372 related articles for article (PubMed ID: 27638601)
1. Gene therapy for metachromatic leukodystrophy.
Rosenberg JB; Kaminsky SM; Aubourg P; Crystal RG; Sondhi D
J Neurosci Res; 2016 Nov; 94(11):1169-79. PubMed ID: 27638601
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. Developing therapeutic approaches for metachromatic leukodystrophy.
Patil SA; Maegawa GH
Drug Des Devel Ther; 2013; 7():729-45. PubMed ID: 23966770
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. Metachromatic leukodystrophy: recent research developments.
Gieselmann V
J Child Neurol; 2003 Sep; 18(9):591-4. PubMed ID: 14572136
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.
Rosenberg JB; Sondhi D; Rubin DG; Monette S; Chen A; Cram S; De BP; Kaminsky SM; Sevin C; Aubourg P; Crystal RG
Hum Gene Ther Clin Dev; 2014 Sep; 25(3):164-77. PubMed ID: 25144894
[TBL] [Abstract][Full Text] [Related]
8. Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy.
Rosenberg JB; Chen A; De BP; Dyke JP; Ballon DJ; Monette S; Ricart Arbona RJ; Kaminsky SM; Crystal RG; Sondhi D
Hum Gene Ther; 2021 Jun; 32(11-12):563-580. PubMed ID: 33380277
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. 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]
11. 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]
12. 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]
13. 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]
14. Transduced fibroblasts and metachromatic leukodystrophy lymphocytes transfer arylsulfatase A to myelinating glia and deficient cells in vitro.
Sangalli A; Taveggia C; Salviati A; Wrabetz L; Bordignon C; Severini GM
Hum Gene Ther; 1998 Sep; 9(14):2111-9. PubMed ID: 9759937
[TBL] [Abstract][Full Text] [Related]
15. Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.
Zerah M; Piguet F; Colle MA; Raoul S; Deschamps JY; Deniaud J; Gautier B; Toulgoat F; Bieche I; Laurendeau I; Sondhi D; Souweidane MM; Cartier-Lacave N; Moullier P; Crystal RG; Roujeau T; Sevin C; Aubourg P
Hum Gene Ther Clin Dev; 2015 Jun; 26(2):113-24. PubMed ID: 25758611
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector.
Hironaka K; Yamazaki Y; Hirai Y; Yamamoto M; Miyake N; Miyake K; Okada T; Morita A; Shimada T
Sci Rep; 2015 Aug; 5():13104. PubMed ID: 26283284
[TBL] [Abstract][Full Text] [Related]
18. 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]
19. Developing treatment options for metachromatic leukodystrophy.
Batzios SP; Zafeiriou DI
Mol Genet Metab; 2012 Jan; 105(1):56-63. PubMed ID: 22078456
[TBL] [Abstract][Full Text] [Related]
20. Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.
Matthes F; Stroobants S; Gerlach D; Wohlenberg C; Wessig C; Fogh J; Gieselmann V; Eckhardt M; D'Hooge R; Matzner U
Hum Mol Genet; 2012 Jun; 21(11):2599-609. PubMed ID: 22388935
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
