Publication & Citation Trends
Publications
0 total
Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease
Cited by 0
Semantic Scholar
A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB OA
Cited by 2
Semantic Scholar
Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T‐cells for the treatment of Hyper IgM1 OA
Cited by 3
Semantic Scholar
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates OA
Cited by 33
Semantic Scholar
Conditioning Regimens in Long-Term Pre-Clinical Studies to Support Development of Ex Vivo Gene Therapy: Review of Nonproliferative and Proliferative Changes
Cited by 8
Semantic Scholar
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy OA
Cited by 19
Semantic Scholar
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates OA
Cited by 103
Semantic Scholar
Research Topics
Virus-based gene therapy research
(12)
Lysosomal Storage Disorders Research
(11)
CRISPR and Genetic Engineering
(9)
CAR-T cell therapy research
(7)
Proteoglycans and glycosaminoglycans research
(4)
Affiliations
Vita-Salute San Raffaele University
IRCCS Ospedale San Raffaele
The San Raffaele Telethon Institute for Gene Therapy
Istituti di Ricovero e Cura a Carattere Scientifico
Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele