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 *

241 related articles for article (PubMed ID: 31179759)

  • 1. A New Era for Rare Genetic Diseases: Messenger RNA Therapy.
    Martini PGV; Guey LT
    Hum Gene Ther; 2019 Oct; 30(10):1180-1189. PubMed ID: 31179759
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Messenger RNA therapy for rare genetic metabolic diseases.
    Berraondo P; Martini PGV; Avila MA; Fontanellas A
    Gut; 2019 Jul; 68(7):1323-1330. PubMed ID: 30796097
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Systemic Administered mRNA as Therapy for Metabolic Diseases.
    Puy H; Deybach JC; Gouya L
    Trends Mol Med; 2019 Jan; 25(1):3-5. PubMed ID: 30528119
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Breaking the Barriers of Genetic and Metabolic Disorders.
    Büning H; Bosch F; Mingozzi F
    Hum Gene Ther; 2019 Oct; 30(10):1177-1179. PubMed ID: 31573872
    [No Abstract]   [Full Text] [Related]  

  • 6. mRNA therapies: Pioneering a new era in rare genetic disease treatment.
    Shen G; Liu J; Yang H; Xie N; Yang Y
    J Control Release; 2024 May; 369():696-721. PubMed ID: 38580137
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. 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]  

  • 9. Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria.
    Baek R; Coughlan K; Jiang L; Liang M; Ci L; Singh H; Zhang H; Kaushal N; Rajlic IL; Van L; Dimen R; Cavedon A; Yin L; Rice L; Frassetto A; Guey L; Finn P; Martini PGV
    Nat Commun; 2024 May; 15(1):3804. PubMed ID: 38714648
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Genetics and Gene Therapy of Anderson-Fabry Disease.
    Simonetta I; Tuttolomondo A; Di Chiara T; Miceli S; Vogiatzis D; Corpora F; Pinto A
    Curr Gene Ther; 2018; 18(2):96-106. PubMed ID: 29618309
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Current Status of Messenger RNA Delivery Systems.
    Stanton MG
    Nucleic Acid Ther; 2018 Jun; 28(3):158-165. PubMed ID: 29688817
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Messenger RNA as a personalized therapy: The moment of truth for rare metabolic diseases.
    Córdoba KM; Jericó D; Sampedro A; Jiang L; Iraburu MJ; Martini PGV; Berraondo P; Avila MA; Fontanellas A
    Int Rev Cell Mol Biol; 2022; 372():55-96. PubMed ID: 36064267
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.
    Pañeda A; Lopez-Franco E; Kaeppel C; Unzu C; Gil-Royo AG; D'Avola D; Beattie SG; Olagüe C; Ferrero R; Sampedro A; Mauleon I; Hermening S; Salmon F; Benito A; Gavira JJ; Cornet ME; del Mar Municio M; von Kalle C; Petry H; Prieto J; Schmidt M; Fontanellas A; González-Aseguinolaza G
    Hum Gene Ther; 2013 Dec; 24(12):1007-17. PubMed ID: 24070415
    [TBL] [Abstract][Full Text] [Related]  

  • 14. RNA delivery biomaterials for the treatment of genetic and rare diseases.
    Zhao W; Hou X; Vick OG; Dong Y
    Biomaterials; 2019 Oct; 217():119291. PubMed ID: 31255978
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Helper-dependent adenoviral liver gene therapy protects against induced attacks and corrects protein folding stress in acute intermittent porphyria mice.
    Unzu C; Sampedro A; Mauleón I; González-Aparicio M; Enríquez de Salamanca R; Prieto J; Aragón T; Fontanellas A
    Hum Mol Genet; 2013 Jul; 22(14):2929-40. PubMed ID: 23562909
    [TBL] [Abstract][Full Text] [Related]  

  • 16. An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria.
    Serrano-Mendioroz I; Sampedro A; Alegre M; Enríquez de Salamanca R; Berraondo P; Fontanellas A
    Hum Gene Ther; 2018 Apr; 29(4):480-491. PubMed ID: 28990424
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Gene therapy for Fabry disease.
    Siatskas C; Medin JA
    J Inherit Metab Dis; 2001; 24 Suppl 2():25-41; discussion 11-2. PubMed ID: 11758676
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Fetal somatic gene therapy.
    Douar AM; Themis M; Coutelle C
    Mol Hum Reprod; 1996 Sep; 2(9):633-41. PubMed ID: 9239676
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Non-viral delivery of the porphobilinogen deaminase cDNA into a mouse model of acute intermittent porphyria.
    Johansson A; Nowak G; Möller C; Harper P
    Mol Genet Metab; 2004 May; 82(1):20-6. PubMed ID: 15110317
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Organic Nanoparticle-Based Combinatory Approaches for Gene Therapy.
    Singh BN; Prateeksha ; Gupta VK; Chen J; Atanasov AG
    Trends Biotechnol; 2017 Dec; 35(12):1121-1124. PubMed ID: 28818304
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.