Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
Bernhard GentnerFrancesca TucciStefania GalimbertiFrancesca FumagalliMaurizio De PellegrinPaolo SilvaniChiara CamesascaSilvia PontesilliSilvia DarinFrancesca CiottiMarina SarzanaGiulia ConsiglieriChiara FilisettiGiulia ForniLaura PasseriniDaniela TomasoniDaniela CesanaAndrea CalabriaGiulio SpinozziMaria-Pia CicaleseValeria CalbiMaddalena MigliavaccaFederica BarzaghiFrancesca FerruaVera GalloSimona MigliettaErika ZonariPatali S CherukuClaudia ForniMarcella FacchiniAmbra CortiMichela GabaldoStefano ZancanSerena GasperiniAttilio RovelliJaap-Jan BoelensSimon A JonesRobert WynnCristina BaldoliEugenio MontiniSilvia GregoriFabio CiceriMaria G ValsecchiGiancarlo la MarcaRossella PariniLuigi NaldiniAlessandro AiutiMaria-Ester Bernardonull nullPublished in: The New England journal of medicine (2021)
The delivery of HSPC gene therapy in patients with MPSIH resulted in extensive metabolic correction in peripheral tissues and the central nervous system. (Funded by Fondazione Telethon and others; ClinicalTrials.gov number, NCT03488394; EudraCT number, 2017-002430-23.).