L
Luigi Naldini
Researcher at Vita-Salute San Raffaele University
Publications - 370
Citations - 59320
Luigi Naldini is an academic researcher from Vita-Salute San Raffaele University. The author has contributed to research in topics: Genetic enhancement & Viral vector. The author has an hindex of 108, co-authored 345 publications receiving 55080 citations. Previous affiliations of Luigi Naldini include Università telematica San Raffaele & Sangamo BioSciences.
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Journal ArticleDOI
A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A
Hideto Matsui,Carol Hegadorn,Margareth C. Ozelo,Erin Burnett,Angie Tuttle,Andrea Labelle,Paul B. McCray,Luigi Naldini,Brian D. Brown,Christine Hough,David Lillicrap +10 more
TL;DR: A modified lentiviral delivery strategy that facilitates liver-restricted transgene expression and prevents off-target expression in hematopoietic cells by incorporating microRNA (miRNA) target sequences was evaluated and was insufficient to prevent an anti-FVIII immune response in treated hemophilia A mice.
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SUMF1 Enhances Sulfatase Activities in Vivo in Five Sulfatase Deficiencies
Alessandro Fraldi,Alessandra Biffi,Alessia Lombardi,Ilaria Visigalli,Stefano Pepe,Carmine Settembre,Edoardo Nusco,Alberto Auricchio,Luigi Naldini,Andrea Ballabio,Maria Pia Cosma +10 more
TL;DR: It is demonstrated that co-delivery of SUMF1 may enhance the efficacy of gene therapy in several sulfatase deficiencies, and resulted in an improved clearance of the intracellular GAG or sulfolipid accumulation.
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Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.
Carmela Di Domenico,Guglielmo R. D. Villani,Daniele Di Napoli,Enrico Gonzalez Y Reyero,Angelo Lombardo,Luigi Naldini,Paola Di Natale +6 more
TL;DR: The results show the promising potential and the limitations of lentiviral-IDUA vector-mediated gene therapy in an in vivo model and suggest that the transgene-specific immune response interfered with long-term therapeutic correction and led to clearance of transduced cells.
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Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction
TL;DR: It is demonstrated that antiviral responses ultimately mediated by proteasomes strongly limit the efficiency of HSC transduction by LVs and establish improved conditions for HSC-based gene therapy.
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Targeted gene therapy and cell reprogramming in Fanconi anemia.
Paula Río,Rocío Baños,Angelo Lombardo,Oscar Quintana-Bustamante,Lara Álvarez,Zita Garate,Pietro Genovese,Elena Almarza,Antonio Valeri,Begoña Díez,Susana Navarro,Yaima Torres,Juan P. Trujillo,Juan P. Trujillo,Rodolfo Murillas,José C. Segovia,Enrique Samper,Jordi Surrallés,Philip D. Gregory,Michael C. Holmes,Luigi Naldini,Juan A. Bueren +21 more
TL;DR: The results demonstrate for the first time the feasibility of correcting the phenotype of a DNA repair deficiency syndrome using gene‐targeting and cell reprogramming strategies.