BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

147 related articles for article (PubMed ID: 31479430)

  • 1. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.
    Klein AF; Varela MA; Arandel L; Holland A; Naouar N; Arzumanov A; Seoane D; Revillod L; Bassez G; Ferry A; Jauvin D; Gourdon G; Puymirat J; Gait MJ; Furling D; Wood MJ
    J Clin Invest; 2019 Nov; 129(11):4739-4744. PubMed ID: 31479430
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Splicing biomarkers of disease severity in myotonic dystrophy.
    Nakamori M; Sobczak K; Puwanant A; Welle S; Eichinger K; Pandya S; Dekdebrun J; Heatwole CR; McDermott MP; Chen T; Cline M; Tawil R; Osborne RJ; Wheeler TM; Swanson MS; Moxley RT; Thornton CA
    Ann Neurol; 2013 Dec; 74(6):862-72. PubMed ID: 23929620
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide.
    Yeoh YQ; Amin A; Cuic B; Tomas D; Turner BJ; Shabanpoor F
    Biomed Pharmacother; 2024 Jun; 175():116737. PubMed ID: 38749176
    [TBL] [Abstract][Full Text] [Related]  

  • 4. CaMKIIβ deregulation contributes to neuromuscular junction destabilization in Myotonic Dystrophy type I.
    Falcetta D; Quirim S; Cocchiararo I; Chabry F; Théodore M; Stiefvater A; Lin S; Tintignac L; Ivanek R; Kinter J; Rüegg MA; Sinnreich M; Castets P
    Skelet Muscle; 2024 May; 14(1):11. PubMed ID: 38769542
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Immortalized human myotonic dystrophy type 1 muscle cell lines to address patient heterogeneity.
    Núñez-Manchón J; Capó J; Martínez-Piñeiro A; Juanola E; Pesovic J; Mosqueira-Martín L; González-Imaz K; Maestre-Mora P; Odria R; Savic-Pavicevic D; Vallejo-Illarramendi A; Mamchaoui K; Bigot A; Mouly V; Suelves M; Nogales-Gadea G
    iScience; 2024 Jun; 27(6):109930. PubMed ID: 38832025
    [TBL] [Abstract][Full Text] [Related]  

  • 6. RNA therapeutics: beyond RNA interference and antisense oligonucleotides.
    Kole R; Krainer AR; Altman S
    Nat Rev Drug Discov; 2012 Jan; 11(2):125-40. PubMed ID: 22262036
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides.
    Wolfe JM; Fadzen CM; Holden RL; Yao M; Hanson GJ; Pentelute BL
    Angew Chem Int Ed Engl; 2018 Apr; 57(17):4756-4759. PubMed ID: 29479836
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Therapeutic potential of oleic acid supplementation in myotonic dystrophy muscle cell models.
    Moreno N; Sabater-Arcis M; Sevilla T; Alonso MP; Ohana J; Bargiela A; Artero R
    Biol Res; 2024 May; 57(1):29. PubMed ID: 38760841
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery.
    Wolfe JM; Fadzen CM; Choo ZN; Holden RL; Yao M; Hanson GJ; Pentelute BL
    ACS Cent Sci; 2018 Apr; 4(4):512-520. PubMed ID: 29721534
    [TBL] [Abstract][Full Text] [Related]  

  • 10. The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
    Batra R; Nelles DA; Roth DM; Krach F; Nutter CA; Tadokoro T; Thomas JD; Sznajder ŁJ; Blue SM; Gutierrez HL; Liu P; Aigner S; Platoshyn O; Miyanohara A; Marsala M; Swanson MS; Yeo GW
    Nat Biomed Eng; 2021 Feb; 5(2):157-168. PubMed ID: 32929188
    [TBL] [Abstract][Full Text] [Related]  

  • 11. The Dimeric Form of 1,3-Diaminoisoquinoline Derivative Rescued the Mis-splicing of Atp2a1 and Clcn1 Genes in Myotonic Dystrophy Type 1 Mouse Model.
    Matsumoto J; Nakamori M; Okamoto T; Murata A; Dohno C; Nakatani K
    Chemistry; 2020 Nov; 26(63):14305-14309. PubMed ID: 32449537
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Advances in oligonucleotide drug delivery.
    Roberts TC; Langer R; Wood MJA
    Nat Rev Drug Discov; 2020 Oct; 19(10):673-694. PubMed ID: 32782413
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Systemic therapy in an RNA toxicity mouse model with an antisense oligonucleotide therapy targeting a non-CUG sequence within the DMPK 3'UTR RNA.
    Yadava RS; Yu Q; Mandal M; Rigo F; Bennett CF; Mahadevan MS
    Hum Mol Genet; 2020 Jun; 29(9):1440-1453. PubMed ID: 32242217
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in vitro and in vivo models.
    Bargiela A; Sabater-Arcis M; Espinosa-Espinosa J; Zulaica M; Lopez de Munain A; Artero R
    Proc Natl Acad Sci U S A; 2019 Dec; 116(50):25203-25213. PubMed ID: 31754023
    [TBL] [Abstract][Full Text] [Related]  

  • 15. AAV9-DOK7 gene therapy reduces disease severity in Smn
    Kaifer KA; Villalón E; Smith CE; Simon ME; Marquez J; Hopkins AE; Morcos TI; Lorson CL
    Biochem Biophys Res Commun; 2020 Sep; 530(1):107-114. PubMed ID: 32828271
    [TBL] [Abstract][Full Text] [Related]  

  • 16. AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria.
    Richards DY; Winn SR; Dudley S; Nygaard S; Mighell TL; Grompe M; Harding CO
    Mol Ther Methods Clin Dev; 2020 Jun; 17():234-245. PubMed ID: 31970201
    [TBL] [Abstract][Full Text] [Related]  

  • 17. A mouse model for the cystic fibrosis delta F508 mutation.
    van Doorninck JH; French PJ; Verbeek E; Peters RH; Morreau H; Bijman J; Scholte BJ
    EMBO J; 1995 Sep; 14(18):4403-11. PubMed ID: 7556083
    [TBL] [Abstract][Full Text] [Related]  

  • 18. The hallmarks of myotonic dystrophy type 1 muscle dysfunction.
    Ozimski LL; Sabater-Arcis M; Bargiela A; Artero R
    Biol Rev Camb Philos Soc; 2021 Apr; 96(2):716-730. PubMed ID: 33269537
    [TBL] [Abstract][Full Text] [Related]  

  • 19. AON-induced splice-switching and DMPK pre-mRNA degradation as potential therapeutic approaches for Myotonic Dystrophy type 1.
    Stepniak-Konieczna E; Konieczny P; Cywoniuk P; Dluzewska J; Sobczak K
    Nucleic Acids Res; 2020 Mar; 48(5):2531-2543. PubMed ID: 31965181
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.
    Marsh S; Hanson B; Wood MJA; Varela MA; Roberts TC
    Mol Ther; 2020 Dec; 28(12):2527-2539. PubMed ID: 33171139
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 8.