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Results: 1 to 20 of 235

Related Citations for PubMed (Select 16311251)

1.

Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

Sevin C, Benraiss A, Van Dam D, Bonnin D, Nagels G, Verot L, Laurendeau I, Vidaud M, Gieselmann V, Vanier M, De Deyn PP, Aubourg P, Cartier N.

Hum Mol Genet. 2006 Jan 1;15(1):53-64. Epub 2005 Nov 25.

2.

Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer.

Sevin C, Verot L, Benraiss A, Van Dam D, Bonnin D, Nagels G, Fouquet F, Gieselmann V, Vanier MT, De Deyn PP, Aubourg P, Cartier N.

Gene Ther. 2007 Mar;14(5):405-14. Epub 2006 Nov 9.

PMID:
17093507
3.

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. doi: 10.1089/hum.2012.015. Epub 2012 Jul 23.

4.

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 1;19(1):147-58. doi: 10.1093/hmg/ddp475. Epub .

5.

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. doi: 10.1007/s11011-008-9107-4. Epub 2008 Sep 17.

PMID:
18797988
6.

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice.

Consiglio A, Quattrini A, Martino S, Bensadoun JC, Dolcetta D, Trojani A, Benaglia G, Marchesini S, Cestari V, Oliverio A, Bordignon C, Naldini L.

Nat Med. 2001 Mar;7(3):310-6.

PMID:
11231629
7.

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 1;19(11):2208-27. doi: 10.1093/hmg/ddq099. Epub 2010 Mar 4.

8.

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 13;1094(1):13-23. Epub 2006 May 26.

PMID:
16729983
9.

Gene therapy in metachromatic leukodystrophy.

Sevin C, Cartier-Lacave N, Aubourg P.

Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S128-31. Review.

PMID:
20040324
10.

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.

PMID:
17845130
11.

Adult onset metachromatic leukodystrophy without electroclinical peripheral nervous system involvement: a new mutation in the ARSA gene.

Marcão AM, Wiest R, Schindler K, Wiesmann U, Weis J, Schroth G, Miranda MC, Sturzenegger M, Gieselmann V.

Arch Neurol. 2005 Feb;62(2):309-13.

PMID:
15710861
12.

[Molecular screening of the major mutations in the ARSA gene in patients with metachromatic leukodystrophy].

Horovenko NH, Ol'khovych NV, Pichkur NO.

Tsitol Genet. 2002 Sep-Oct;36(5):43-8. Ukrainian.

PMID:
12442547
13.
14.

Gene therapy for metachromatic leukodystrophy.

Ohashi T, Watabe K, Sato Y, Saito I, Barranger JA, Matalon R, Eto Y.

Acta Paediatr Jpn. 1996 Apr;38(2):193-201.

PMID:
8677802
15.
16.

Gene therapy of metachromatic leukodystrophy.

Matzner U, Gieselmann V.

Expert Opin Biol Ther. 2005 Jan;5(1):55-65. Review.

PMID:
15709909
18.

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.

PMID:
16087406
19.

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. Epub 2006 Jul 27.

PMID:
16871228
20.

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.

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