BR112021012240A2 - Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich - Google Patents

Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich

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Publication number
BR112021012240A2
BR112021012240A2 BR112021012240A BR112021012240A BR112021012240A2 BR 112021012240 A2 BR112021012240 A2 BR 112021012240A2 BR 112021012240 A BR112021012240 A BR 112021012240A BR 112021012240 A BR112021012240 A BR 112021012240A BR 112021012240 A2 BR112021012240 A2 BR 112021012240A2
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BR
Brazil
Prior art keywords
wiskott
aldrich syndrome
stem cell
gene therapy
hematopoietic stem
Prior art date
Application number
BR112021012240A
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English (en)
Inventor
Chao-Guang Cheng
Chi-Lin Lee
Pierre Vonarburg Cédric
Ming Yan
Jhan Azar Walid
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Csl Behring Llc
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Publication date
Application filed by Csl Behring Llc filed Critical Csl Behring Llc
Publication of BR112021012240A2 publication Critical patent/BR112021012240A2/pt

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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
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    • 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
    • C12N15/1137—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 enzymes
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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • 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
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    • C12N2740/00011—Details
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    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Abstract

terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich. a presente invenção refere-se a vetores de expressão compreendendo pelo menos duas sequências de ácido nucleico, a saber, uma sequência de ácido nucleico codificando um arni anti-hprt e uma sequência de ácido nucleico codificando uma proteína da síndrome de wiskott-aldrich. em algumas modalidades, o vetor de expressão é um vetor lentiviral auto-inativador. em algumas modalidades, a proteína da síndrome de wiskott-aldrich é utilizada para aliviar as patologias associadas à síndrome de wiskott-aldrich.
BR112021012240A 2018-12-23 2019-12-23 Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich BR112021012240A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862784508P 2018-12-23 2018-12-23
PCT/US2019/068233 WO2020139796A1 (en) 2018-12-23 2019-12-23 Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome

Publications (1)

Publication Number Publication Date
BR112021012240A2 true BR112021012240A2 (pt) 2022-01-18

Family

ID=69326699

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021012240A BR112021012240A2 (pt) 2018-12-23 2019-12-23 Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich

Country Status (8)

Country Link
US (1) US20210316013A1 (pt)
EP (1) EP3897745A1 (pt)
JP (1) JP2022514955A (pt)
KR (1) KR20210118402A (pt)
CN (1) CN113518825A (pt)
AU (1) AU2019417697A1 (pt)
BR (1) BR112021012240A2 (pt)
WO (1) WO2020139796A1 (pt)

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Also Published As

Publication number Publication date
AU2019417697A1 (en) 2021-07-08
EP3897745A1 (en) 2021-10-27
WO2020139796A1 (en) 2020-07-02
KR20210118402A (ko) 2021-09-30
US20210316013A1 (en) 2021-10-14
CN113518825A (zh) 2021-10-19
JP2022514955A (ja) 2022-02-16

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