Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Marktel S, Scaramuzza S, Cicalese MP, Giglio F, Galimberti S, Lidonnici MR, Calbi V, Assanelli A, Bernardo ME, Rossi C, Calabria A, Milani R, Gattillo S, Benedicenti F, Spinozzi G, Aprile A, Bergami A, Casiraghi M, Consiglieri G, Masera N, D'Angelo E, Mirra N, Origa R, Tartaglione I, Perrotta S, Winter R, Coppola M, Viarengo G, Santoleri L, Graziadei G, Gabaldo M, Valsecchi MG, Montini E, Naldini L, Cappellini MD, Ciceri F, Aiuti A, Ferrari G.
Marktel S, et al. Among authors: calbi v.
Nat Med. 2019 Feb;25(2):234-241. doi: 10.1038/s41591-018-0301-6. Epub 2019 Jan 21.
Nat Med. 2019.
PMID: 30664781
Clinical Trial.