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Herzog, Roland W.; Pierce, Glenn F.
Molecular therapy, 11/2019, Letnik: 27, Številka: 11Journal Article
Even the coding sequence of the shorter (but fully biologically active) B domain-deleted version is at the packaging limit of AAV, necessitating use of small promoter elements. ...FVIII is more difficult for cells to secrete. ...large vector doses have been employed to reach therapeutic levels. ...despite successes, there are still important known unknowns in liver-directed gene therapy.
