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Journal Abstract Search

456 related items for PubMed ID: 25030840

  • 1.
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  • 2. Pharmacological Amelioration of Cone Survival and Vision in a Mouse Model for Leber Congenital Amaurosis.
    Li S, Samardzija M, Yang Z, Grimm C, Jin M.
    J Neurosci; 2016 May 25; 36(21):5808-19. PubMed ID: 27225770
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  • 3. Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT).
    Scholl HP, Moore AT, Koenekoop RK, Wen Y, Fishman GA, van den Born LI, Bittner A, Bowles K, Fletcher EC, Collison FT, Dagnelie G, Degli Eposti S, Michaelides M, Saperstein DA, Schuchard RA, Barnes C, Zein W, Zobor D, Birch DG, Mendola JD, Zrenner E, RET IRD 01 Study Group.
    PLoS One; 2015 May 25; 10(12):e0143846. PubMed ID: 26656277
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  • 4. Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.
    Batten ML, Imanishi Y, Tu DC, Doan T, Zhu L, Pang J, Glushakova L, Moise AR, Baehr W, Van Gelder RN, Hauswirth WW, Rieke F, Palczewski K.
    PLoS Med; 2005 Nov 25; 2(11):e333. PubMed ID: 16250670
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  • 5. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
    Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT.
    Ophthalmology; 2016 Jul 25; 123(7):1606-20. PubMed ID: 27102010
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  • 6. Intervisit variability of visual parameters in Leber congenital amaurosis caused by RPE65 mutations.
    Roman AJ, Cideciyan AV, Schwartz SB, Olivares MB, Heon E, Jacobson SG.
    Invest Ophthalmol Vis Sci; 2013 Feb 15; 54(2):1378-83. PubMed ID: 23341016
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  • 8. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
    Jacobson SG, Cideciyan AV, Ratnakaram R, Heon E, Schwartz SB, Roman AJ, Peden MC, Aleman TS, Boye SL, Sumaroka A, Conlon TJ, Calcedo R, Pang JJ, Erger KE, Olivares MB, Mullins CL, Swider M, Kaushal S, Feuer WJ, Iannaccone A, Fishman GA, Stone EM, Byrne BJ, Hauswirth WW.
    Arch Ophthalmol; 2012 Jan 15; 130(1):9-24. PubMed ID: 21911650
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  • 12. Nature of the visual loss in observers with Leber's congenital amaurosis caused by specific mutations in RPE65.
    Ripamonti C, Henning GB, Ali RR, Bainbridge JW, Robbie SJ, Sundaram V, Luong VA, van den Born LI, Casteels I, de Ravel TJ, Moore AT, Stockman A.
    Invest Ophthalmol Vis Sci; 2014 Sep 25; 55(10):6817-28. PubMed ID: 25257057
    [Abstract] [Full Text] [Related]

  • 13. Longitudinal and cross-sectional study of patients with early-onset severe retinal dystrophy associated with RPE65 mutations.
    Paunescu K, Wabbels B, Preising MN, Lorenz B.
    Graefes Arch Clin Exp Ophthalmol; 2005 May 25; 243(5):417-26. PubMed ID: 15565294
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  • 14. Cone-rod dystrophy caused by a novel homozygous RPE65 mutation in Leber congenital amaurosis.
    Jakobsson C, Othman IS, Munier FL, Schorderet DF, Abouzeid H.
    Klin Monbl Augenheilkd; 2014 Apr 25; 231(4):405-10. PubMed ID: 24771178
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  • 15. Evaluation of 9-cis-retinyl acetate therapy in Rpe65-/- mice.
    Maeda T, Maeda A, Casadesus G, Palczewski K, Margaron P.
    Invest Ophthalmol Vis Sci; 2009 Sep 25; 50(9):4368-78. PubMed ID: 19407008
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  • 16. Early onset retinal dystrophy due to mutations in LRAT: molecular analysis and detailed phenotypic study.
    Dev Borman A, Ocaka LA, Mackay DS, Ripamonti C, Henderson RH, Moradi P, Hall G, Black GC, Robson AG, Holder GE, Webster AR, Fitzke F, Stockman A, Moore AT.
    Invest Ophthalmol Vis Sci; 2012 Jun 22; 53(7):3927-38. PubMed ID: 22570351
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  • 17. Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis.
    Deng C, Zhao PY, Branham K, Schlegel D, Fahim AT, Jayasundera TK, Khan N, Besirli CG.
    Graefes Arch Clin Exp Ophthalmol; 2022 May 22; 260(5):1543-1550. PubMed ID: 35001204
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  • 18. [Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial].
    Chacón-Camacho ÓF, Zenteno JC.
    Gac Med Mex; 2017 May 22; 153(2):276-278. PubMed ID: 28474714
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  • 19. Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
    Le Meur G, Lebranchu P, Billaud F, Adjali O, Schmitt S, Bézieau S, Péréon Y, Valabregue R, Ivan C, Darmon C, Moullier P, Rolling F, Weber M.
    Mol Ther; 2018 Jan 03; 26(1):256-268. PubMed ID: 29033008
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  • 20. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
    Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, Roman AJ, Olivares MB, Schwartz SB, Komáromy AM, Hauswirth WW, Aguirre GD.
    Proc Natl Acad Sci U S A; 2013 Feb 05; 110(6):E517-25. PubMed ID: 23341635
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