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.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

326 related articles for article (PubMed ID: 34073041)

  • 1. 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]  

  • 2. 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]  

  • 3. A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome.
    Lau AA; King BM; Thorsen CL; Hassiotis S; Beard H; Trim PJ; Whyte LS; Tamang SJ; Duplock SK; Snel MF; Hopwood JJ; Hemsley KM
    J Inherit Metab Dis; 2017 Sep; 40(5):715-724. PubMed ID: 28451919
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.
    Fraldi A; Hemsley K; Crawley A; Lombardi A; Lau A; Sutherland L; Auricchio A; Ballabio A; Hopwood JJ
    Hum Mol Genet; 2007 Nov; 16(22):2693-702. PubMed ID: 17725987
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Reduction in Brain Heparan Sulfate with Systemic Administration of an IgG Trojan Horse-Sulfamidase Fusion Protein in the Mucopolysaccharidosis Type IIIA Mouse.
    Boado RJ; Lu JZ; Hui EK; Pardridge WM
    Mol Pharm; 2018 Feb; 15(2):602-608. PubMed ID: 29251941
    [TBL] [Abstract][Full Text] [Related]  

  • 6. The mutation p.Ser298Pro in the sulphamidase gene (SGSH) is associated with a slowly progressive clinical phenotype in mucopolysaccharidosis type IIIA (Sanfilippo A syndrome).
    Meyer A; Kossow K; Gal A; Steglich C; Mühlhausen C; Ullrich K; Braulke T; Muschol N
    Hum Mutat; 2008 May; 29(5):770. PubMed ID: 18407553
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction.
    Magat J; Jones S; Baridon B; Agrawal V; Wong H; Giaramita A; Mangini L; Handyside B; Vitelli C; Parker M; Yeung N; Zhou Y; Pungor E; Slabodkin I; Gorostiza O; Aguilera A; Lo MJ; Alcozie S; Christianson TM; Tiger PMN; Vincelette J; Fong S; Gil G; Hague C; Lawrence R; Wendt DJ; Lebowitz JH; Bunting S; Bullens S; Crawford BE; Roy SM; Woloszynek JC
    J Biol Chem; 2022 Dec; 298(12):102625. PubMed ID: 36306823
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Glycosaminoglycan levels and structure in a mucopolysaccharidosis IIIA mice and the effect of a highly secreted sulfamidase engineered to cross the blood-brain barrier.
    Maccari F; Sorrentino NC; Mantovani V; Galeotti F; Fraldi A; Volpi N
    Metab Brain Dis; 2017 Feb; 32(1):203-210. PubMed ID: 27585464
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.
    Chen Y; Zheng S; Tecedor L; Davidson BL
    Mol Ther; 2018 Apr; 26(4):1118-1126. PubMed ID: 29503202
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications.
    Yogalingam G; Hopwood JJ
    Hum Mutat; 2001 Oct; 18(4):264-81. PubMed ID: 11668611
    [TBL] [Abstract][Full Text] [Related]  

  • 12. [Postnatal and prenatal diagnosis of mucopolysaccharidosis type III (Sanfilippo syndrome)].
    Zhang WM; Shi HP; Meng Y; Li BT; Qiu ZQ; Liu JT
    Zhonghua Er Ke Za Zhi; 2008 Jun; 46(6):407-10. PubMed ID: 19099774
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Bioinformatics classification of mutations in patients with Mucopolysaccharidosis IIIA.
    Tanwar H; Kumar DT; Doss CGP; Zayed H
    Metab Brain Dis; 2019 Dec; 34(6):1577-1594. PubMed ID: 31385193
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Insulin receptor antibody-sulfamidase fusion protein penetrates the primate blood-brain barrier and reduces glycosoaminoglycans in Sanfilippo type A cells.
    Boado RJ; Lu JZ; Hui EK; Pardridge WM
    Mol Pharm; 2014 Aug; 11(8):2928-34. PubMed ID: 24949884
    [TBL] [Abstract][Full Text] [Related]  

  • 15. A multiparametric computational algorithm for comprehensive assessment of genetic mutations in mucopolysaccharidosis type IIIA (Sanfilippo syndrome).
    Ugrinov KG; Freed SD; Thomas CL; Lee SW
    PLoS One; 2015; 10(3):e0121511. PubMed ID: 25807448
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Late-onset mucopolysaccharidosis type IIIA mimicking Usher syndrome.
    De Falco A; Karali M; Criscuolo C; Testa F; Barillari MR; Scarpato M; Gaudieri V; Cuocolo A; Russo A; Nigro V; Simonelli F; Banfi S; Brunetti-Pierri N
    Am J Med Genet A; 2024 May; 194(5):e63517. PubMed ID: 38149346
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Residual activity and proteasomal degradation of p.Ser298Pro sulfamidase identified in patients with a mild clinical phenotype of Sanfilippo A syndrome.
    Muschol N; Pohl S; Meyer A; Gal A; Ullrich K; Braulke T
    Am J Med Genet A; 2011 Jul; 155A(7):1634-9. PubMed ID: 21671382
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group.
    Köhn AF; Grigull L; du Moulin M; Kabisch S; Ammer L; Rudolph C; Muschol NM
    Mol Genet Metab Rep; 2020 Jun; 23():100578. PubMed ID: 32226768
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 17.