BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

161 related articles for article (PubMed ID: 37371072)

  • 1. Dysregulation of Metabolism and Proteostasis in Skeletal Muscle of a Presymptomatic Pompe Mouse Model.
    Rohm M; Volke L; Schlaffke L; Rehmann R; Südkamp N; Roos A; Schänzer A; Hentschel A; Vorgerd M
    Cells; 2023 Jun; 12(12):. PubMed ID: 37371072
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease.
    Liang Q; Vlaar EC; Pijnenburg JM; Rijkers E; Demmers JAA; Vulto AG; van der Ploeg AT; van Til NP; Pijnappel WWMP
    J Proteomics; 2024 Jan; 291():105037. PubMed ID: 38288553
    [TBL] [Abstract][Full Text] [Related]  

  • 3. GAA deficiency disrupts distal airway cells in Pompe disease.
    El Haddad L; Lai E; Murthy PKL; Biswas DD; Soufny R; Roger AL; Tata PR; ElMallah MK
    Am J Physiol Lung Cell Mol Physiol; 2023 Sep; 325(3):L288-L298. PubMed ID: 37366541
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
    Keeler AM; Zieger M; Todeasa SH; McCall AL; Gifford JC; Birsak S; Choudhury SR; Byrne BJ; Sena-Esteves M; ElMallah MK
    Hum Gene Ther; 2019 Jan; 30(1):57-68. PubMed ID: 29901418
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.
    Cagin U; Puzzo F; Gomez MJ; Moya-Nilges M; Sellier P; Abad C; Van Wittenberghe L; Daniele N; Guerchet N; Gjata B; Collaud F; Charles S; Sola MS; Boyer O; Krijnse-Locker J; Ronzitti G; Colella P; Mingozzi F
    Mol Ther; 2020 Sep; 28(9):2056-2072. PubMed ID: 32526204
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice.
    Foley JW; Bercury SD; Finn P; Cheng SH; Scheule RK; Ziegler RJ
    Mol Ther; 2010 Sep; 18(9):1584-91. PubMed ID: 20551907
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.
    Ziegler RJ; Bercury SD; Fidler J; Zhao MA; Foley J; Taksir TV; Ryan S; Hodges BL; Scheule RK; Shihabuddin LS; Cheng SH
    Hum Gene Ther; 2008 Jun; 19(6):609-21. PubMed ID: 18500944
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Dysregulation of multiple facets of glycogen metabolism in a murine model of Pompe disease.
    Taylor KM; Meyers E; Phipps M; Kishnani PS; Cheng SH; Scheule RK; Moreland RJ
    PLoS One; 2013; 8(2):e56181. PubMed ID: 23457523
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Lysosomal glycogen accumulation in Pompe disease results in disturbed cytoplasmic glycogen metabolism.
    Canibano-Fraile R; Harlaar L; Dos Santos CA; Hoogeveen-Westerveld M; Demmers JAA; Snijders T; Lijnzaad P; Verdijk RM; van der Beek NAME; van Doorn PA; van der Ploeg AT; Brusse E; Pijnappel WWMP; Schaaf GJ
    J Inherit Metab Dis; 2023 Jan; 46(1):101-115. PubMed ID: 36111639
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease.
    Douillard-Guilloux G; Raben N; Takikita S; Ferry A; Vignaud A; Guillet-Deniau I; Favier M; Thurberg BL; Roach PJ; Caillaud C; Richard E
    Hum Mol Genet; 2010 Feb; 19(4):684-96. PubMed ID: 19959526
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice.
    Sidman RL; Taksir T; Fidler J; Zhao M; Dodge JC; Passini MA; Raben N; Thurberg BL; Cheng SH; Shihabuddin LS
    J Neuropathol Exp Neurol; 2008 Aug; 67(8):803-18. PubMed ID: 18648322
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Airway smooth muscle dysfunction in Pompe (
    Keeler AM; Liu D; Zieger M; Xiong L; Salemi J; Bellvé K; Byrne BJ; Fuller DD; ZhuGe R; ElMallah MK
    Am J Physiol Lung Cell Mol Physiol; 2017 Jun; 312(6):L873-L881. PubMed ID: 28336814
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
    Colella P; Sellier P; Gomez MJ; Biferi MG; Tanniou G; Guerchet N; Cohen-Tannoudji M; Moya-Nilges M; van Wittenberghe L; Daniele N; Gjata B; Krijnse-Locker J; Collaud F; Simon-Sola M; Charles S; Cagin U; Mingozzi F
    EBioMedicine; 2020 Nov; 61():103052. PubMed ID: 33039711
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 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]  

  • 16. Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease.
    Schaaf GJ; van Gestel TJM; In 't Groen SLM; de Jong B; Boomaars B; Tarallo A; Cardone M; Parenti G; van der Ploeg AT; Pijnappel WWMP
    Acta Neuropathol Commun; 2018 Nov; 6(1):119. PubMed ID: 30404653
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.
    Raben N; Fukuda T; Gilbert AL; de Jong D; Thurberg BL; Mattaliano RJ; Meikle P; Hopwood JJ; Nagashima K; Nagaraju K; Plotz PH
    Mol Ther; 2005 Jan; 11(1):48-56. PubMed ID: 15585405
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme.
    Raben N; Jatkar T; Lee A; Lu N; Dwivedi S; Nagaraju K; Plotz PH
    Mol Ther; 2002 Nov; 6(5):601-8. PubMed ID: 12409258
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease.
    Peng J; Dalton J; Butt M; Tracy K; Kennedy D; Haroldsen P; Cahayag R; Zoog S; O'Neill CA; Tsuruda LS
    J Pharmacol Exp Ther; 2017 Feb; 360(2):313-323. PubMed ID: 27856936
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Clinical course, mutations and its functional characteristics of infantile-onset Pompe disease in Thailand.
    Ngiwsara L; Wattanasirichaigoon D; Tim-Aroon T; Rojnueangnit K; Noojaroen S; Khongkraparn A; Sawangareetrakul P; Ketudat-Cairns JR; Charoenwattanasatien R; Champattanachai V; Kuptanon C; Pangkanon S; Svasti J
    BMC Med Genet; 2019 Sep; 20(1):156. PubMed ID: 31510962
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.