309 related articles for article (PubMed ID: 31910043)
21. Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin.
Ye GJ; Budzynski E; Sonnentag P; Miller PE; Sharma AK; Ver Hoeve JN; Howard K; Knop DR; Neuringer M; McGill T; Stoddard J; Chulay JD
Hum Gene Ther Clin Dev; 2015 Sep; 26(3):165-76. PubMed ID: 26390090
[TBL] [Abstract][Full Text] [Related]
22. Phenotypic high myopia in X-linked retinitis pigmentosa secondary to a novel mutation in the RPGR gene.
Sanchez Tocino H; Diez Montero C; Villanueva Gómez A; Lobo Valentin R; Montero-Moreno JA
Ophthalmic Genet; 2019 Apr; 40(2):170-176. PubMed ID: 31033374
[TBL] [Abstract][Full Text] [Related]
23. Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.
Cehajic-Kapetanovic J; Xue K; Martinez-Fernandez de la Camara C; Nanda A; Davies A; Wood LJ; Salvetti AP; Fischer MD; Aylward JW; Barnard AR; Jolly JK; Luo E; Lujan BJ; Ong T; Girach A; Black GCM; Gregori NZ; Davis JL; Rosa PR; Lotery AJ; Lam BL; Stanga PE; MacLaren RE
Nat Med; 2020 Mar; 26(3):354-359. PubMed ID: 32094925
[TBL] [Abstract][Full Text] [Related]
24. Advancing Gene Therapy for PDE6A Retinitis Pigmentosa.
Petersen-Jones SM; Occelli LM; Biel M; Michalakis S
Adv Exp Med Biol; 2019; 1185():103-107. PubMed ID: 31884596
[TBL] [Abstract][Full Text] [Related]
25. Clinical characteristics of high myopia in female carriers of pathogenic
Tran M; Kolesnikova M; Kim AH; Kowal T; Ning K; Mahajan VB; Tsang SH; Sun Y
Ophthalmic Genet; 2023 Jun; 44(3):295-303. PubMed ID: 36017691
[TBL] [Abstract][Full Text] [Related]
26. Mapping of X-linked progressive retinal atrophy (XLPRA), the canine homolog of retinitis pigmentosa 3 (RP3).
Zeiss CJ; Ray K; Acland GM; Aguirre GD
Hum Mol Genet; 2000 Mar; 9(4):531-7. PubMed ID: 10699176
[TBL] [Abstract][Full Text] [Related]
27. 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]
28. A frameshift mutation in RPGR exon ORF15 causes photoreceptor degeneration and inner retina remodeling in a model of X-linked retinitis pigmentosa.
Beltran WA; Hammond P; Acland GM; Aguirre GD
Invest Ophthalmol Vis Sci; 2006 Apr; 47(4):1669-81. PubMed ID: 16565408
[TBL] [Abstract][Full Text] [Related]
29. In Vivo CRISPR/Cas9-Mediated Genome Editing Mitigates Photoreceptor Degeneration in a Mouse Model of X-Linked Retinitis Pigmentosa.
Hu S; Du J; Chen N; Jia R; Zhang J; Liu X; Yang L
Invest Ophthalmol Vis Sci; 2020 Apr; 61(4):31. PubMed ID: 32330228
[TBL] [Abstract][Full Text] [Related]
30. RPGR gene therapy presents challenges in cloning the coding sequence.
Martinez-Fernandez De La Camara C; Cehajic-Kapetanovic J; MacLaren RE
Expert Opin Biol Ther; 2020 Jan; 20(1):63-71. PubMed ID: 31612744
[No Abstract] [Full Text] [Related]
31. Disease expression in X-linked retinitis pigmentosa caused by a putative null mutation in the RPGR gene.
Jacobson SG; Buraczynska M; Milam AH; Chen C; Järvaläinen M; Fujita R; Wu W; Huang Y; Cideciyan AV; Swaroop A
Invest Ophthalmol Vis Sci; 1997 Sep; 38(10):1983-97. PubMed ID: 9331262
[TBL] [Abstract][Full Text] [Related]
32. RPGR-associated retinal degeneration in human X-linked RP and a murine model.
Huang WC; Wright AF; Roman AJ; Cideciyan AV; Manson FD; Gewaily DY; Schwartz SB; Sadigh S; Limberis MP; Bell P; Wilson JM; Swaroop A; Jacobson SG
Invest Ophthalmol Vis Sci; 2012 Aug; 53(9):5594-608. PubMed ID: 22807293
[TBL] [Abstract][Full Text] [Related]
33. Early Cone Photoreceptor Outer Segment Length Shortening in RPGR X-Linked Retinitis Pigmentosa.
Menghini M; Jolly JK; Nanda A; Wood L; Cehajic-Kapetanovic J; MacLaren RE
Ophthalmologica; 2021; 244(4):281-290. PubMed ID: 32209785
[TBL] [Abstract][Full Text] [Related]
34. Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function.
Bush RA; Zeng Y; Colosi P; Kjellstrom S; Hiriyanna S; Vijayasarathy C; Santos M; Li J; Wu Z; Sieving PA
Hum Gene Ther; 2016 May; 27(5):376-89. PubMed ID: 27036983
[TBL] [Abstract][Full Text] [Related]
35. Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice.
Ye GJ; Conlon T; Erger K; Sonnentag P; Sharma AK; Howard K; Knop DR; Chulay JD
Hum Gene Ther Clin Dev; 2015 Sep; 26(3):177-84. PubMed ID: 26390091
[TBL] [Abstract][Full Text] [Related]
36. Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Ye GJ; Budzynski E; Sonnentag P; Nork TM; Miller PE; McPherson L; Ver Hoeve JN; Smith LM; Arndt T; Mandapati S; Robinson PM; Calcedo R; Knop DR; Hauswirth WW; Chulay JD
Hum Gene Ther Clin Dev; 2016 Mar; 27(1):27-36. PubMed ID: 27003752
[TBL] [Abstract][Full Text] [Related]
37. Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.
Pang JJ; Dai X; Boye SE; Barone I; Boye SL; Mao S; Everhart D; Dinculescu A; Liu L; Umino Y; Lei B; Chang B; Barlow R; Strettoi E; Hauswirth WW
Mol Ther; 2011 Feb; 19(2):234-42. PubMed ID: 21139570
[TBL] [Abstract][Full Text] [Related]
38. Prevalence of
Vinikoor-Imler LC; Simpson C; Narayanan D; Abbasi S; Lally C
Ophthalmic Genet; 2022 Oct; 43(5):581-588. PubMed ID: 36004681
[TBL] [Abstract][Full Text] [Related]
39. Zebrafish model for the genetic basis of X-linked retinitis pigmentosa.
Raghupathy RK; McCulloch DL; Akhtar S; Al-mubrad TM; Shu X
Zebrafish; 2013 Mar; 10(1):62-9. PubMed ID: 23536988
[TBL] [Abstract][Full Text] [Related]
40. Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.
Conlon TJ; Deng WT; Erger K; Cossette T; Pang JJ; Ryals R; Clément N; Cleaver B; McDoom I; Boye SE; Peden MC; Sherwood MB; Abernathy CR; Alkuraya FS; Boye SL; Hauswirth WW
Hum Gene Ther Clin Dev; 2013 Mar; 24(1):23-8. PubMed ID: 23692380
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
