Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 7

1.

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.

Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, Del Carro U, Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A.

Lancet. 2016 Jul 30;388(10043):476-87. doi: 10.1016/S0140-6736(16)30374-9. Epub 2016 Jun 8.

PMID:
27289174
2.

Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy.

Cesani M, Lorioli L, Grossi S, Amico G, Fumagalli F, Spiga I, Filocamo M, Biffi A.

Hum Mutat. 2016 Jan;37(1):16-27. doi: 10.1002/humu.22919. Epub 2015 Nov 4. Review.

PMID:
26462614
3.

Abnormalities of acid-base balance and predisposition to metabolic acidosis in Metachromatic Leukodystrophy patients.

Lorioli L, Cicalese MP, Silvani P, Assanelli A, Salvo I, Mandelli A, Fumagalli F, Fiori R, Ciceri F, Aiuti A, Sessa M, Roncarolo MG, Lanzani C, Biffi A.

Mol Genet Metab. 2015 May;115(1):48-52. doi: 10.1016/j.ymgme.2015.02.009. Epub 2015 Mar 6.

PMID:
25796965
4.

Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting.

Cesani M, Plati T, Lorioli L, Benedicenti F, Redaelli D, Dionisio F, Biasco L, Montini E, Naldini L, Biffi A.

Gene Ther. 2015 Jun;22(6):496-502. doi: 10.1038/gt.2015.10. Epub 2015 Mar 12.

PMID:
25762283
5.

Critical issues for the proper diagnosis of Metachromatic Leukodystrophy.

Lorioli L, Cesani M, Regis S, Morena F, Grossi S, Fumagalli F, Acquati S, Redaelli D, Pini A, Sessa M, Martino S, Filocamo M, Biffi A.

Gene. 2014 Mar 10;537(2):348-51. doi: 10.1016/j.gene.2013.11.062. Epub 2013 Dec 12.

PMID:
24334127
6.

Metallothioneins as dynamic markers for brain disease in lysosomal disorders.

Cesani M, Cavalca E, Macco R, Leoncini G, Terreni MR, Lorioli L, Furlan R, Comi G, Doglioni C, Zacchetti D, Sessa M, Scherzer CR, Biffi A.

Ann Neurol. 2014 Jan;75(1):127-37. doi: 10.1002/ana.24053.

7.

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L.

Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.

Supplemental Content

Loading ...
Support Center