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Neuromuscular Disorders : NMD|September 11, 2002
Myogenic stem cells from the bone marrow: a therapeutic alternative for muscular dystrophy?Giuliana Ferrari, Fulvio Mavilio
Blood Cells, Molecules & Diseases|January 17, 2018
Gene therapy and gene editing strategies for hemoglobinopathiesMaria Rosa Lidonnici, Giuliana Ferrari
Hematology/Oncology Clinics of North America|September 13, 2017
Gene Therapy Approaches to HemoglobinopathiesGiuliana Ferrari, Marina Cavazzana, Fulvio Mavilio
Human Gene Therapy|September 7, 2023
Gene Therapy for HemoglobinopathiesMaria Rosa Lidonnici, Samantha Scaramuzza, Giuliana Ferrari
Nature Reviews. Genetics|December 11, 2020
Gene therapy using haematopoietic stem and progenitor cellsGiuliana Ferrari, Adrian J Thrasher, Alessandro Aiuti
Cell Reports. Medicine|November 18, 2025
Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow nicheAnnamaria Aprile, Maria Rosa Lidonnici, Giuliana Ferrari
Pharmaceuticals (Basel, Switzerland)|May 28, 2022
Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell DiseaseAnnamaria Aprile, Silvia Sighinolfi, Laura Raggi, et al.
Stem Cells (Dayton, Ohio)|March 25, 2010
Bone marrow-derived hematopoietic cells undergo myogenic differentiation following a Pax-7 independent pathwayAlexandros Xynos, Paola Corbella, Nathalie Belmonte, et al.
Journal of Virology|March 22, 2002
Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacementFrancesco Lotti, Emilio Menguzzato, Claudia Rossi, et al.
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