Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
2017; American Association for the Advancement of Science; Volume: 9; Issue: 411 Linguagem: Inglês
10.1126/scitranslmed.aan0820
ISSN1946-6242
AutoresGiulia Schiroli, Samuele Ferrari, Anthony Conway, Aurélien Jacob, Valentina Capo, Luisa Albano, Tiziana Plati, Maria Carmina Castiello, Francesca Sanvito, Andrew R. Gennery, Chiara Bovolenta, Rahul Palchaudhuri, David T. Scadden, Michael C. Holmes, Anna Villa, Giovanni Sitia, Angelo Lombardo, Pietro Genovese, Luigi Naldini,
Tópico(s)CAR-T cell therapy research
ResumoPreclinical studies establish the conditions for safe and effective correction of SCID-X1 by targeted gene editing of hematopoietic stem cells.
Referência(s)