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pro vyhledávání: '"Stephanie Patriarca"'
Autor:
David R. Williams, Olivier Negre, Erica B. Esrick, Sarah Nikiforow, Christian Brendel, Shanna Richard, Daniela Abriss, Jerome Ritz, Myriam Armant, Maureen Achebe, John P. Manis, Stephen Braunewell, Lauryn Christiansen, Matthew M. Heeney, Marioara F. Ciuculescu, Helene Negre, Heather Daley, Leslie Lehmann, Alessandra Biffi, Renee Maxwell, Stephanie Patriarca, Brenda Mackinnon, Colleen Dansereau
Publikováno v:
Blood. 132:1023-1023
Autologous gene therapy (GT) for beta-hemoglobinopathies has demonstrated encouraging early safety and efficacy using addition of a sickling-resistant globin gene to stem cells. Another therapeutic strategy for sickle cell disease (SCD) is erythroid-