176 related articles for article (PubMed ID: 29673930)
1. Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.
Song JY; Aravand P; Nikonov S; Leo L; Lyubarsky A; Bennicelli JL; Pan J; Wei Z; Shpylchak I; Herrera P; Bennett DJ; Commins N; Maguire AM; Pham J; den Hollander AI; Cremers FPM; Koenekoop RK; Roepman R; Nishina P; Zhou S; Pan W; Ying GS; Aleman TS; de Melo J; McNamara I; Sun J; Mills J; Bennett J
Mol Ther; 2018 Jun; 26(6):1581-1593. PubMed ID: 29673930
[TBL] [Abstract][Full Text] [Related]
2. Treatment Potential for LCA5-Associated Leber Congenital Amaurosis.
Uyhazi KE; Aravand P; Bell BA; Wei Z; Leo L; Serrano LW; Pearson DJ; Shpylchak I; Pham J; Vasireddy V; Bennett J; Aleman TS
Invest Ophthalmol Vis Sci; 2020 May; 61(5):30. PubMed ID: 32428231
[TBL] [Abstract][Full Text] [Related]
3. Knocking out lca5 in zebrafish causes cone-rod dystrophy due to impaired outer segment protein trafficking.
Qu Z; Yimer TA; Xie S; Wong F; Yu S; Liu X; Han S; Ma J; Lu Z; Hu X; Qin Y; Huang Y; Lv Y; Li J; Tang Z; Liu F; Liu M
Biochim Biophys Acta Mol Basis Dis; 2019 Oct; 1865(10):2694-2705. PubMed ID: 31348989
[TBL] [Abstract][Full Text] [Related]
4. Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.
Pawlyk BS; Smith AJ; Buch PK; Adamian M; Hong DH; Sandberg MA; Ali RR; Li T
Invest Ophthalmol Vis Sci; 2005 Sep; 46(9):3039-45. PubMed ID: 16123399
[TBL] [Abstract][Full Text] [Related]
5. Screening of a large cohort of leber congenital amaurosis and retinitis pigmentosa patients identifies novel LCA5 mutations and new genotype-phenotype correlations.
Mackay DS; Borman AD; Sui R; van den Born LI; Berson EL; Ocaka LA; Davidson AE; Heckenlively JR; Branham K; Ren H; Lopez I; Maria M; Azam M; Henkes A; Blokland E; Qamar R; Webster AR; Cremers FPM; Moore AT; Koenekoop RK; ; Andreasson S; de Baere E; Bennett J; Chader GJ; Berger W; Golovleva I; Greenberg J; den Hollander AI; Klaver CCW; Klevering BJ; Lorenz B; Preising MN; Ramsear R; Roberts L; Roepman R; Rohrschneider K; Wissinger B
Hum Mutat; 2013 Nov; 34(11):1537-1546. PubMed ID: 23946133
[TBL] [Abstract][Full Text] [Related]
6. Leber congenital amaurosis caused by Lebercilin (LCA5) mutation: retained photoreceptors adjacent to retinal disorganization.
Jacobson SG; Aleman TS; Cideciyan AV; Sumaroka A; Schwartz SB; Windsor EA; Swider M; Herrera W; Stone EM
Mol Vis; 2009 Jun; 15():1098-106. PubMed ID: 19503738
[TBL] [Abstract][Full Text] [Related]
7. Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis.
Aguirre GD; Cideciyan AV; Dufour VL; Ripolles-García A; Sudharsan R; Swider M; Nikonov R; Iwabe S; Boye SL; Hauswirth WW; Jacobson SG; Beltran WA
Mol Ther; 2021 Aug; 29(8):2456-2468. PubMed ID: 33781914
[TBL] [Abstract][Full Text] [Related]
8. Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis.
Li X; Li W; Dai X; Kong F; Zheng Q; Zhou X; Lü F; Chang B; Rohrer B; Hauswirth WW; Qu J; Pang JJ
Invest Ophthalmol Vis Sci; 2011 Jan; 52(1):7-15. PubMed ID: 21169527
[TBL] [Abstract][Full Text] [Related]
9. Mutations in LCA5, encoding the ciliary protein lebercilin, cause Leber congenital amaurosis.
den Hollander AI; Koenekoop RK; Mohamed MD; Arts HH; Boldt K; Towns KV; Sedmak T; Beer M; Nagel-Wolfrum K; McKibbin M; Dharmaraj S; Lopez I; Ivings L; Williams GA; Springell K; Woods CG; Jafri H; Rashid Y; Strom TM; van der Zwaag B; Gosens I; Kersten FF; van Wijk E; Veltman JA; Zonneveld MN; van Beersum SE; Maumenee IH; Wolfrum U; Cheetham ME; Ueffing M; Cremers FP; Inglehearn CF; Roepman R
Nat Genet; 2007 Jul; 39(7):889-95. PubMed ID: 17546029
[TBL] [Abstract][Full Text] [Related]
10. Identification of a novel splice-site mutation in the Lebercilin (LCA5) gene causing Leber congenital amaurosis.
Ramprasad VL; Soumittra N; Nancarrow D; Sen P; McKibbin M; Williams GA; Arokiasamy T; Lakshmipathy P; Inglehearn CF; Kumaramanickavel G
Mol Vis; 2008 Mar; 14():481-6. PubMed ID: 18334959
[TBL] [Abstract][Full Text] [Related]
11. Rescue of cone function in cone-only
Hanke-Gogokhia C; Chiodo VA; Hauswirth WW; Frederick JM; Baehr W
Mol Vis; 2018; 24():834-846. PubMed ID: 30713422
[TBL] [Abstract][Full Text] [Related]
12. Gene augmentation of LCA5-associated Leber congenital amaurosis ameliorates bulge region defects of the photoreceptor ciliary axoneme.
Faber S; Mercey O; Junger K; Garanto A; May-Simera H; Ueffing M; Collin RW; Boldt K; Guichard P; Hamel V; Roepman R
JCI Insight; 2023 May; 8(10):. PubMed ID: 37071472
[TBL] [Abstract][Full Text] [Related]
13. Involvement of LCA5 in Leber congenital amaurosis and retinitis pigmentosa in the Spanish population.
Corton M; Avila-Fernandez A; Vallespín E; López-Molina MI; Almoguera B; Martín-Garrido E; Tatu SD; Khan MI; Blanco-Kelly F; Riveiro-Alvarez R; Brión M; García-Sandoval B; Cremers FPM; Carracedo A; Ayuso C
Ophthalmology; 2014 Jan; 121(1):399-407. PubMed ID: 24144451
[TBL] [Abstract][Full Text] [Related]
14. Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and mice.
Boldt K; Mans DA; Won J; van Reeuwijk J; Vogt A; Kinkl N; Letteboer SJ; Hicks WL; Hurd RE; Naggert JK; Texier Y; den Hollander AI; Koenekoop RK; Bennett J; Cremers FP; Gloeckner CJ; Nishina PM; Roepman R; Ueffing M
J Clin Invest; 2011 Jun; 121(6):2169-80. PubMed ID: 21606596
[TBL] [Abstract][Full Text] [Related]
15. Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.
Ku CA; Chiodo VA; Boye SL; Goldberg AF; Li T; Hauswirth WW; Ramamurthy V
Hum Mol Genet; 2011 Dec; 20(23):4569-81. PubMed ID: 21880665
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.
Boye SL; Peshenko IV; Huang WC; Min SH; McDoom I; Kay CN; Liu X; Dyka FM; Foster TC; Umino Y; Karan S; Jacobson SG; Baehr W; Dizhoor A; Hauswirth WW; Boye SE
Hum Gene Ther; 2013 Feb; 24(2):189-202. PubMed ID: 23210611
[TBL] [Abstract][Full Text] [Related]
18. Gene therapy restores vision in a canine model of childhood blindness.
Acland GM; Aguirre GD; Ray J; Zhang Q; Aleman TS; Cideciyan AV; Pearce-Kelling SE; Anand V; Zeng Y; Maguire AM; Jacobson SG; Hauswirth WW; Bennett J
Nat Genet; 2001 May; 28(1):92-5. PubMed ID: 11326284
[TBL] [Abstract][Full Text] [Related]
19. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.
Jacobson SG; Boye SL; Aleman TS; Conlon TJ; Zeiss CJ; Roman AJ; Cideciyan AV; Schwartz SB; Komaromy AM; Doobrajh M; Cheung AY; Sumaroka A; Pearce-Kelling SE; Aguirre GD; Kaushal S; Maguire AM; Flotte TR; Hauswirth WW
Hum Gene Ther; 2006 Aug; 17(8):845-58. PubMed ID: 16942444
[TBL] [Abstract][Full Text] [Related]
20. RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.
Molday LL; Djajadi H; Yan P; Szczygiel L; Boye SL; Chiodo VA; Gregory-Evans K; Sarunic MV; Hauswirth WW; Molday RS
Hum Mol Genet; 2013 Oct; 22(19):3894-905. PubMed ID: 23740938
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]