These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
4. Short-Term Outcomes of the First in Vivo Gene Therapy for RPE65-Mediated Retinitis Pigmentosa. Kwak JJ; Kim HR; Byeon SH Yonsei Med J; 2022 Jul; 63(7):701-705. PubMed ID: 35748082 [TBL] [Abstract][Full Text] [Related]
5. Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis. Gange WS; Sisk RA; Besirli CG; Lee TC; Havunjian M; Schwartz H; Borchert M; Sengillo JD; Mendoza C; Berrocal AM; Nagiel A Ophthalmol Retina; 2022 Jan; 6(1):58-64. PubMed ID: 33838313 [TBL] [Abstract][Full Text] [Related]
6. Gene therapy for inherited retinal diseases: progress and possibilities. Hu ML; Edwards TL; O'Hare F; Hickey DG; Wang JH; Liu Z; Ayton LN Clin Exp Optom; 2021 May; 104(4):444-454. PubMed ID: 33689657 [TBL] [Abstract][Full Text] [Related]
7. Gene Therapy in Hereditary Retinal Dystrophies: The Usefulness of Diagnostic Tools in Candidate Patient Selections. Malvasi M; Casillo L; Avogaro F; Abbouda A; Vingolo EM Int J Mol Sci; 2023 Sep; 24(18):. PubMed ID: 37762059 [TBL] [Abstract][Full Text] [Related]
8. [Inherited retinal dystrophy: first results of RPE65 gene replacement therapy in Russia]. Kadyshev VV; Zolnikova IV; Khalanskaya OV; Stepanova AA; Kutsev SI Vestn Oftalmol; 2022; 138(4):48-57. PubMed ID: 36004591 [TBL] [Abstract][Full Text] [Related]
9. Voretigene neparvovec-rzyl for treatment of Ciulla TA; Hussain RM; Berrocal AM; Nagiel A Expert Opin Biol Ther; 2020 Jun; 20(6):565-578. PubMed ID: 32149547 [No Abstract] [Full Text] [Related]
10. Current Management of Patients with RPE65 Mutation-Associated Inherited Retinal Degenerations in Europe: Results of a Multinational Survey by the European Vision Institute Clinical Research Network. Lorenz B; Tavares J; van den Born LI; Marques JP; Scholl HPN; Ophthalmic Res; 2021; 64(5):740-753. PubMed ID: 33684911 [TBL] [Abstract][Full Text] [Related]
11. [Diagnostics of inherited retinal degenerations by gene therapy]. Shurygina MF; Khoteeva AM Vestn Oftalmol; 2021; 137(4):145-151. PubMed ID: 34410070 [TBL] [Abstract][Full Text] [Related]
12. Epidemiology of Mutations in the 65-kDa Retinal Pigment Epithelium (RPE65) Gene-Mediated Inherited Retinal Dystrophies: A Systematic Literature Review. Sallum JMF; Kaur VP; Shaikh J; Banhazi J; Spera C; Aouadj C; Viriato D; Fischer MD Adv Ther; 2022 Mar; 39(3):1179-1198. PubMed ID: 35098484 [TBL] [Abstract][Full Text] [Related]
13. 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]
14. An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials. Chiu W; Lin TY; Chang YC; Isahwan-Ahmad Mulyadi Lai H; Lin SC; Ma C; Yarmishyn AA; Lin SC; Chang KJ; Chou YB; Hsu CC; Lin TC; Chen SJ; Chien Y; Yang YP; Hwang DK Int J Mol Sci; 2021 Apr; 22(9):. PubMed ID: 33926102 [TBL] [Abstract][Full Text] [Related]
15. Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy. Yang U; Gentleman S; Gai X; Gorin MB; Borchert MS; Lee TC; Villanueva A; Koenekoop R; Maguire AM; Bennett J; Redmond TM; Nagiel A JAMA Ophthalmol; 2019 Dec; 137(12):1381-1388. PubMed ID: 31580392 [TBL] [Abstract][Full Text] [Related]
16. Properties and Therapeutic Implications of an Enigmatic D477G RPE65 Variant Associated with Autosomal Dominant Retinitis Pigmentosa. Kiang AS; Kenna PF; Humphries MM; Ozaki E; Koenekoop RK; Campbell M; Farrar GJ; Humphries P Genes (Basel); 2020 Nov; 11(12):. PubMed ID: 33261050 [TBL] [Abstract][Full Text] [Related]
17. Newer therapeutic options for inherited retinal diseases: Gene and cell replacement therapy. Battu R; Ratra D; Gopal L Indian J Ophthalmol; 2022 Jul; 70(7):2316-2325. PubMed ID: 35791112 [TBL] [Abstract][Full Text] [Related]
18. Gene therapy for Leber congenital amaurosis: advances and future directions. Hufnagel RB; Ahmed ZM; Corrêa ZM; Sisk RA Graefes Arch Clin Exp Ophthalmol; 2012 Aug; 250(8):1117-28. PubMed ID: 22644094 [TBL] [Abstract][Full Text] [Related]