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

Cell Therapy, Immunology, Blood Cell +17

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

Cell Therapy, Lentiviral Gene Therapy, Immunology +17

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 · 772 citations · Full text

Genetic Diseases, Neurodegenerative Diseases, Miracle Foundation +13

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Yuxuan Wu, Jing Zeng, Benjamin P. Roscoe et al. · Nature Medicine · 2019 · 504 citations

Induced Pluripotent Stem Cell, Genome Surgery, Medicine +7

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 · 496 citations

Ad-hoc Analysis, Cell Therapy, Bone Marrow Failure +8