483 related articles for article (PubMed ID: 30335549)
1. Gene therapy for RPE65-related retinal disease.
Miraldi Utz V; Coussa RG; Antaki F; Traboulsi EI
Ophthalmic Genet; 2018 Dec; 39(6):671-677. PubMed ID: 30335549
[TBL] [Abstract][Full Text] [Related]
2. Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient With RPE65-Associated Leber's Congenital Amaurosis.
Hussain RM; Tran KD; Maguire AM; Berrocal AM
Ophthalmic Surg Lasers Imaging Retina; 2019 Oct; 50(10):661-663. PubMed ID: 31671202
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. Available Evidence on Leber Congenital Amaurosis and Gene Therapy.
Alkharashi M; Fulton AB
Semin Ophthalmol; 2017; 32(1):14-21. PubMed ID: 27686653
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. 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]
7. [Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial].
Chacón-Camacho ÓF; Zenteno JC
Gac Med Mex; 2017; 153(2):276-278. PubMed ID: 28474714
[TBL] [Abstract][Full Text] [Related]
8. 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]
9. 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]
10. 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]
11. Inherited Retinal Diseases Due to
Aoun M; Passerini I; Chiurazzi P; Karali M; De Rienzo I; Sartor G; Murro V; Filimonova N; Seri M; Banfi S
Int J Mol Sci; 2021 Jul; 22(13):. PubMed ID: 34281261
[TBL] [Abstract][Full Text] [Related]
12. 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]
13. Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.
Le Meur G; Lebranchu P; Billaud F; Adjali O; Schmitt S; Bézieau S; Péréon Y; Valabregue R; Ivan C; Darmon C; Moullier P; Rolling F; Weber M
Mol Ther; 2018 Jan; 26(1):256-268. PubMed ID: 29033008
[TBL] [Abstract][Full Text] [Related]
14. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
Weleber RG; Pennesi ME; Wilson DJ; Kaushal S; Erker LR; Jensen L; McBride MT; Flotte TR; Humphries M; Calcedo R; Hauswirth WW; Chulay JD; Stout JT
Ophthalmology; 2016 Jul; 123(7):1606-20. PubMed ID: 27102010
[TBL] [Abstract][Full Text] [Related]
15. Short term morphological rescue of the fovea after gene therapy with voretigene neparvovec.
Kortüm FC; Kempf M; Jung R; Kohl S; Ott S; Kortuem C; Sting K; Stingl K
Acta Ophthalmol; 2022 May; 100(3):e807-e812. PubMed ID: 34289237
[TBL] [Abstract][Full Text] [Related]
16. Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy.
Kang C; Scott LJ
Mol Diagn Ther; 2020 Aug; 24(4):487-495. PubMed ID: 32535767
[TBL] [Abstract][Full Text] [Related]
17. [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]
18. Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis.
Rakoczy EP; Narfström K
Int J Biochem Cell Biol; 2014 Nov; 56():153-7. PubMed ID: 25286304
[TBL] [Abstract][Full Text] [Related]
19. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
Cideciyan AV; Jacobson SG; Beltran WA; Sumaroka A; Swider M; Iwabe S; Roman AJ; Olivares MB; Schwartz SB; Komáromy AM; Hauswirth WW; Aguirre GD
Proc Natl Acad Sci U S A; 2013 Feb; 110(6):E517-25. PubMed ID: 23341635
[TBL] [Abstract][Full Text] [Related]
20. 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]
[Next] [New Search]