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MX2020009487A - Sistemas y metodos para el tratamiento de hemoglobinopatias. - Google Patents

Sistemas y metodos para el tratamiento de hemoglobinopatias.

Info

Publication number
MX2020009487A
MX2020009487A MX2020009487A MX2020009487A MX2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A
Authority
MX
Mexico
Prior art keywords
methods
systems
hemoglobinopathies
treatment
portions
Prior art date
Application number
MX2020009487A
Other languages
English (en)
Inventor
Jennifer Leah Gori
De Lamothe-Dreuzy Edouard Aupepin
Jack Heath
John Anthony Zuris
Kaihsin Chang
Original Assignee
Editas Medicine Inc
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 Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of MX2020009487A publication Critical patent/MX2020009487A/es

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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • 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
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • 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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Cell Biology (AREA)
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  • Developmental Biology & Embryology (AREA)
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Abstract

Se proporcionan sistemas de edición genómica, ARN guía y métodos mediados por CRISPR para alterar porciones de los locus de HBG1 y HBG2, porciones del potenciador específico de eritroides del gen BCL11A, o una combinación de los mismos, en las células, y para aumentar la expresión de hemoglobina fetal.
MX2020009487A 2018-03-14 2019-03-14 Sistemas y metodos para el tratamiento de hemoglobinopatias. MX2020009487A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862643168P 2018-03-14 2018-03-14
US201862767488P 2018-11-14 2018-11-14
US201862773073P 2018-11-29 2018-11-29
PCT/US2019/022374 WO2019178426A1 (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
MX2020009487A true MX2020009487A (es) 2020-12-10

Family

ID=65952168

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020009487A MX2020009487A (es) 2018-03-14 2019-03-14 Sistemas y metodos para el tratamiento de hemoglobinopatias.

Country Status (12)

Country Link
US (2) US12031132B2 (es)
EP (1) EP3765614A1 (es)
JP (2) JP7706888B2 (es)
KR (2) KR20260006713A (es)
CN (1) CN112020558A (es)
AU (2) AU2019236209B2 (es)
BR (1) BR112020018364A2 (es)
CA (1) CA3093702A1 (es)
IL (2) IL277234B1 (es)
MX (1) MX2020009487A (es)
SG (1) SG11202008956XA (es)
WO (1) WO2019178426A1 (es)

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