BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

392 related articles for article (PubMed ID: 29262333)

  • 1. Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
    An D; Schneller JL; Frassetto A; Liang S; Zhu X; Park JS; Theisen M; Hong SJ; Zhou J; Rajendran R; Levy B; Howell R; Besin G; Presnyak V; Sabnis S; Murphy-Benenato KE; Kumarasinghe ES; Salerno T; Mihai C; Lukacs CM; Chandler RJ; Guey LT; Venditti CP; Martini PGV
    Cell Rep; 2017 Dec; 21(12):3548-3558. PubMed ID: 29262333
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia.
    An D; Frassetto A; Jacquinet E; Eybye M; Milano J; DeAntonis C; Nguyen V; Laureano R; Milton J; Sabnis S; Lukacs CM; Guey LT
    EBioMedicine; 2019 Jul; 45():519-528. PubMed ID: 31303505
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.
    Chandler RJ; Venditti CP
    Hum Gene Ther; 2019 Oct; 30(10):1236-1244. PubMed ID: 31303064
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.
    Chandler RJ; Venturoni LE; Liao J; Hubbard BT; Schneller JL; Hoffmann V; Gordo S; Zang S; Ko CW; Chau N; Chiang K; Kay MA; Barzel A; Venditti CP
    Hepatology; 2021 Jun; 73(6):2223-2237. PubMed ID: 32976669
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Tricarboxylic acid cycle enzyme activities in a mouse model of methylmalonic aciduria.
    Wongkittichote P; Cunningham G; Summar ML; Pumbo E; Forny P; Baumgartner MR; Chapman KA
    Mol Genet Metab; 2019 Dec; 128(4):444-451. PubMed ID: 31648943
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA).
    Chandler RJ; Venditti CP
    Mol Genet Metab; 2012 Nov; 107(3):617-9. PubMed ID: 23046887
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing.
    Venturoni LE; Chandler RJ; Liao J; Hoffmann V; Ramesh N; Gordo S; Chau N; Venditti CP
    Mol Genet Metab; 2022; 137(1-2):1-8. PubMed ID: 35868241
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle.
    Chandler RJ; Sloan J; Fu H; Tsai M; Stabler S; Allen R; Kaestner KH; Kazazian HH; Venditti CP
    BMC Med Genet; 2007 Oct; 8():64. PubMed ID: 17937813
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy.
    Chandler RJ; Venditti CP
    Mol Ther; 2010 Jan; 18(1):11-6. PubMed ID: 19861951
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Correction of methylmalonic aciduria in vivo using a codon-optimized lentiviral vector.
    Wong ES; McIntyre C; Peters HL; Ranieri E; Anson DS; Fletcher JM
    Hum Gene Ther; 2014 Jun; 25(6):529-38. PubMed ID: 24568291
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.
    Harrington EA; Sloan JL; Manoli I; Chandler RJ; Schneider M; McGuire PJ; Calcedo R; Wilson JM; Venditti CP
    Hum Gene Ther; 2016 May; 27(5):345-53. PubMed ID: 26790480
    [TBL] [Abstract][Full Text] [Related]  

  • 12. TAT-MTS-MCM fusion proteins reduce MMA levels and improve mitochondrial activity and liver function in MCM-deficient cells.
    Erlich-Hadad T; Hadad R; Feldman A; Greif H; Lictenstein M; Lorberboum-Galski H
    J Cell Mol Med; 2018 Mar; 22(3):1601-1613. PubMed ID: 29265583
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes.
    Chandler RJ; Tsai MS; Dorko K; Sloan J; Korson M; Freeman R; Strom S; Venditti CP
    BMC Med Genet; 2007 Apr; 8():24. PubMed ID: 17470278
    [TBL] [Abstract][Full Text] [Related]  

  • 14. New insights into the pathophysiology of methylmalonic acidemia.
    Head PE; Meier JL; Venditti CP
    J Inherit Metab Dis; 2023 May; 46(3):436-449. PubMed ID: 37078237
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Aberrant methylmalonylation underlies methylmalonic acidemia and is attenuated by an engineered sirtuin.
    Head PE; Myung S; Chen Y; Schneller JL; Wang C; Duncan N; Hoffman P; Chang D; Gebremariam A; Gucek M; Manoli I; Venditti CP
    Sci Transl Med; 2022 May; 14(646):eabn4772. PubMed ID: 35613279
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Systematic literature review and meta-analysis on the epidemiology of methylmalonic acidemia (MMA) with a focus on MMA caused by methylmalonyl-CoA mutase (mut) deficiency.
    Almási T; Guey LT; Lukacs C; Csetneki K; Vokó Z; Zelei T
    Orphanet J Rare Dis; 2019 Apr; 14(1):84. PubMed ID: 31023387
    [TBL] [Abstract][Full Text] [Related]  

  • 17. FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.
    Manoli I; Sysol JR; Epping MW; Li L; Wang C; Sloan JL; Pass A; Gagné J; Ktena YP; Li L; Trivedi NS; Ouattara B; Zerfas PM; Hoffmann V; Abu-Asab M; Tsokos MG; Kleiner DE; Garone C; Cusmano-Ozog K; Enns GM; Vernon HJ; Andersson HC; Grunewald S; Elkahloun AG; Girard CL; Schnermann J; DiMauro S; Andres-Mateos E; Vandenberghe LH; Chandler RJ; Venditti CP
    JCI Insight; 2018 Dec; 3(23):. PubMed ID: 30518688
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Biochemical and anaplerotic applications of in vitro models of propionic acidemia and methylmalonic acidemia using patient-derived primary hepatocytes.
    Collado MS; Armstrong AJ; Olson M; Hoang SA; Day N; Summar M; Chapman KA; Reardon J; Figler RA; Wamhoff BR
    Mol Genet Metab; 2020 Jul; 130(3):183-196. PubMed ID: 32451238
    [TBL] [Abstract][Full Text] [Related]  

  • 19. A novel small molecule approach for the treatment of propionic and methylmalonic acidemias.
    Armstrong AJ; Collado MS; Henke BR; Olson MW; Hoang SA; Hamilton CA; Pourtaheri TD; Chapman KA; Summar MM; Johns BA; Wamhoff BR; Reardon JE; Figler RA
    Mol Genet Metab; 2021 May; 133(1):71-82. PubMed ID: 33741272
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Treatment of a methylmalonyl-CoA mutase stopcodon mutation.
    Buck NE; Wood LR; Hamilton NJ; Bennett MJ; Peters HL
    Biochem Biophys Res Commun; 2012 Nov; 427(4):753-7. PubMed ID: 23041189
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 20.