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 *

244 related articles for article (PubMed ID: 28451919)

  • 1. 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]  

  • 2. 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]  

  • 3. 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]  

  • 4. 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]  

  • 5. 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]  

  • 6. A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome.
    Winner LK; Beard H; Hassiotis S; Lau AA; Luck AJ; Hopwood JJ; Hemsley KM
    Hum Gene Ther; 2016 May; 27(5):363-75. PubMed ID: 26975339
    [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. Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA.
    Crawley AC; Gliddon BL; Auclair D; Brodie SL; Hirte C; King BM; Fuller M; Hemsley KM; Hopwood JJ
    Brain Res; 2006 Aug; 1104(1):1-17. PubMed ID: 16828069
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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]  

  • 11. 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]  

  • 12. An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome).
    Pollock K; Noritake S; Imai DM; Pastenkos G; Olson M; Cary W; Yang S; Fierro FA; White J; Graham J; Dahlenburg H; Johe K; Nolta JA
    Sci Rep; 2023 Oct; 13(1):18439. PubMed ID: 37891179
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice.
    Mader KM; Beard H; King BM; Hopwood JJ
    Genes Brain Behav; 2008 Oct; 7(7):740-53. PubMed ID: 18518922
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Accelerated clinical disease and pathology in mucopolysaccharidosis type IIIB and GalNAc transferase double knockout mice.
    Mohammed EE; Snella EM; Rutz-Mendicino MM; Echevarria FD; Awedikian R; Whitley EM; Ellinwood NM
    Mol Genet Metab; 2012 Sep; 107(1-2):129-35. PubMed ID: 22867887
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 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]  

  • 16. [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]  

  • 17. Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease.
    Sergijenko A; Langford-Smith A; Liao AY; Pickford CE; McDermott J; Nowinski G; Langford-Smith KJ; Merry CL; Jones SA; Wraith JE; Wynn RF; Wilkinson FL; Bigger BW
    Mol Ther; 2013 Oct; 21(10):1938-49. PubMed ID: 23748415
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA.
    Gray AL; O'Leary C; Liao A; Agúndez L; Youshani AS; Gleitz HF; Parker H; Taylor JT; Danos O; Hocquemiller M; Palomar N; Linden RM; Henckaerts E; Holley RJ; Bigger BW
    Hum Gene Ther; 2019 Sep; 30(9):1052-1066. PubMed ID: 31020862
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Axonal dystrophy in the brain of mice with Sanfilippo syndrome.
    Beard H; Hassiotis S; Gai WP; Parkinson-Lawrence E; Hopwood JJ; Hemsley KM
    Exp Neurol; 2017 Sep; 295():243-255. PubMed ID: 28601604
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.