200 related articles for article (PubMed ID: 38339118)
1. AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of
Sladen PE; Naeem A; Adefila-Ideozu T; Vermeule T; Busson SL; Michaelides M; Naylor S; Forbes A; Lane A; Georgiadis A
Int J Mol Sci; 2024 Feb; 25(3):. PubMed ID: 38339118
[TBL] [Abstract][Full Text] [Related]
2. Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa.
Fischer MD; McClements ME; Martinez-Fernandez de la Camara C; Bellingrath JS; Dauletbekov D; Ramsden SC; Hickey DG; Barnard AR; MacLaren RE
Mol Ther; 2017 Aug; 25(8):1854-1865. PubMed ID: 28549772
[TBL] [Abstract][Full Text] [Related]
3. Gelsolin dysfunction causes photoreceptor loss in induced pluripotent cell and animal retinitis pigmentosa models.
Megaw R; Abu-Arafeh H; Jungnickel M; Mellough C; Gurniak C; Witke W; Zhang W; Khanna H; Mill P; Dhillon B; Wright AF; Lako M; Ffrench-Constant C
Nat Commun; 2017 Aug; 8(1):271. PubMed ID: 28814713
[TBL] [Abstract][Full Text] [Related]
4. Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Song C; Conlon TJ; Deng WT; Coleman KE; Zhu P; Plummer C; Mandapati S; Van Hoosear M; Green KB; Sonnentag P; Sharma AK; Timmers A; Robinson PM; Knop DR; Hauswirth WW; Chulay JD; Shearman MS; Ye GJ
Hum Gene Ther Clin Dev; 2018 Dec; 29(4):188-197. PubMed ID: 30280954
[TBL] [Abstract][Full Text] [Related]
5. Loss of RPGR glutamylation underlies the pathogenic mechanism of retinal dystrophy caused by TTLL5 mutations.
Sun X; Park JH; Gumerson J; Wu Z; Swaroop A; Qian H; Roll-Mecak A; Li T
Proc Natl Acad Sci U S A; 2016 May; 113(21):E2925-34. PubMed ID: 27162334
[TBL] [Abstract][Full Text] [Related]
6. A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration.
Wu Z; Hiriyanna S; Qian H; Mookherjee S; Campos MM; Gao C; Fariss R; Sieving PA; Li T; Colosi P; Swaroop A
Hum Mol Genet; 2015 Jul; 24(14):3956-70. PubMed ID: 25877300
[TBL] [Abstract][Full Text] [Related]
7. Dose Range Finding Studies with Two
Song C; Dufour VL; Cideciyan AV; Ye GJ; Swider M; Newmark JA; Timmers AM; Robinson PM; Knop DR; Chulay JD; Jacobson SG; Aguirre GD; Beltran WA; Shearman MS
Hum Gene Ther; 2020 Jul; 31(13-14):743-755. PubMed ID: 32414297
[TBL] [Abstract][Full Text] [Related]
8. Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Beltran WA; Cideciyan AV; Boye SE; Ye GJ; Iwabe S; Dufour VL; Marinho LF; Swider M; Kosyk MS; Sha J; Boye SL; Peterson JJ; Witherspoon CD; Alexander JJ; Ying GS; Shearman MS; Chulay JD; Hauswirth WW; Gamlin PD; Jacobson SG; Aguirre GD
Mol Ther; 2017 Aug; 25(8):1866-1880. PubMed ID: 28566226
[TBL] [Abstract][Full Text] [Related]
9. Rd9 is a naturally occurring mouse model of a common form of retinitis pigmentosa caused by mutations in RPGR-ORF15.
Thompson DA; Khan NW; Othman MI; Chang B; Jia L; Grahek G; Wu Z; Hiriyanna S; Nellissery J; Li T; Khanna H; Colosi P; Swaroop A; Heckenlively JR
PLoS One; 2012; 7(5):e35865. PubMed ID: 22563472
[TBL] [Abstract][Full Text] [Related]
10. Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized
Dufour VL; Cideciyan AV; Ye GJ; Song C; Timmers A; Habecker PL; Pan W; Weinstein NM; Swider M; Durham AC; Ying GS; Robinson PM; Jacobson SG; Knop DR; Chulay JD; Shearman MS; Aguirre GD; Beltran WA
Hum Gene Ther; 2020 Feb; 31(3-4):253-267. PubMed ID: 31910043
[TBL] [Abstract][Full Text] [Related]
11. Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.
Mookherjee S; Hiriyanna S; Kaneshiro K; Li L; Li Y; Li W; Qian H; Li T; Khanna H; Colosi P; Swaroop A; Wu Z
Hum Mol Genet; 2015 Nov; 24(22):6446-58. PubMed ID: 26358772
[TBL] [Abstract][Full Text] [Related]
12. A single, abbreviated RPGR-ORF15 variant reconstitutes RPGR function in vivo.
Hong DH; Pawlyk BS; Adamian M; Sandberg MA; Li T
Invest Ophthalmol Vis Sci; 2005 Feb; 46(2):435-41. PubMed ID: 15671266
[TBL] [Abstract][Full Text] [Related]
13. Development of a Molecularly Stable Gene Therapy Vector for the Treatment of
Giacalone JC; Andorf JL; Zhang Q; Burnight ER; Ochoa D; Reutzel AJ; Collins MM; Sheffield VC; Mullins RF; Han IC; Stone EM; Tucker BA
Hum Gene Ther; 2019 Aug; 30(8):967-974. PubMed ID: 31106594
[TBL] [Abstract][Full Text] [Related]
14. Molecular Strategies for RPGR Gene Therapy.
Cehajic Kapetanovic J; McClements ME; Martinez-Fernandez de la Camara C; MacLaren RE
Genes (Basel); 2019 Sep; 10(9):. PubMed ID: 31487940
[TBL] [Abstract][Full Text] [Related]
15. Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa.
Martinez-Fernandez de la Camara C; Cehajic-Kapetanovic J; MacLaren RE
Expert Opin Emerg Drugs; 2022 Dec; 27(4):431-443. PubMed ID: 36562395
[TBL] [Abstract][Full Text] [Related]
16. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.
Beltran WA; Cideciyan AV; Lewin AS; Iwabe S; Khanna H; Sumaroka A; Chiodo VA; Fajardo DS; Román AJ; Deng WT; Swider M; Alemán TS; Boye SL; Genini S; Swaroop A; Hauswirth WW; Jacobson SG; Aguirre GD
Proc Natl Acad Sci U S A; 2012 Feb; 109(6):2132-7. PubMed ID: 22308428
[TBL] [Abstract][Full Text] [Related]
17. Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids.
Lane A; Jovanovic K; Shortall C; Ottaviani D; Panes AB; Schwarz N; Guarascio R; Hayes MJ; Palfi A; Chadderton N; Farrar GJ; Hardcastle AJ; Cheetham ME
Stem Cell Reports; 2020 Jul; 15(1):67-79. PubMed ID: 32531192
[TBL] [Abstract][Full Text] [Related]
18. Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing.
Gumerson JD; Alsufyani A; Yu W; Lei J; Sun X; Dong L; Wu Z; Li T
Gene Ther; 2022 Feb; 29(1-2):81-93. PubMed ID: 34257417
[TBL] [Abstract][Full Text] [Related]
19. Gene Correction Reverses Ciliopathy and Photoreceptor Loss in iPSC-Derived Retinal Organoids from Retinitis Pigmentosa Patients.
Deng WL; Gao ML; Lei XL; Lv JN; Zhao H; He KW; Xia XX; Li LY; Chen YC; Li YP; Pan D; Xue T; Jin ZB
Stem Cell Reports; 2018 Apr; 10(4):1267-1281. PubMed ID: 29526738
[TBL] [Abstract][Full Text] [Related]
20. Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa.
Pawlyk BS; Bulgakov OV; Sun X; Adamian M; Shu X; Smith AJ; Berson EL; Ali RR; Khani S; Wright AF; Sandberg MA; Li T
Gene Ther; 2016 Feb; 23(2):196-204. PubMed ID: 26348595
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]