950 related articles for article (PubMed ID: 28191778)
1. 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]
2. 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]
3. 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]
4. Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy.
Frati G; Luciani M; Meneghini V; De Cicco S; Ståhlman M; Blomqvist M; Grossi S; Filocamo M; Morena F; Menegon A; Martino S; Gritti A
Cell Death Dis; 2018 Jun; 9(6):698. PubMed ID: 29899471
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. 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]
7. 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]
8. 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]
9. 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]
10. Generation of a human iPSC line (MPIi007-A) from a patient with Metachromatic leukodystrophy.
Kim KP; Yoon J; Kim J; Röpke A; Shin B; Wook Han D; Greber B; Schöler HR
Stem Cell Res; 2020 Oct; 48():101993. PubMed ID: 32950023
[TBL] [Abstract][Full Text] [Related]
11. 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]
12. 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]
13. In vitro correction of ARSA deficiency in human skin fibroblasts from metachromatic leukodystrophy patients after treatment with microencapsulated recombinant cells.
Lagranha VL; Baldo G; de Carvalho TG; Burin M; Saraiva-Pereira ML; Matte U; Giugliani R
Metab Brain Dis; 2008 Dec; 23(4):469-84. PubMed ID: 18797988
[TBL] [Abstract][Full Text] [Related]
14. 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]
15. 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]
16. High residual arylsulfatase A (ARSA) activity in a patient with late-infantile metachromatic leukodystrophy.
Kreysing J; Bohne W; Bösenberg C; Marchesini S; Turpin JC; Baumann N; von Figura K; Gieselmann V
Am J Hum Genet; 1993 Aug; 53(2):339-46. PubMed ID: 8101038
[TBL] [Abstract][Full Text] [Related]
17. Embryonic stem cell-based reduction of central nervous system sulfatide storage in an animal model of metachromatic leukodystrophy.
Klein D; Schmandt T; Muth-Köhne E; Perez-Bouza A; Segschneider M; Gieselmann V; Brüstle O
Gene Ther; 2006 Dec; 13(24):1686-95. PubMed ID: 16871228
[TBL] [Abstract][Full Text] [Related]
18. 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]
19. 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]
20. Identification of a new Arylsulfatase A (ARSA) gene mutation in Tunisian patients with metachromatic leukodystrophy (MLD).
Dorboz I; Eymard-Pierre E; Kefi R; Abdelhak S; Miladi N; Boespflug-Tanguy O;
J Neurol Sci; 2009 Dec; 287(1-2):278-80. PubMed ID: 19699491
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
