BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

169 related articles for article (PubMed ID: 35997776)

  • 1. 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]  

  • 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. Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10.
    Gilkes JA; Bloom MD; Heldermon CD
    Gene Ther; 2016 Mar; 23(3):263-71. PubMed ID: 26674264
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Residual N-acetyl-α-glucosaminidase activity in fibroblasts correlates with disease severity in patients with mucopolysaccharidosis type IIIB.
    Meijer OLM; Welling L; Valstar MJ; Hoefsloot LH; Brüggenwirth HT; van der Ploeg AT; Ruijter GJG; Wagemans T; Wijburg FA; van Vlies N
    J Inherit Metab Dis; 2016 May; 39(3):437-445. PubMed ID: 26907177
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells.
    Huang W; Cheng YS; Yang S; Swaroop M; Xu M; Huang W; Zheng W
    Exp Cell Res; 2021 Oct; 407(1):112785. PubMed ID: 34411609
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 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. Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice.
    Fu H; Kang L; Jennings JS; Moy SS; Perez A; Dirosario J; McCarty DM; Muenzer J
    Gene Ther; 2007 Jul; 14(14):1065-77. PubMed ID: 17460717
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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]  

  • 11. 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]  

  • 12. Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse.
    Gilkes JA; Judkins BL; Herrera BN; Mandel RJ; Boye SL; Boye SE; Srivastava A; Heldermon CD
    Gene Ther; 2021 Aug; 28(7-8):447-455. PubMed ID: 33244179
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
    Kan SH; Aoyagi-Scharber M; Le SQ; Vincelette J; Ohmi K; Bullens S; Wendt DJ; Christianson TM; Tiger PM; Brown JR; Lawrence R; Yip BK; Holtzinger J; Bagri A; Crippen-Harmon D; Vondrak KN; Chen Z; Hague CM; Woloszynek JC; Cheung DS; Webster KA; Adintori EG; Lo MJ; Wong W; Fitzpatrick PA; LeBowitz JH; Crawford BE; Bunting S; Dickson PI; Neufeld EF
    Proc Natl Acad Sci U S A; 2014 Oct; 111(41):14870-5. PubMed ID: 25267636
    [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. 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]  

  • 16. 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]  

  • 17. 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]  

  • 18. Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.
    Ellinwood NM; Valentine BN; Hess AS; Jens JK; Snella EM; Jamil M; Hostetter SJ; Jeffery ND; Smith JD; Millman ST; Parsons RL; Butt MT; Chandra S; Egeland MT; Assis AB; Nelvagal HR; Cooper JD; Nestrasil I; Mueller BA; Labounek R; Paulson A; Prill H; Liu XY; Zhou H; Lawrence R; Crawford BE; Grover A; Cherala G; Melton AC; Cherukuri A; Vuillemenot BR; Wait JCM; O'Neill CA; Pinkstaff J; Kovalchin J; Zanelli E; McCullagh E
    J Pharmacol Exp Ther; 2022 Sep; 382(3):277-286. PubMed ID: 35717448
    [TBL] [Abstract][Full Text] [Related]  

  • 19. [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]  

  • 20. A model of mucopolysaccharidosis IIIB (Sanfilippo syndrome type IIIB): N-acetyl-alpha-D-glucosaminidase deficiency in Schipperke dogs.
    Ellinwood NM; Wang P; Skeen T; Sharp NJ; Cesta M; Decker S; Edwards NJ; Bublot I; Thompson JN; Bush W; Hardam E; Haskins ME; Giger U
    J Inherit Metab Dis; 2003; 26(5):489-504. PubMed ID: 14518829
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.