{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,4]],"date-time":"2026-11-04T03:55:29Z","timestamp":1793764529110,"version":"4.6.1"},"reference-count":54,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"745","content-domain":{"domain":["www.science.org"],"crossmark-restriction":true},"short-container-title":["Sci. Transl. Med."],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:p>Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive skeletal dysplasia that is not fully addressed by allogeneic hematopoietic stem cell transplantation (HSCT). Autologous hematopoietic stem progenitor cell\u2013gene therapy (HSPC-GT) provides superior metabolic correction in patients with MPSIH compared with HSCT; however, its ability to affect skeletal manifestations is unknown. Eight patients with MPSIH (mean age at treatment: 1.9 years) received lentiviral-based HSPC-GT in a phase 1\/2 clinical trial (NCT03488394). Clinical (growth, measures of kyphosis and genu velgum), functional (motor function, joint range of motion), and radiological [acetabular index (AI), migration percentage (MP) in hip x-rays and MRIs and spine MRI score] parameters of skeletal dysplasia were evaluated at baseline and multiple time points up to 4 years after treatment. Specific skeletal measures were retrospectively compared with an external cohort of HSCT-treated patients. At a median follow-up of 3.78 years after HSPC-GT, all patients treated with HSPC-GT exhibited longitudinal growth within WHO reference ranges and a median height gain greater than that observed in patients treated with HSCT after 3-year follow-up. Patients receiving HSPC-GT experienced complete and earlier normalization of joint mobility compared with patients treated with HSCT. Mean AI and MP showed progressive decreases after HSPC-GT, suggesting a reduction in acetabular dysplasia. Typical spine alterations measured through a spine MRI score stabilized after HSPC-GT. Clinical, functional, and radiological measures suggested an early beneficial effect of HSPC-GT on MPSIH-typical skeletal features. Longer follow-up is needed to draw definitive conclusions on HSPC-GT\u2019s impact on MPSIH skeletal dysplasia.<\/jats:p>","DOI":"10.1126\/scitranslmed.adi8214","type":"journal-article","created":{"date-parts":[[2024,5,1]],"date-time":"2024-05-01T17:59:05Z","timestamp":1714586345000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":26,"title":["Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome"],"prefix":"10.1126","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1750-5745","authenticated-orcid":true,"given":"Giulia","family":"Consiglieri","sequence":"first","affiliation":[{"name":"Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy."},{"name":"San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy."},{"name":"Department of Systems Medicine, University of Rome Tor 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