191 related articles for article (PubMed ID: 34654893)
21. Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.
Colle MA; Piguet F; Bertrand L; Raoul S; Bieche I; Dubreil L; Sloothaak D; Bouquet C; Moullier P; Aubourg P; Cherel Y; Cartier N; Sevin C
Hum Mol Genet; 2010 Jan; 19(1):147-58. PubMed ID: 19837699
[TBL] [Abstract][Full Text] [Related]
22. Ex vivo cell-mediated gene therapy for metachromatic leukodystrophy using neurospheres.
Kawabata K; Migita M; Mochizuki H; Miyake K; Igarashi T; Fukunaga Y; Shimada T
Brain Res; 2006 Jun; 1094(1):13-23. PubMed ID: 16729983
[TBL] [Abstract][Full Text] [Related]
23. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.
Biffi A; Capotondo A; Fasano S; del Carro U; Marchesini S; Azuma H; Malaguti MC; Amadio S; Brambilla R; Grompe M; Bordignon C; Quattrini A; Naldini L
J Clin Invest; 2006 Nov; 116(11):3070-82. PubMed ID: 17080200
[TBL] [Abstract][Full Text] [Related]
24. 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; 341(6148):1233158. PubMed ID: 23845948
[TBL] [Abstract][Full Text] [Related]
25. Olaparib Attenuates Demyelination and Neuroinflammation in an Organotypic Slice Culture Model of Metachromatic Leukodystrophy.
Mekhaeil M; Conroy MJ; Dev KK
Neurotherapeutics; 2023 Sep; 20(5):1347-1368. PubMed ID: 37525026
[TBL] [Abstract][Full Text] [Related]
26. AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy.
Kurai T; Hisayasu S; Kitagawa R; Migita M; Suzuki H; Hirai Y; Shimada T
Mol Ther; 2007 Jan; 15(1):38-43. PubMed ID: 17164773
[TBL] [Abstract][Full Text] [Related]
27. Gene therapy in metachromatic leukodystrophy.
Sevin C; Cartier-Lacave N; Aubourg P
Int J Clin Pharmacol Ther; 2009; 47 Suppl 1():S128-31. PubMed ID: 20040324
[TBL] [Abstract][Full Text] [Related]
28. Enzymatic characterization of novel arylsulfatase A variants using human arylsulfatase A-deficient immortalized mesenchymal stromal cells.
Böhringer J; Santer R; Schumacher N; Gieseke F; Cornils K; Pechan M; Kustermann-Kuhn B; Handgretinger R; Schöls L; Harzer K; Krägeloh-Mann I; Müller I
Hum Mutat; 2017 Nov; 38(11):1511-1520. PubMed ID: 28762252
[TBL] [Abstract][Full Text] [Related]
29. Chinese Cases of Metachromatic Leukodystrophy with the Novel Missense Mutations in ARSA Gene.
Wu S; Hou M; Zhang Y; Song J; Guo Y; Liu P; Liu Y; Yi L; Pan X; We W; Chen Z
J Mol Neurosci; 2021 Feb; 71(2):245-251. PubMed ID: 32617873
[TBL] [Abstract][Full Text] [Related]
30. Coexpression of formylglycine-generating enzyme is essential for synthesis and secretion of functional arylsulfatase A in a mouse model of metachromatic leukodystrophy.
Takakusaki Y; Hisayasu S; Hirai Y; Shimada T
Hum Gene Ther; 2005 Aug; 16(8):929-36. PubMed ID: 16076251
[TBL] [Abstract][Full Text] [Related]
31. Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment.
Wu THY; Brown HA; Church HJ; Kershaw CJ; Hutton R; Egerton C; Cooper J; Tylee K; Cohen RN; Gokhale D; Ram D; Morton G; Henderson M; Bigger BW; Jones SA
Mol Genet Metab; 2024 May; 142(1):108349. PubMed ID: 38458124
[TBL] [Abstract][Full Text] [Related]
32. Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.
Capotondo A; Cesani M; Pepe S; Fasano S; Gregori S; Tononi L; Venneri MA; Brambilla R; Quattrini A; Ballabio A; Cosma MP; Naldini L; Biffi A
Hum Gene Ther; 2007 Sep; 18(9):821-36. PubMed ID: 17845130
[TBL] [Abstract][Full Text] [Related]
33. Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery.
Luca T; Givogri MI; Perani L; Galbiati F; Follenzi A; Naldini L; Bongarzone ER
Mol Ther; 2005 Oct; 12(4):669-79. PubMed ID: 16087406
[TBL] [Abstract][Full Text] [Related]
34. Metachromatic Leukodystrophy (MLD): a Pakistani Family with Novel ARSA Gene Mutation.
Shahzad MA; Khaliq S; Amar A; Mahmood S
J Mol Neurosci; 2017 Sep; 63(1):84-90. PubMed ID: 28799099
[TBL] [Abstract][Full Text] [Related]
35. Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models.
Lattanzi A; Neri M; Maderna C; di Girolamo I; Martino S; Orlacchio A; Amendola M; Naldini L; Gritti A
Hum Mol Genet; 2010 Jun; 19(11):2208-27. PubMed ID: 20203170
[TBL] [Abstract][Full Text] [Related]
36. Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: cellular alterations and topographic distribution.
Wittke D; Hartmann D; Gieselmann V; Lüllmann-Rauch R
Acta Neuropathol; 2004 Oct; 108(4):261-71. PubMed ID: 15322834
[TBL] [Abstract][Full Text] [Related]
37. Evolutionary redesign of the lysosomal enzyme arylsulfatase A increases efficacy of enzyme replacement therapy for metachromatic leukodystrophy.
Simonis H; Yaghootfam C; Sylvester M; Gieselmann V; Matzner U
Hum Mol Genet; 2019 Jun; 28(11):1810-1821. PubMed ID: 30657900
[TBL] [Abstract][Full Text] [Related]
38. Deletion of fatty acid amide hydrolase reduces lyso-sulfatide levels but exacerbates metachromatic leukodystrophy in mice.
Yaghootfam C; Gehrig B; Sylvester M; Gieselmann V; Matzner U
J Biol Chem; 2021 Sep; 297(3):101064. PubMed ID: 34375644
[TBL] [Abstract][Full Text] [Related]
39. Multipotential neural precursors transplanted into the metachromatic leukodystrophy brain fail to generate oligodendrocytes but contribute to limit brain dysfunction.
Givogri MI; Bottai D; Zhu HL; Fasano S; Lamorte G; Brambilla R; Vescovi A; Wrabetz L; Bongarzone E
Dev Neurosci; 2008; 30(5):340-57. PubMed ID: 18667806
[TBL] [Abstract][Full Text] [Related]
40. Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts.
Consiglio A; Martino S; Dolcetta D; Cusella G; Conese M; Marchesini S; Benaglia G; Wrabetz L; Orlacchio A; Déglon N; Aebischer P; Severini GM; Bordignon C
J Neurol Sci; 2007 Apr; 255(1-2):7-16. PubMed ID: 17341424
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]