These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
170 related articles for article (PubMed ID: 33313335)
1. Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector. Bobo TA; Samowitz PN; Robinson MI; Fu H Mol Ther Methods Clin Dev; 2020 Dec; 19():474-485. PubMed ID: 33313335 [TBL] [Abstract][Full Text] [Related]
2. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery. Fu H; Cataldi MP; Ware TA; Zaraspe K; Meadows AS; Murrey DA; McCarty DM Mol Ther Methods Clin Dev; 2016; 3():16036. PubMed ID: 27331076 [TBL] [Abstract][Full Text] [Related]
3. Systemic scAAV9.U1a.hSGSH Delivery Corrects Brain Biochemistry in Mucopolysaccharidosis Type IIIA at Early and Later Stages of Disease. Saville JT; Derrick-Roberts ALK; McIntyre C; Fuller M Hum Gene Ther; 2021 Apr; 32(7-8):420-430. PubMed ID: 33339477 [TBL] [Abstract][Full Text] [Related]
5. Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice. Duncan FJ; Naughton BJ; Zaraspe K; Murrey DA; Meadows AS; Clark KR; Newsom DE; White P; Fu H; McCarty DM Mol Ther; 2015 Apr; 23(4):638-47. PubMed ID: 25592334 [TBL] [Abstract][Full Text] [Related]
6. AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains. Hocquemiller M; Hemsley KM; Douglass ML; Tamang SJ; Neumann D; King BM; Beard H; Trim PJ; Winner LK; Lau AA; Snel MF; Gomila C; Ausseil J; Mei X; Giersch L; Plavsic M; Laufer R Mol Ther Methods Clin Dev; 2020 Jun; 17():174-187. PubMed ID: 31909089 [TBL] [Abstract][Full Text] [Related]
7. An Engineered Douek AM; Amiri Khabooshan M; Henry J; Stamatis SA; Kreuder F; Ramm G; Änkö ML; Wlodkowic D; Kaslin J Int J Mol Sci; 2021 May; 22(11):. PubMed ID: 34073041 [TBL] [Abstract][Full Text] [Related]
8. Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice. Gliddon BL; Hopwood JJ Pediatr Res; 2004 Jul; 56(1):65-72. PubMed ID: 15128919 [TBL] [Abstract][Full Text] [Related]
9. SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors. Lau AA; Hopwood JJ; Kremer EJ; Hemsley KM Mol Genet Metab; 2010 Jun; 100(2):168-75. PubMed ID: 20231109 [TBL] [Abstract][Full Text] [Related]
10. Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA. Webber DL; Choo A; Hewson LJ; Trim PJ; Snel MF; Hopwood JJ; Richards RI; Hemsley KM; O'Keefe LV Exp Neurol; 2018 May; 303():38-47. PubMed ID: 29408731 [TBL] [Abstract][Full Text] [Related]
11. Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery. McIntyre C; Derrick-Roberts AL; Byers S; Anson DS J Gene Med; 2014; 16(11-12):374-87. PubMed ID: 25418946 [TBL] [Abstract][Full Text] [Related]
12. Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Fu H; Dirosario J; Killedar S; Zaraspe K; McCarty DM Mol Ther; 2011 Jun; 19(6):1025-33. PubMed ID: 21386820 [TBL] [Abstract][Full Text] [Related]
13. Neonatal Bone Marrow Transplantation in MPS IIIA Mice. Lau AA; Shamsani NJ; Winner LK; Hassiotis S; King BM; Hopwood JJ; Hemsley KM JIMD Rep; 2013; 8():121-32. PubMed ID: 23430528 [TBL] [Abstract][Full Text] [Related]
15. Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Murrey DA; Naughton BJ; Duncan FJ; Meadows AS; Ware TA; Campbell KJ; Bremer WG; Walker CM; Goodchild L; Bolon B; La Perle K; Flanigan KM; McBride KL; McCarty DM; Fu H Hum Gene Ther Clin Dev; 2014 Jun; 25(2):72-84. PubMed ID: 24720466 [TBL] [Abstract][Full Text] [Related]
16. A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB. Meadows AS; Duncan FJ; Camboni M; Waligura K; Montgomery C; Zaraspe K; Naughton BJ; Bremer WG; Shilling C; Walker CM; Bolon B; Flanigan KM; McBride KL; McCarty DM; Fu H Hum Gene Ther Clin Dev; 2015 Dec; 26(4):228-42. PubMed ID: 26684447 [TBL] [Abstract][Full Text] [Related]
17. 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]
18. Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA. King B; Hassiotis S; Rozaklis T; Beard H; Trim PJ; Snel MF; Hopwood JJ; Hemsley KM J Neurochem; 2016 May; 137(3):409-22. PubMed ID: 26762778 [TBL] [Abstract][Full Text] [Related]
19. High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector. Quiviger M; Arfi A; Mansard D; Delacotte L; Pastor M; Scherman D; Marie C Gene Ther; 2014 Dec; 21(12):1001-7. PubMed ID: 25142140 [TBL] [Abstract][Full Text] [Related]