FIELD: biotechnology.
SUBSTANCE: invention relates to a method of obtaining a genetically modified population of cells enriched in CD34 containing a polynucleotide sequence encoding a polypeptide of the Fanconi anemia complementation group (FANC), or a functional variant or a fragment thereof functionally related to a promoter sequence active in eukaryotes, and CD34+ cells with a purity of <30%.
EFFECT: invention is effective for treating Fanconi anemia in a subject.
22 cl, 1 dwg, 3 tbl, 4 ex
Title | Year | Author | Number |
---|---|---|---|
GENE THERAPY IN PATIENTS WITH FANCONI ANEMIA | 2017 |
|
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RU2773358C2 |
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RU2747384C2 |
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RU2618864C2 |
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RU2735700C2 |
CONSTITUTIVELY ACTIVE CHIMERIC CYTOKINE RECEPTORS | 2020 |
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RU2824672C2 |
T-CELLULAR CANCER IMMUNOTHERAPY AIMED AT A1 CYCLINE | 2012 |
|
RU2632462C2 |
Authors
Dates
2023-12-19—Published
2019-04-11—Filed