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 *

511 related articles for article (PubMed ID: 30404653)

  • 41. Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.
    Xu F; Ding E; Liao SX; Migone F; Dai J; Schneider A; Serra D; Chen YT; Amalfitano A
    Gene Ther; 2004 Nov; 11(21):1590-8. PubMed ID: 15356673
    [TBL] [Abstract][Full Text] [Related]  

  • 42. Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology.
    Turner SMF; Falk DJ; Byrne BJ; Fuller DD
    Physiol Genomics; 2016 Nov; 48(11):785-794. PubMed ID: 27614205
    [TBL] [Abstract][Full Text] [Related]  

  • 43. Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.
    Qiu K; Falk DJ; Reier PJ; Byrne BJ; Fuller DD
    Mol Ther; 2012 Jan; 20(1):21-7. PubMed ID: 22008916
    [TBL] [Abstract][Full Text] [Related]  

  • 44. Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression.
    Sun B; Zhang H; Bird A; Li S; Young SP; Koeberl DD
    J Gene Med; 2009 Oct; 11(10):913-20. PubMed ID: 19621331
    [TBL] [Abstract][Full Text] [Related]  

  • 45. Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease.
    Bijvoet AG; van de Kamp EH; Kroos MA; Ding JH; Yang BZ; Visser P; Bakker CE; Verbeet MP; Oostra BA; Reuser AJ; van der Ploeg AT
    Hum Mol Genet; 1998 Jan; 7(1):53-62. PubMed ID: 9384603
    [TBL] [Abstract][Full Text] [Related]  

  • 46. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
    Puzzo F; Colella P; Biferi MG; Bali D; Paulk NK; Vidal P; Collaud F; Simon-Sola M; Charles S; Hardet R; Leborgne C; Meliani A; Cohen-Tannoudji M; Astord S; Gjata B; Sellier P; van Wittenberghe L; Vignaud A; Boisgerault F; Barkats M; Laforet P; Kay MA; Koeberl DD; Ronzitti G; Mingozzi F
    Sci Transl Med; 2017 Nov; 9(418):. PubMed ID: 29187643
    [TBL] [Abstract][Full Text] [Related]  

  • 47. Adenovirus-mediated transfer of the acid alpha-glucosidase gene into fibroblasts, myoblasts and myotubes from patients with glycogen storage disease type II leads to high level expression of enzyme and corrects glycogen accumulation.
    Nicolino MP; Puech JP; Kremer EJ; Reuser AJ; Mbebi C; Verdière-Sahuqué M; Kahn A; Poenaru L
    Hum Mol Genet; 1998 Oct; 7(11):1695-702. PubMed ID: 9736771
    [TBL] [Abstract][Full Text] [Related]  

  • 48. Adjunctive β2-agonist treatment reduces glycogen independently of receptor-mediated acid α-glucosidase uptake in the limb muscles of mice with Pompe disease.
    Farah BL; Madden L; Li S; Nance S; Bird A; Bursac N; Yen PM; Young SP; Koeberl DD
    FASEB J; 2014 May; 28(5):2272-80. PubMed ID: 24448824
    [TBL] [Abstract][Full Text] [Related]  

  • 49. Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease.
    Pauly DF; Fraites TJ; Toma C; Bayes HS; Huie ML; Hirschhorn R; Plotz PH; Raben N; Kessler PD; Byrne BJ
    Hum Gene Ther; 2001 Mar; 12(5):527-38. PubMed ID: 11268285
    [TBL] [Abstract][Full Text] [Related]  

  • 50. Degeneration of muscle spindles in a murine model of Pompe disease.
    Watkins B; Schultheiß J; Rafuna A; Hintze S; Meinke P; Schoser B; Kröger S
    Sci Rep; 2023 Apr; 13(1):6555. PubMed ID: 37085544
    [TBL] [Abstract][Full Text] [Related]  

  • 51. Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.
    Yi H; Sun T; Armstrong D; Borneman S; Yang C; Austin S; Kishnani PS; Sun B
    J Mol Med (Berl); 2017 May; 95(5):513-521. PubMed ID: 28154884
    [TBL] [Abstract][Full Text] [Related]  

  • 52. Enzyme replacement therapy in the mouse model of Pompe disease.
    Raben N; Danon M; Gilbert AL; Dwivedi S; Collins B; Thurberg BL; Mattaliano RJ; Nagaraju K; Plotz PH
    Mol Genet Metab; 2003; 80(1-2):159-69. PubMed ID: 14567965
    [TBL] [Abstract][Full Text] [Related]  

  • 53. Vacuolated PAS-positive lymphocytes as an hallmark of Pompe disease and other myopathies related to impaired autophagy.
    Pascarella A; Terracciano C; Farina O; Lombardi L; Esposito T; Napolitano F; Franzese G; Panella G; Tuccillo F; la Marca G; Bernardini S; Boffo S; Giordano A; Di Iorio G; Melone MAB; Sampaolo S
    J Cell Physiol; 2018 Aug; 233(8):5829-5837. PubMed ID: 29215735
    [TBL] [Abstract][Full Text] [Related]  

  • 54. Hyaluronidase increases the biodistribution of acid alpha-1,4 glucosidase in the muscle of Pompe disease mice: an approach to enhance the efficacy of enzyme replacement therapy.
    Matalon R; Surendran S; Campbell GA; Michals-Matalon K; Tyring SK; Grady J; Cheng S; Kaye E
    Biochem Biophys Res Commun; 2006 Nov; 350(3):783-7. PubMed ID: 17027913
    [TBL] [Abstract][Full Text] [Related]  

  • 55. Enhanced delivery of α-glucosidase for Pompe disease by ICAM-1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders.
    Hsu J; Northrup L; Bhowmick T; Muro S
    Nanomedicine; 2012 Jul; 8(5):731-9. PubMed ID: 21906578
    [TBL] [Abstract][Full Text] [Related]  

  • 56. Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction.
    Kyosen SO; Iizuka S; Kobayashi H; Kimura T; Fukuda T; Shen J; Shimada Y; Ida H; Eto Y; Ohashi T
    Gene Ther; 2010 Apr; 17(4):521-30. PubMed ID: 20033064
    [TBL] [Abstract][Full Text] [Related]  

  • 57. Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.
    Han SO; Li S; Brooks ED; Masat E; Leborgne C; Banugaria S; Bird A; Mingozzi F; Waldmann H; Koeberl D
    Hum Gene Ther; 2015 Jan; 26(1):26-35. PubMed ID: 25382056
    [TBL] [Abstract][Full Text] [Related]  

  • 58. What's new and what's next for gene therapy in Pompe disease?
    Roger AL; Sethi R; Huston ML; Scarrow E; Bao-Dai J; Lai E; Biswas DD; El Haddad L; Strickland LM; Kishnani PS; ElMallah MK
    Expert Opin Biol Ther; 2022 Sep; 22(9):1117-1135. PubMed ID: 35428407
    [TBL] [Abstract][Full Text] [Related]  

  • 59. Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail.
    Kikuchi T; Yang HW; Pennybacker M; Ichihara N; Mizutani M; Van Hove JL; Chen YT
    J Clin Invest; 1998 Feb; 101(4):827-33. PubMed ID: 9466978
    [TBL] [Abstract][Full Text] [Related]  

  • 60. Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.
    Sun B; Zhang H; Franco LM; Brown T; Bird A; Schneider A; Koeberl DD
    Mol Ther; 2005 Jun; 11(6):889-98. PubMed ID: 15922959
    [TBL] [Abstract][Full Text] [Related]  

    [Previous]   [Next]    [New Search]
    of 26.