Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
Giulia Schiroli(Vita-Salute San Raffaele University), Samuele Ferrari(Vita-Salute San Raffaele University), Anthony Conway(Sangamo BioSciences (United States)), Aurélien Jacob(The San Raffaele Telethon Institute for Gene Therapy), Valentina Capo(The San Raffaele Telethon Institute for Gene Therapy), Luisa Albano(The San Raffaele Telethon Institute for Gene Therapy), Tiziana Plati(The San Raffaele Telethon Institute for Gene Therapy), Maria Carmina Castiello(The San Raffaele Telethon Institute for Gene Therapy), Francesca Sanvito(San Raffaele University of Rome), Andrew R. Gennery(Newcastle University), Chiara Bovolenta(MolMed (Italy)), Rahul Palchaudhuri(Intellia Therapeutics (United States)), David T. Scadden(Harvard Stem Cell Institute), Michael C. Holmes(Sangamo BioSciences (United States)), Anna Villa(Institute of Genetic and Biomedical Research), Giovanni Sitia(San Raffaele University of Rome), Angelo Lombardo(Vita-Salute San Raffaele University), Pietro Genovese(The San Raffaele Telethon Institute for Gene Therapy), Luigi Naldini(Vita-Salute San Raffaele University)
Cited by 215
Abstract
editing in long-term repopulating cells predicted to safely rescue the disease, using clinically relevant HSPC sources and highly specific zinc finger nucleases or CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 (CRISPR-associated protein 9). Overall, our work establishes the rationale and guiding principles for clinical translation of SCID-X1 gene editing and provides a framework for developing gene correction for other diseases.
Related Papers
No related papers found
Powered by citation graph analysis