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L Naldini

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Articles 68
Citations 8477
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Recent Articles
1.
Santoni de Sio F, Passerini L, Valente M, Russo F, Naldini L, Roncarolo M, et al.
Sci Rep . 2017 Nov; 7(1):15820. PMID: 29150659
FOXP3 is the transcription factor ruling regulatory T cell function and maintenance of peripheral immune tolerance, and mutations in its coding gene causes IPEX autoimmune syndrome. FOXP3 is also a...
2.
Cesani M, Plati T, Lorioli L, Benedicenti F, Redaelli D, Dionisio F, et al.
Gene Ther . 2015 Mar; 22(6):496-502. PMID: 25762283
Gene therapy using viral vectors that stably integrate into ex vivo cultured cells holds great promises for the treatment of monogenic diseases as well as cancer. However, carry-over of infectious...
3.
Gentner B, Naldini L
Tissue Antigens . 2012 Oct; 80(5):393-403. PMID: 23020307
RNA interference (RNAi) has been a landmark discovery in science. A typical application is to knock down the expression of endogenous genes by delivering small interfering RNA (siRNA) into cells...
4.
Rizzi R, Di Pasquale E, Portararo P, Papait R, Cattaneo P, Latronico M, et al.
Cell Death Differ . 2012 Jan; 19(7):1162-74. PMID: 22261617
Adult mammalian cells can be reprogrammed to a pluripotent state by forcing the expression of a few embryonic transcription factors. The resulting induced pluripotent stem (iPS) cells can differentiate into...
5.
Pasi C, Dereli-Oz A, Negrini S, Friedli M, Fragola G, Lombardo A, et al.
Cell Death Differ . 2011 Feb; 18(5):745-53. PMID: 21311564
The ability to reprogram adult cells into stem cells has raised hopes for novel therapies for many human diseases. Typical stem cell reprogramming protocols involve expression of a small number...
6.
Capotondo A, Cesani M, Pepe S, Fasano S, Gregori S, Tononi L, et al.
Hum Gene Ther . 2007 Sep; 18(9):821-36. PMID: 17845130
Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) gene transfer, highlighted a requirement for high levels of...
7.
Di Domenico C, Di Napoli D, Gonzalez Y Reyero E, Lombardo A, Naldini L, Di Natale P
Hum Gene Ther . 2006 Oct; 17(11):1112-21. PMID: 17044753
Mucopolysaccharidosis type I (MPS I) due to deficient alpha-L-iduronidase (IDUA) activity results in the accumulation of glycosaminoglycans (GAGs) in many of the cells of affected patients. Stable gene replacement by...
8.
Dolcetta D, Perani L, Givogri M, Galbiati F, Amadio S, Del Carro U, et al.
J Gene Med . 2006 May; 8(8):962-71. PMID: 16732552
Background: Demyelination in globoid cell leukodystrophy (GLD) is due to a deficiency of galactocerebrosidase (GALC) activity. Up to now, in vivo brain viral gene transfer of GALC showed modest impact...
9.
Serafini M, Naldini L, Introna M
Virology . 2004 Jul; 325(2):413-24. PMID: 15246279
Lentiviral vectors are attractive tools to transduce dividing and nondividing cells. Human tonsillar B lymphocytes have been purified and induced to proliferate by the addition of anti-CD40 + IL-4 or...
10.
Bonci D, Cittadini A, Latronico M, Borello U, Aycock J, Drusco A, et al.
Gene Ther . 2003 Apr; 10(8):630-6. PMID: 12692591
Efficient gene transduction in cardiomyocytes is a task that can be accomplished only by viral vectors. Up to now, the most commonly used vectors for this purpose have been adenoviral-derived...