US20220160788A1 (en) * 2019-03-22 2022-05-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b-hemoglobinopathies
WO2021067613A1 (en) * 2019-10-01 2021-04-08 Children's Medical Center Corporation Compositions and methods for treating amyotrophic lateral sclerosis
KR20230023612A (ko) 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제
WO2022266366A1 (en) * 2021-06-16 2022-12-22 Duke University Compositions and methods for the prevention and treatment of hemoglobinopathies
CN114990164B (zh) * 2022-06-16 2023-12-05 北京大学 中介体复合物亚基抑制剂及其应用
AU2024299328A1 (en) 2023-07-21 2026-01-22 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
WO2025096404A1 (en) * 2023-11-01 2025-05-08 The Children's Hospital Of Philadelphia Compositions and methods for hemoglobin production
CN119746077B (zh) * 2025-01-16 2025-07-29 中国医学科学院基础医学研究所 ATR/Chk1信号通路抑制剂在促进红细胞脱核中的应用

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5635387A (en) 1990-04-23 1997-06-03 Cellpro, Inc. Methods and device for culturing human hematopoietic cells and their precursors
CA2131620A1 (en) 1992-03-20 1993-09-30 Louis C. Smith A dna transporter system and method of use
US5460964A (en) 1992-04-03 1995-10-24 Regents Of The University Of Minnesota Method for culturing hematopoietic cells
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US8101349B2 (en) * 1997-12-23 2012-01-24 Novartis Vaccines And Diagnostics, Inc. Gene products differentially expressed in cancerous cells and their methods of use II
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
CA2328404C (en) 1998-05-13 2007-07-24 Genetix Pharmaceuticals, Inc. Novel lentiviral packaging cells
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
DE60333487D1 (de) 2002-12-13 2010-09-02 Genetix Pharmaceuticals Inc Therapeutische retrovirus-vektoren für gentherapie
WO2005116204A1 (ja) 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
EP1969147B1 (en) * 2006-01-05 2014-07-30 The Ohio State University Research Foundation microRNA-based methods for the diagnosis of stomach cancer
US20080051431A1 (en) 2006-05-26 2008-02-28 Dominique Verhelle Methods and compositions using immunomodulatory compounds in combination therapy
KR101363928B1 (ko) 2007-06-11 2014-02-19 고쿠리츠다이가쿠호진 미에다이가쿠 특이적 유전자 발현 방법
GB0713183D0 (en) 2007-07-06 2007-08-15 King S College London Method
US8383604B2 (en) * 2008-09-15 2013-02-26 Children's Medical Center Corporation Modulation of BCL11A for treatment of hemoglobinopathies
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
WO2011072086A1 (en) 2009-12-08 2011-06-16 Hemaquest Pharmaceuticals, Inc. Methods and low dose regimens for treating red blood cell disorders
US8993532B2 (en) 2010-04-23 2015-03-31 Cold Spring Harbor Laboratory Structurally designed shRNAs
GB201012420D0 (en) * 2010-07-23 2010-09-08 Univ Erasmus Medical Ct Foetal heamoglobin inhibitor
WO2012073047A2 (en) 2010-12-03 2012-06-07 Genome Research Limited Compositions and methods
EP2648763A4 (en) * 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
KR20150029756A (ko) 2011-06-10 2015-03-18 블루버드 바이오, 인코포레이티드. 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터
ES2769270T3 (es) 2011-09-30 2020-06-25 Bluebird Bio Inc Compuestos para transducción viral mejorada
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CN109554350B (zh) 2012-11-27 2022-09-23 儿童医疗中心有限公司 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
WO2014093965A1 (en) 2012-12-14 2014-06-19 Case Western Reserve University Genomic rna packaging enhancer element
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
DK3492593T3 (da) 2013-11-13 2021-11-08 Childrens Medical Center Nukleasemedieret regulering af genekspression
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US20170049819A1 (en) 2014-04-25 2017-02-23 Bluebird Bio, Inc. Kappa/lambda chimeric antigen receptors
PL3134432T3 (pl) 2014-04-25 2020-10-19 Bluebird Bio, Inc. Promotor mnd chimeryczne receptory antygenowe
MX375592B (es) 2014-04-25 2025-03-06 The Children´S Medical Center Corp Composiciones y su uso para tratar hemoglobinopatias.
WO2015183667A1 (en) 2014-05-28 2015-12-03 The Regents Of The University Of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
HRP20191873T1 (hr) 2014-12-12 2020-01-24 Bluebird Bio, Inc. Kimerni antigenski receptori
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
CN107614680A (zh) 2015-05-14 2018-01-19 南加利福尼亚大学 利用重组核酸内切酶系统的最佳化基因编辑
WO2017040529A1 (en) 2015-08-31 2017-03-09 Bluebird Bio, Inc. Anti-sialyl tn chimeric antigen receptors
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
IL261002B2 (en) 2016-02-12 2024-07-01 Bluebird Bio Inc Vcn enhancer compositions and methods of using the same
WO2017173092A1 (en) 2016-03-31 2017-10-05 The Regents Of The University Of California Methods for genome editing in zygotes
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery

Also Published As

Publication number Publication date
KR20170005025A (ko) 2017-01-11
AU2015249381B2 (en) 2020-04-30
US20170218372A1 (en) 2017-08-03
RU2016145964A3 (es) 2019-04-23
EP3134130B1 (en) 2019-06-12
US11739329B2 (en) 2023-08-29
SG11201608482UA (en) 2016-11-29
JP2019129846A (ja) 2019-08-08
SA516380143B1 (ar) 2020-05-14
MX2016013832A (es) 2017-03-09
US11124794B2 (en) 2021-09-21
ES2744186T3 (es) 2020-02-24
EP3134130A2 (en) 2017-03-01
CA2946309A1 (en) 2015-10-29
RU2016145964A (ru) 2018-05-25
WO2015164750A2 (en) 2015-10-29
US20190316136A1 (en) 2019-10-17
SG10201809290SA (en) 2019-01-30
EP3134130A4 (en) 2018-04-04
AU2015249381A2 (en) 2018-03-29
IL248469B (en) 2020-04-30
DK3134130T3 (da) 2019-08-26
KR102390629B1 (ko) 2022-04-26
US10287588B2 (en) 2019-05-14
AU2015249381A1 (en) 2016-11-10
EP3598984B1 (en) 2024-04-10
JP6514717B2 (ja) 2019-05-15
CN106794260B (zh) 2021-02-23
JP2017513505A (ja) 2017-06-01
BR112016024565A2 (pt) 2018-01-23
US10662429B2 (en) 2020-05-26
CN106794260A (zh) 2017-05-31
EP3598984A1 (en) 2020-01-29
CA2946309C (en) 2021-11-09
NZ726792A (en) 2020-09-25
JP6713073B2 (ja) 2020-06-24
US20200370048A1 (en) 2020-11-26
US20220056453A1 (en) 2022-02-24
WO2015164750A3 (en) 2015-12-17
ZA201607540B (en) 2019-04-24
CN112852812A (zh) 2021-05-28
RU2707540C2 (ru) 2019-11-27
IL248469A0 (en) 2016-12-29

Similar Documents

Publication Publication Date Title
MX375592B (es) Composiciones y su uso para tratar hemoglobinopatias.
CL2018000194A1 (es) Compuestos de piridina
CO2018003974A2 (es) Compuestos de bifenilo
CR20150370A (es) Compuestos antivirales
UY34697A (es) Métodos para preparar análogos de nucleótidos
DOP2022000085A (es) Composiciones y métodos para inhibir la expresión del gen alas1
UY35812A (es) ?loci de maíz óptimos?.
UY34750A (es) ?compuestos para el tratamiento del hiv, composiciones,métodos de preparación, intermediarios y métodos terapéuticos?.
UY33917A (es) ?composiciones para recubrimiento que comprenden submicropartículas que comprenden carbonato de calcio, proceso para prepararlas, y uso de las submicropartículas?.
MX379044B (es) Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.
CO2017001884A2 (es) Polimorfos de selinexor
MX373949B (es) Dispositivo para la aptitud biologica del tejido fascia.
EA201491953A1 (ru) СОЕДИНЕНИЯ НА ОСНОВЕ МИКРОРНК И СПОСОБЫ МОДУЛИРОВАНИЯ АКТИВНОСТИ miR-21
EA201790069A1 (ru) Дифторметилникотиновые инданилкарбоксамиды
CL2016003277A1 (es) Métodos para preparar análogos de nucleótido sustituidos
CO7200270A2 (es) Azaindolcarboxamidas y azaindoltiocarboxamidas como insecticidas y acaricidas
MX392001B (es) Composiciones fertilizantes y métodos para hacer y utilizar las mismas.
MX2016003892A (es) Uso de dihidrooxindolilsulfonamidas sustituidas, o sus sales, para aumentar la tolerancia de plantas al estres.
UY34773A (es) Ácidos indanoiloxidihidrobenzofuranilacéticos como moduladores de la actividad de gpr40, composiciones que los contienen y sus usos
CO2017001528A2 (es) Compuestos novedosos de pirimidina sustituidos
CO2017001506A2 (es) Compuestos novedosos de pirimidina sustituidos
DOP2016000278A (es) Nuevos compuestos macrocíclicos
CO2017003004A2 (es) Nuevas triazolo[4,5-d]pirimidinas
BR112015008824A2 (pt) linhagens melhoradas de microalgas e processo das mesmas
CO2017006736A2 (es) Supresión de arni parental de gen hunchback para el control de las plagas de hemípteros

Legal Events

Date Code Title Description
FG Grant or registration