Update on clinical ex vivo hematopoietic stem cell gene therapy for inherited monogenic diseases
F Tucci, S Scaramuzza, A Aiuti, A Mortellaro - Molecular Therapy, 2021 - cell.com
Gene transfer into autologous hematopoietic stem progenitor cells (HSPCs) has the
potential to cure monogenic inherited disorders caused by an altered development and/or …
potential to cure monogenic inherited disorders caused by an altered development and/or …
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial …
Background Effective treatment for metachromatic leukodystrophy (MLD) remains a
substantial unmet medical need. In this study we investigated the safety and efficacy of …
substantial unmet medical need. In this study we investigated the safety and efficacy of …