Alessandra Biffi

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Alessandra Biffi, Eugenio Montini, Laura Lorioli et al. · Science · 2013 · 1.2K citations · Full text

Life Sciences, Genetics, Hematology +16

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Alessandro Aiuti, Luca Biasco, Samantha Scaramuzza et al. · Science · 2013 · 1K citations · Full text

Medicine, Cell Biology, Hematology +16

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

Jin‐Kuk Kim, Chunguang Hu, Christelle Moufawad El Achkar et al. · New England Journal of Medicine · 2019 · 805 citations · Full text

Life Sciences, Molecular Medicine, Neurology +17

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Yuxuan Wu, Jing Zeng, Benjamin P. Roscoe et al. · Nature Medicine · 2019 · 518 citations

Life Sciences, Genetics, Hematology +14

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Maria Sessa, Laura Lorioli, Francesca Fumagalli et al. · The Lancet · 2016 · 507 citations

Medicine, Hematology, Molecular Medicine +13