FIELD: biotechnology.
SUBSTANCE: following was proposed: A CD34+ cell for the treatment of Fanconi anemia containing an expression cassette containing a polynucleotide sequence. A pharmaceutical composition for the treatment of Fanconi anemia containing a pharmaceutically acceptable excipient and an effective amount of CD34+ cells, as well as methods for treating Fanconi anemia in a subject in need thereof.
EFFECT: invention makes it possible to use it for the treatment of Fanconi anemia in patients.
15 cl, 44 dwg, 2 tbl, 10 ex
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