Lentiviral Haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
Lancet 399:372-383, Fumagalli, F.,et al, 2022
Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
NEJM 385:1929-1940, Gentner, B.,et al, 2021
Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinsons Disease
NEJM 382:1926-1932, Schweitzer, J.S.,et al, 2020
Long-Term Outcomes after Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis
JAMA Neurol 74:459-469,392, Muraro, P.A.,et al, 2017
Myasthenia Gravis Treated with Autologous Hematopoietic Stem Cell Transplantation
JAMA Neurol 73:652-658,624, Bryant, A.,et al, 2016
Long-Term Clinical Outcomes After Fetal Cell Transplantation in Parkinson Disease Implications for the Future of Cell Therapy
JAMA 311:617-618, Bega, D. & Krainc, D., 2014
Stem Cell Transplantation for Ischemic Stroke
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