205 related articles for article (PubMed ID: 24035979)
1. Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases.
Koirala A; Conley SM; Makkia R; Liu Z; Cooper MJ; Sparrow JR; Naash MI
J Control Release; 2013 Dec; 172(3):745-52. PubMed ID: 24035979
[TBL] [Abstract][Full Text] [Related]
2. S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA.
Koirala A; Makkia RS; Conley SM; Cooper MJ; Naash MI
Hum Mol Genet; 2013 Apr; 22(8):1632-42. PubMed ID: 23335596
[TBL] [Abstract][Full Text] [Related]
3. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.
Li W; Kong F; Li X; Dai X; Liu X; Zheng Q; Wu R; Zhou X; Lü F; Chang B; Li Q; Hauswirth WW; Qu J; Pang JJ
Mol Vis; 2009; 15():267-75. PubMed ID: 19190735
[TBL] [Abstract][Full Text] [Related]
4. Nanoparticle-mediated gene transfer specific to retinal pigment epithelial cells.
Koirala A; Makkia RS; Cooper MJ; Naash MI
Biomaterials; 2011 Dec; 32(35):9483-93. PubMed ID: 21885113
[TBL] [Abstract][Full Text] [Related]
5. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
Acland GM; Aguirre GD; Bennett J; Aleman TS; Cideciyan AV; Bennicelli J; Dejneka NS; Pearce-Kelling SE; Maguire AM; Palczewski K; Hauswirth WW; Jacobson SG
Mol Ther; 2005 Dec; 12(6):1072-82. PubMed ID: 16226919
[TBL] [Abstract][Full Text] [Related]
6. Episomal maintenance of S/MAR-containing non-viral vectors for RPE-based diseases.
Koirala A; Conley SM; Naash MI
Adv Exp Med Biol; 2014; 801():703-9. PubMed ID: 24664761
[TBL] [Abstract][Full Text] [Related]
7. The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.
Pierce EA; Bennett J
Cold Spring Harb Perspect Med; 2015 Jan; 5(9):a017285. PubMed ID: 25635059
[TBL] [Abstract][Full Text] [Related]
8. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis.
Pang JJ; Chang B; Kumar A; Nusinowitz S; Noorwez SM; Li J; Rani A; Foster TC; Chiodo VA; Doyle T; Li H; Malhotra R; Teusner JT; McDowell JH; Min SH; Li Q; Kaushal S; Hauswirth WW
Mol Ther; 2006 Mar; 13(3):565-72. PubMed ID: 16223604
[TBL] [Abstract][Full Text] [Related]
9. Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis.
Bemelmans AP; Kostic C; Crippa SV; Hauswirth WW; Lem J; Munier FL; Seeliger MW; Wenzel A; Arsenijevic Y
PLoS Med; 2006 Oct; 3(10):e347. PubMed ID: 17032058
[TBL] [Abstract][Full Text] [Related]
10. Rpe65 is a retinyl ester binding protein that presents insoluble substrate to the isomerase in retinal pigment epithelial cells.
Mata NL; Moghrabi WN; Lee JS; Bui TV; Radu RA; Horwitz J; Travis GH
J Biol Chem; 2004 Jan; 279(1):635-43. PubMed ID: 14532273
[TBL] [Abstract][Full Text] [Related]
11. Long-term effect of gene therapy on Leber's congenital amaurosis.
Bainbridge JW; Mehat MS; Sundaram V; Robbie SJ; Barker SE; Ripamonti C; Georgiadis A; Mowat FM; Beattie SG; Gardner PJ; Feathers KL; Luong VA; Yzer S; Balaggan K; Viswanathan A; de Ravel TJ; Casteels I; Holder GE; Tyler N; Fitzke FW; Weleber RG; Nardini M; Moore AT; Thompson DA; Petersen-Jones SM; Michaelides M; van den Born LI; Stockman A; Smith AJ; Rubin G; Ali RR
N Engl J Med; 2015 May; 372(20):1887-97. PubMed ID: 25938638
[TBL] [Abstract][Full Text] [Related]
12. RPE65 and the Accumulation of Retinyl Esters in Mouse Retinal Pigment Epithelium.
Sheridan C; Boyer NP; Crouch RK; Koutalos Y
Photochem Photobiol; 2017 May; 93(3):844-848. PubMed ID: 28500718
[TBL] [Abstract][Full Text] [Related]
13. 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; 36(21):5808-19. PubMed ID: 27225770
[TBL] [Abstract][Full Text] [Related]
14. Altered expression of β-galactosidase-1-like protein 3 (Glb1l3) in the retinal pigment epithelium (RPE)-specific 65-kDa protein knock-out mouse model of Leber's congenital amaurosis.
Le Carré J; Schorderet DF; Cottet S
Mol Vis; 2011; 17():1287-97. PubMed ID: 21633714
[TBL] [Abstract][Full Text] [Related]
15. Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.
Narfström K; Katz ML; Bragadottir R; Seeliger M; Boulanger A; Redmond TM; Caro L; Lai CM; Rakoczy PE
Invest Ophthalmol Vis Sci; 2003 Apr; 44(4):1663-72. PubMed ID: 12657607
[TBL] [Abstract][Full Text] [Related]
16. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Hauswirth WW; Aleman TS; Kaushal S; Cideciyan AV; Schwartz SB; Wang L; Conlon TJ; Boye SL; Flotte TR; Byrne BJ; Jacobson SG
Hum Gene Ther; 2008 Oct; 19(10):979-90. PubMed ID: 18774912
[TBL] [Abstract][Full Text] [Related]
17. Bax-induced apoptosis in Leber's congenital amaurosis: a dual role in rod and cone degeneration.
Hamann S; Schorderet DF; Cottet S
PLoS One; 2009 Aug; 4(8):e6616. PubMed ID: 19672311
[TBL] [Abstract][Full Text] [Related]
18. Unlocking therapeutic potential: dual gene therapy for ameliorating the disease phenotypes in a mouse model of
Liu Y; Tai J; Yu C; Xu D; Xiao D; Pang J
Front Med (Lausanne); 2023; 10():1291795. PubMed ID: 38264046
[TBL] [Abstract][Full Text] [Related]
19. Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level.
Udry F; Decembrini S; Gamm DM; Déglon N; Kostic C; Arsenijevic Y
Sci Rep; 2020 Jun; 10(1):8890. PubMed ID: 32483256
[TBL] [Abstract][Full Text] [Related]
20. Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.
Roman AJ; Boye SL; Aleman TS; Pang JJ; McDowell JH; Boye SE; Cideciyan AV; Jacobson SG; Hauswirth WW
Mol Vis; 2007 Sep; 13():1701-10. PubMed ID: 17960108
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
