BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

232 related articles for article (PubMed ID: 31611143)

  • 1. Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles.
    Sun D; Schur RM; Sears AE; Gao SQ; Vaidya A; Sun W; Maeda A; Kern T; Palczewski K; Lu ZR
    Mol Ther; 2020 Jan; 28(1):293-303. PubMed ID: 31611143
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse model.
    Sun D; Sun W; Gao SQ; Wei C; Naderi A; Schilb AL; Scheidt J; Lee S; Kern TS; Palczewski K; Lu ZR
    J Control Release; 2021 Feb; 330():329-340. PubMed ID: 33358976
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Intravitreal Delivery of PEGylated-ECO Plasmid DNA Nanoparticles for Gene Therapy of Stargardt Disease.
    Sun D; Sun W; Gao SQ; Lehrer J; Wang H; Hall R; Lu ZR
    Pharm Res; 2024 Apr; 41(4):807-817. PubMed ID: 38443629
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Dual
    Dyka FM; Molday LL; Chiodo VA; Molday RS; Hauswirth WW
    Hum Gene Ther; 2019 Nov; 30(11):1361-1370. PubMed ID: 31418294
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Expression of ABCA4 in the retinal pigment epithelium and its implications for Stargardt macular degeneration.
    Lenis TL; Hu J; Ng SY; Jiang Z; Sarfare S; Lloyd MB; Esposito NJ; Samuel W; Jaworski C; Bok D; Finnemann SC; Radeke MJ; Redmond TM; Travis GH; Radu RA
    Proc Natl Acad Sci U S A; 2018 Nov; 115(47):E11120-E11127. PubMed ID: 30397118
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Effective gene therapy of Stargardt disease with PEG-ECO/
    Sun D; Sun W; Gao SQ; Lehrer J; Naderi A; Wei C; Lee S; Schilb AL; Scheidt J; Hall RC; Traboulsi EI; Palczewski K; Lu ZR
    Mol Ther Nucleic Acids; 2022 Sep; 29():823-835. PubMed ID: 36159595
    [TBL] [Abstract][Full Text] [Related]  

  • 7. DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.
    Han Z; Conley SM; Makkia RS; Cooper MJ; Naash MI
    J Clin Invest; 2012 Sep; 122(9):3221-6. PubMed ID: 22886305
    [TBL] [Abstract][Full Text] [Related]  

  • 8. An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult
    McClements ME; Barnard AR; Singh MS; Charbel Issa P; Jiang Z; Radu RA; MacLaren RE
    Hum Gene Ther; 2019 May; 30(5):590-600. PubMed ID: 30381971
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Localization and functional characterization of the p.Asn965Ser (N965S) ABCA4 variant in mice reveal pathogenic mechanisms underlying Stargardt macular degeneration.
    Molday LL; Wahl D; Sarunic MV; Molday RS
    Hum Mol Genet; 2018 Jan; 27(2):295-306. PubMed ID: 29145636
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Gene therapy for Stargardt disease associated with ABCA4 gene.
    Han Z; Conley SM; Naash MI
    Adv Exp Med Biol; 2014; 801():719-24. PubMed ID: 24664763
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy.
    Kong J; Kim SR; Binley K; Pata I; Doi K; Mannik J; Zernant-Rajang J; Kan O; Iqball S; Naylor S; Sparrow JR; Gouras P; Allikmets R
    Gene Ther; 2008 Oct; 15(19):1311-20. PubMed ID: 18463687
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Impaired cathepsin D in retinal pigment epithelium cells mediates Stargardt disease pathogenesis.
    Ng ESY; Hu J; Jiang Z; Radu RA
    FASEB J; 2024 Jun; 38(11):e23720. PubMed ID: 38837708
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Fundus autofluorescence in the Abca4(-/-) mouse model of Stargardt disease--correlation with accumulation of A2E, retinal function, and histology.
    Charbel Issa P; Barnard AR; Singh MS; Carter E; Jiang Z; Radu RA; Schraermeyer U; MacLaren RE
    Invest Ophthalmol Vis Sci; 2013 Aug; 54(8):5602-12. PubMed ID: 23761084
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Split AAV8 Mediated ABCA4 Expression for Gene Therapy of Mouse Stargardt Disease (STGD1).
    Li R; Jing Q; She K; Wang Q; Jin X; Zhao Q; Su J; Song L; Fu J; Wu X; Xu Q; Lu F; Wei Y; Yang Y
    Hum Gene Ther; 2023 Jul; 34(13-14):616-628. PubMed ID: 37227014
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Gene Therapy of ABCA4-Associated Diseases.
    Auricchio A; Trapani I; Allikmets R
    Cold Spring Harb Perspect Med; 2015 Jan; 5(5):a017301. PubMed ID: 25573774
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Structure and function of ABCA4 and its role in the visual cycle and Stargardt macular degeneration.
    Molday RS; Garces FA; Scortecci JF; Molday LL
    Prog Retin Eye Res; 2022 Jul; 89():101036. PubMed ID: 34954332
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Functional Characterization of ABCA4 Missense Variants Linked to Stargardt Macular Degeneration.
    Garces FA; Scortecci JF; Molday RS
    Int J Mol Sci; 2020 Dec; 22(1):. PubMed ID: 33375396
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Cell-autonomous lipid-handling defects in Stargardt iPSC-derived retinal pigment epithelium cells.
    Farnoodian M; Bose D; Khristov V; Susaimanickam PJ; Maddileti S; Mariappan I; Abu-Asab M; Campos M; Villasmil R; Wan Q; Maminishkis A; McGaughey D; Barone F; Gundry RL; Riordon DR; Boheler KR; Sharma R; Bharti K
    Stem Cell Reports; 2022 Nov; 17(11):2438-2450. PubMed ID: 36306781
    [TBL] [Abstract][Full Text] [Related]  

  • 19. All-trans retinal levels and formation of lipofuscin precursors after bleaching in rod photoreceptors from wild type and Abca4
    Adler L; Chen C; Koutalos Y
    Exp Eye Res; 2017 Feb; 155():121-127. PubMed ID: 28219732
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Complement modulation in the retinal pigment epithelium rescues photoreceptor degeneration in a mouse model of Stargardt disease.
    Lenis TL; Sarfare S; Jiang Z; Lloyd MB; Bok D; Radu RA
    Proc Natl Acad Sci U S A; 2017 Apr; 114(15):3987-3992. PubMed ID: 28348233
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 12.