200 related articles for article (PubMed ID: 15649123)
1. Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector.
Di Natale P; Di Domenico C; Gargiulo N; Castaldo S; Gonzalez Y Reyero E; Mithbaokar P; De Felice M; Follenzi A; Naldini L; Villani GR
Biochem J; 2005 Jun; 388(Pt 2):639-46. PubMed ID: 15649123
[TBL] [Abstract][Full Text] [Related]
2. Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy.
Holley RJ; Ellison SM; Fil D; O'Leary C; McDermott J; Senthivel N; Langford-Smith AWW; Wilkinson FL; D'Souza Z; Parker H; Liao A; Rowlston S; Gleitz HFE; Kan SH; Dickson PI; Bigger BW
Brain; 2018 Jan; 141(1):99-116. PubMed ID: 29186350
[TBL] [Abstract][Full Text] [Related]
3. Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.
Cressant A; Desmaris N; Verot L; Bréjot T; Froissart R; Vanier MT; Maire I; Heard JM
J Neurosci; 2004 Nov; 24(45):10229-39. PubMed ID: 15537895
[TBL] [Abstract][Full Text] [Related]
4. Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer.
Villani GR; Follenzi A; Vanacore B; Di Domenico C; Naldini L; Di Natale P
Biochem J; 2002 Jun; 364(Pt 3):747-53. PubMed ID: 12049639
[TBL] [Abstract][Full Text] [Related]
5. Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.
Di Domenico C; Villani GR; Di Napoli D; Reyero EG; Lombardo A; Naldini L; Di Natale P
Hum Gene Ther; 2005 Jan; 16(1):81-90. PubMed ID: 15703491
[TBL] [Abstract][Full Text] [Related]
6. Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.
Zheng Y; Ryazantsev S; Ohmi K; Zhao HZ; Rozengurt N; Kohn DB; Neufeld EF
Mol Genet Metab; 2004 Aug; 82(4):286-95. PubMed ID: 15308126
[TBL] [Abstract][Full Text] [Related]
7. Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB.
Di Domenico C; Villani GR; Di Napoli D; Nusco E; Calì G; Nitsch L; Di Natale P
Am J Med Genet A; 2009 Jun; 149A(6):1209-18. PubMed ID: 19449420
[TBL] [Abstract][Full Text] [Related]
8. Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.
Kan SH; Elsharkawi I; Le SQ; Prill H; Mangini L; Cooper JD; Lawrence R; Sands MS; Crawford BE; Dickson PI
Mol Genet Metab; 2021 Jun; 133(2):185-192. PubMed ID: 33839004
[TBL] [Abstract][Full Text] [Related]
9. Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery.
Fu H; DiRosario J; Kang L; Muenzer J; McCarty DM
J Gene Med; 2010 Jul; 12(7):624-33. PubMed ID: 20603889
[TBL] [Abstract][Full Text] [Related]
10. Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B.
Yu WH; Zhao KW; Ryazantsev S; Rozengurt N; Neufeld EF
Mol Genet Metab; 2000 Dec; 71(4):573-80. PubMed ID: 11136549
[TBL] [Abstract][Full Text] [Related]
11. Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.
Heldermon CD; Qin EY; Ohlemiller KK; Herzog ED; Brown JR; Vogler C; Hou W; Orrock JL; Crawford BE; Sands MS
Gene Ther; 2013 Sep; 20(9):913-21. PubMed ID: 23535899
[TBL] [Abstract][Full Text] [Related]
12. Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery.
Fu H; Samulski RJ; McCown TJ; Picornell YJ; Fletcher D; Muenzer J
Mol Ther; 2002 Jan; 5(1):42-9. PubMed ID: 11786044
[TBL] [Abstract][Full Text] [Related]
13. Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome.
Gougeon ML; Poirier-Beaudouin B; Ausseil J; Zérah M; Artaud C; Heard JM; Deiva K; Tardieu M
Front Immunol; 2021; 12():655478. PubMed ID: 34040605
[TBL] [Abstract][Full Text] [Related]
14. Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector.
Rouse CJ; Hawkins K; Kabbej N; Dalugdug J; Kunta A; Kim MJ; Someya S; Herbst Z; Gelb M; Dinelli I; Butterworth E; Falk DJ; Rosenkrantz E; Elmohd H; Khaledi H; Mowafy S; Ashby F; Heldermon CD
Hum Mol Genet; 2023 Jan; 32(3):417-430. PubMed ID: 35997776
[TBL] [Abstract][Full Text] [Related]
15. Processing of mutant N-acetyl-α-glucosaminidase in mucopolysaccharidosis type IIIB fibroblasts cultured at low temperature.
Meijer OLM; Te Brinke H; Ofman R; IJlst L; Wijburg FA; van Vlies N
Mol Genet Metab; 2017 Sep; 122(1-2):100-106. PubMed ID: 28751108
[TBL] [Abstract][Full Text] [Related]
16. Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.
Fu H; Meadows AS; Ware T; Mohney RP; McCarty DM
Mol Ther; 2017 Mar; 25(3):792-802. PubMed ID: 28143737
[TBL] [Abstract][Full Text] [Related]
17. Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase.
Li HH; Yu WH; Rozengurt N; Zhao HZ; Lyons KM; Anagnostaras S; Fanselow MS; Suzuki K; Vanier MT; Neufeld EF
Proc Natl Acad Sci U S A; 1999 Dec; 96(25):14505-10. PubMed ID: 10588735
[TBL] [Abstract][Full Text] [Related]
18. Serum MIP-1 alpha level: a biomarker for the follow-up of lentiviral therapy in mucopolysaccharidosis IIIB mice.
Di Natale P; Di Domenico C; Di Napoli D
J Inherit Metab Dis; 2010 Apr; 33(2):159-65. PubMed ID: 20162365
[TBL] [Abstract][Full Text] [Related]
19. Molecular defects in Sanfilippo syndrome type B (mucopolysaccharidosis IIIB).
Beesley CE; Jackson M; Young EP; Vellodi A; Winchester BG
J Inherit Metab Dis; 2005; 28(5):759-67. PubMed ID: 16151907
[TBL] [Abstract][Full Text] [Related]
20. Molecular basis of mucopolysaccharidosis type IIIB in emu (Dromaius novaehollandiae): an avian model of Sanfilippo syndrome type B.
Aronovich EL; Johnston JM; Wang P; Giger U; Whitley CB
Genomics; 2001 Jun; 74(3):299-305. PubMed ID: 11414757
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
