261 related articles for article (PubMed ID: 21515587)
1. Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy.
Stroobants S; Gerlach D; Matthes F; Hartmann D; Fogh J; Gieselmann V; D'Hooge R; Matzner U
Hum Mol Genet; 2011 Jul; 20(14):2760-9. PubMed ID: 21515587
[TBL] [Abstract][Full Text] [Related]
2. Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy.
Matzner U; Herbst E; Hedayati KK; Lüllmann-Rauch R; Wessig C; Schröder S; Eistrup C; Möller C; Fogh J; Gieselmann V
Hum Mol Genet; 2005 May; 14(9):1139-52. PubMed ID: 15772092
[TBL] [Abstract][Full Text] [Related]
3. Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.
Matzner U; Lüllmann-Rauch R; Stroobants S; Andersson C; Weigelt C; Eistrup C; Fogh J; D'Hooge R; Gieselmann V
Mol Ther; 2009 Apr; 17(4):600-6. PubMed ID: 19174759
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. Induction of tolerance to human arylsulfatase A in a mouse model of metachromatic leukodystrophy.
Matzner U; Matthes F; Herbst E; Lüllmann-Rauch R; Callaerts-Vegh Z; D'Hooge R; Weigelt C; Eistrup C; Fogh J; Gieselmann V
Mol Med; 2007; 13(9-10):471-9. PubMed ID: 17660863
[TBL] [Abstract][Full Text] [Related]
6. A spontaneously immortalized Schwann cell line to study the molecular aspects of metachromatic leukodystrophy.
Saravanan K; Büssow H; Weiler N; Gieselmann V; Franken S
J Neurosci Methods; 2007 Apr; 161(2):223-33. PubMed ID: 17204333
[TBL] [Abstract][Full Text] [Related]
7. 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]
8. 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]
9. Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial.
Í Dali C; Sevin C; Krägeloh-Mann I; Giugliani R; Sakai N; Wu J; Wasilewski M
Mol Genet Metab; 2020; 131(1-2):235-244. PubMed ID: 32792226
[TBL] [Abstract][Full Text] [Related]
10. Potential of surfactant-coated nanoparticles to improve brain delivery of arylsulfatase A.
Schuster T; Mühlstein A; Yaghootfam C; Maksimenko O; Shipulo E; Gelperina S; Kreuter J; Gieselmann V; Matzner U
J Control Release; 2017 May; 253():1-10. PubMed ID: 28215668
[TBL] [Abstract][Full Text] [Related]
11. Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector.
Miyake N; Miyake K; Asakawa N; Yamamoto M; Shimada T
Gene Ther; 2014 Apr; 21(4):427-33. PubMed ID: 24572788
[TBL] [Abstract][Full Text] [Related]
12. 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]
13. Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations.
Matzner U; Hartmann D; Lüllmann-Rauch R; Coenen R; Rothert F; Månsson JE; Fredman P; D'Hooge R; De Deyn PP; Gieselmann V
Gene Ther; 2002 Jan; 9(1):53-63. PubMed ID: 11850723
[TBL] [Abstract][Full Text] [Related]
14. 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]
15. Comparison of five peptide vectors for improved brain delivery of the lysosomal enzyme arylsulfatase A.
Böckenhoff A; Cramer S; Wölte P; Knieling S; Wohlenberg C; Gieselmann V; Galla HJ; Matzner U
J Neurosci; 2014 Feb; 34(9):3122-9. PubMed ID: 24573272
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Increasing sulfatide synthesis in myelin-forming cells of arylsulfatase A-deficient mice causes demyelination and neurological symptoms reminiscent of human metachromatic leukodystrophy.
Ramakrishnan H; Hedayati KK; Lüllmann-Rauch R; Wessig C; Fewou SN; Maier H; Goebel HH; Gieselmann V; Eckhardt M
J Neurosci; 2007 Aug; 27(35):9482-90. PubMed ID: 17728461
[TBL] [Abstract][Full Text] [Related]
18. Transport of arylsulfatase A across the blood-brain barrier in vitro.
Matthes F; Wölte P; Böckenhoff A; Hüwel S; Schulz M; Hyden P; Fogh J; Gieselmann V; Galla HJ; Matzner U
J Biol Chem; 2011 May; 286(20):17487-94. PubMed ID: 21454621
[TBL] [Abstract][Full Text] [Related]
19. Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease.
Matzner U; Matthes F; Weigelt C; Andersson C; Eistrup C; Fogh J; Gieselmann V
J Mol Med (Berl); 2008 Apr; 86(4):433-42. PubMed ID: 18360747
[TBL] [Abstract][Full Text] [Related]
20. 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. PubMed ID: 17093507
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
