MX379044B - Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal. - Google Patents

Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.

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Publication number
MX379044B
MX379044B MX2018015033A MX2018015033A MX379044B MX 379044 B MX379044 B MX 379044B MX 2018015033 A MX2018015033 A MX 2018015033A MX 2018015033 A MX2018015033 A MX 2018015033A MX 379044 B MX379044 B MX 379044B
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Mexico
Prior art keywords
reinduction
fetal hemoglobin
bcl11a
regulatory elements
distal regulatory
Prior art date
Application number
MX2018015033A
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English (en)
Inventor
Daniel E Bauer
Jian Xu
Stuart H Orkin
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Childrens Medical Center
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Publication date
Application filed by Childrens Medical Center filed Critical Childrens Medical Center
Publication of MX379044B publication Critical patent/MX379044B/es

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    • 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
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • Biomedical Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporcionan en la presente métodos y composiciones para incrementar los niveles de hemoglobina fetal en una célula al interrumpir la expresión de BCL11A en el nivel genómico. También se proporcionan en la presente métodos y composiciones relacionados con el tratamiento de hemoglobinopatías mediante la reinducción de los niveles de hemoglobina fetal.
MX2018015033A 2012-11-27 2013-11-27 Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal. MX379044B (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261730323P 2012-11-27 2012-11-27
US201261730369P 2012-11-27 2012-11-27
US201361776144P 2013-03-11 2013-03-11
US201361889174P 2013-10-10 2013-10-10
PCT/US2013/072236 WO2014085593A1 (en) 2012-11-27 2013-11-27 Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction

Publications (1)

Publication Number Publication Date
MX379044B true MX379044B (es) 2025-03-10

Family

ID=50828461

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018015033A MX379044B (es) 2012-11-27 2013-11-27 Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.
MX2015006528A MX361412B (es) 2012-11-27 2013-11-27 Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2015006528A MX361412B (es) 2012-11-27 2013-11-27 Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal.

Country Status (17)

Country Link
US (4) US9822355B2 (es)
EP (2) EP2925864B1 (es)
JP (2) JP6542124B2 (es)
KR (1) KR102240555B1 (es)
CN (2) CN104955943B (es)
AU (3) AU2013352156B2 (es)
BR (1) BR112015011995B1 (es)
CA (1) CA2892860C (es)
DK (2) DK3502240T3 (es)
ES (2) ES2708948T3 (es)
IL (1) IL238949B (es)
MX (2) MX379044B (es)
PT (2) PT2925864T (es)
SA (1) SA515360489B1 (es)
SG (3) SG10201704008RA (es)
WO (1) WO2014085593A1 (es)
ZA (1) ZA201504004B (es)

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