310 related articles for article (PubMed ID: 17080200)
1. 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]
2. 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]
3. Microglia: a cellular vehicle for CNS gene therapy.
Neumann H
J Clin Invest; 2006 Nov; 116(11):2857-60. PubMed ID: 17080190
[TBL] [Abstract][Full Text] [Related]
4. Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy.
Doerr J; Böckenhoff A; Ewald B; Ladewig J; Eckhardt M; Gieselmann V; Matzner U; Brüstle O; Koch P
Mol Ther; 2015 Sep; 23(9):1519-31. PubMed ID: 26061647
[TBL] [Abstract][Full Text] [Related]
5. Oligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy.
Givogri MI; Galbiati F; Fasano S; Amadio S; Perani L; Superchi D; Morana P; Del Carro U; Marchesini S; Brambilla R; Wrabetz L; Bongarzone E
J Neurosci; 2006 Mar; 26(12):3109-19. PubMed ID: 16554462
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Atidarsagene autotemcel for metachromatic leukodystrophy.
Messina M; Gissen P
Drugs Today (Barc); 2023 Feb; 59(2):63-70. PubMed ID: 36811406
[TBL] [Abstract][Full Text] [Related]
8. Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.
Meneghini V; Frati G; Sala D; De Cicco S; Luciani M; Cavazzin C; Paulis M; Mentzen W; Morena F; Giannelli S; Sanvito F; Villa A; Bulfone A; Broccoli V; Martino S; Gritti A
Stem Cells Transl Med; 2017 Feb; 6(2):352-368. PubMed ID: 28191778
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A.
Miyake N; Miyake K; Sakai A; Yamamoto M; Suzuki H; Shimada T
Sci Rep; 2021 Oct; 11(1):20513. PubMed ID: 34654893
[TBL] [Abstract][Full Text] [Related]
11. 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]
12. 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]
13. Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.
Piguet F; Sondhi D; Piraud M; Fouquet F; Hackett NR; Ahouansou O; Vanier MT; Bieche I; Aubourg P; Crystal RG; Cartier N; Sevin C
Hum Gene Ther; 2012 Aug; 23(8):903-14. PubMed ID: 22642214
[TBL] [Abstract][Full Text] [Related]
14. 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]
15. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.
Biffi A; De Palma M; Quattrini A; Del Carro U; Amadio S; Visigalli I; Sessa M; Fasano S; Brambilla R; Marchesini S; Bordignon C; Naldini L
J Clin Invest; 2004 Apr; 113(8):1118-29. PubMed ID: 15085191
[TBL] [Abstract][Full Text] [Related]
16. Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy.
Meneghini V; Lattanzi A; Tiradani L; Bravo G; Morena F; Sanvito F; Calabria A; Bringas J; Fisher-Perkins JM; Dufour JP; Baker KC; Doglioni C; Montini E; Bunnell BA; Bankiewicz K; Martino S; Naldini L; Gritti A
EMBO Mol Med; 2016 May; 8(5):489-510. PubMed ID: 27025653
[TBL] [Abstract][Full Text] [Related]
17. Brain cell type-specific endocytosis of arylsulfatase A identifies limitations of enzyme-based therapies for metachromatic leukodystrophy.
Kaminski D; Yaghootfam C; Matthes F; Reßing A; Gieselmann V; Matzner U
Hum Mol Genet; 2021 Feb; 29(23):3807-3817. PubMed ID: 33367737
[TBL] [Abstract][Full Text] [Related]
18. Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Fumagalli F; Calbi V; Natali Sora MG; Sessa M; Baldoli C; Rancoita PMV; Ciotti F; Sarzana M; Fraschini M; Zambon AA; Acquati S; Redaelli D; Attanasio V; Miglietta S; De Mattia F; Barzaghi F; Ferrua F; Migliavacca M; Tucci F; Gallo V; Del Carro U; Canale S; Spiga I; Lorioli L; Recupero S; Fratini ES; Morena F; Silvani P; Calvi MR; Facchini M; Locatelli S; Corti A; Zancan S; Antonioli G; Farinelli G; Gabaldo M; Garcia-Segovia J; Schwab LC; Downey GF; Filippi M; Cicalese MP; Martino S; Di Serio C; Ciceri F; Bernardo ME; Naldini L; Biffi A; Aiuti A
Lancet; 2022 Jan; 399(10322):372-383. PubMed ID: 35065785
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. 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]
[Next] [New Search]
