165 related articles for article (PubMed ID: 29721936)
41. 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]
42. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year.
Cideciyan AV; Hauswirth WW; Aleman TS; Kaushal S; Schwartz SB; Boye SL; Windsor EA; Conlon TJ; Sumaroka A; Pang JJ; Roman AJ; Byrne BJ; Jacobson SG
Hum Gene Ther; 2009 Sep; 20(9):999-1004. PubMed ID: 19583479
[TBL] [Abstract][Full Text] [Related]
43. Inverse correlation between fatty acid transport protein 4 and vision in Leber congenital amaurosis associated with RPE65 mutation.
Li S; Gordon WC; Bazan NG; Jin M
Proc Natl Acad Sci U S A; 2020 Dec; 117(50):32114-32123. PubMed ID: 33257550
[TBL] [Abstract][Full Text] [Related]
44. A subpopulation of activated retinal macrophages selectively migrated to regions of cone photoreceptor stress, but had limited effect on cone death in a mouse model for type 2 Leber congenital amaurosis.
Tang PH; Pierson MJ; Heuss ND; Gregerson DS
Mol Cell Neurosci; 2017 Dec; 85():70-81. PubMed ID: 28889993
[TBL] [Abstract][Full Text] [Related]
45. Expression of the inactivating deiodinase, Deiodinase 3, in the pre-metamorphic tadpole retina.
Le Blay K; Préau L; Morvan-Dubois G; Demeneix B
PLoS One; 2018; 13(4):e0195374. PubMed ID: 29641587
[TBL] [Abstract][Full Text] [Related]
46. Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success.
Jacobson SG; Aleman TS; Cideciyan AV; Sumaroka A; Schwartz SB; Windsor EA; Traboulsi EI; Heon E; Pittler SJ; Milam AH; Maguire AM; Palczewski K; Stone EM; Bennett J
Proc Natl Acad Sci U S A; 2005 Apr; 102(17):6177-82. PubMed ID: 15837919
[TBL] [Abstract][Full Text] [Related]
47. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Hauswirth WW; Aleman TS; Kaushal S; Cideciyan AV; Schwartz SB; Wang L; Conlon TJ; Boye SL; Flotte TR; Byrne BJ; Jacobson SG
Hum Gene Ther; 2008 Oct; 19(10):979-90. PubMed ID: 18774912
[TBL] [Abstract][Full Text] [Related]
48. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.
Cideciyan AV; Aleman TS; Boye SL; Schwartz SB; Kaushal S; Roman AJ; Pang JJ; Sumaroka A; Windsor EA; Wilson JM; Flotte TR; Fishman GA; Heon E; Stone EM; Byrne BJ; Jacobson SG; Hauswirth WW
Proc Natl Acad Sci U S A; 2008 Sep; 105(39):15112-7. PubMed ID: 18809924
[TBL] [Abstract][Full Text] [Related]
49. Pathophysilogical mechanism and treatment strategies for Leber congenital amaurosis.
Fu Y; Zhang T
Adv Exp Med Biol; 2014; 801():791-6. PubMed ID: 24664772
[TBL] [Abstract][Full Text] [Related]
50. Cone opsin determines the time course of cone photoreceptor degeneration in Leber congenital amaurosis.
Zhang T; Zhang N; Baehr W; Fu Y
Proc Natl Acad Sci U S A; 2011 May; 108(21):8879-84. PubMed ID: 21555576
[TBL] [Abstract][Full Text] [Related]
51. Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate.
Maeda T; Cideciyan AV; Maeda A; Golczak M; Aleman TS; Jacobson SG; Palczewski K
Hum Mol Genet; 2009 Jun; 18(12):2277-87. PubMed ID: 19339306
[TBL] [Abstract][Full Text] [Related]
52. AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa.
Zhong H; Eblimit A; Moayedi Y; Boye SL; Chiodo VA; Chen Y; Li Y; Nichols RM; Hauswirth WW; Chen R; Mardon G
Gene Ther; 2015 Aug; 22(8):619-27. PubMed ID: 25965394
[TBL] [Abstract][Full Text] [Related]
53. Type 3 deiodinase, a thyroid-hormone-inactivating enzyme, controls survival and maturation of cone photoreceptors.
Ng L; Lyubarsky A; Nikonov SS; Ma M; Srinivas M; Kefas B; St Germain DL; Hernandez A; Pugh EN; Forrest D
J Neurosci; 2010 Mar; 30(9):3347-57. PubMed ID: 20203194
[TBL] [Abstract][Full Text] [Related]
54. 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]
55. Altered expression of the transcription factor Mef2c during retinal degeneration in Rpe65-/- mice.
Escher P; Schorderet DF; Cottet S
Invest Ophthalmol Vis Sci; 2011 Jul; 52(8):5933-40. PubMed ID: 21715356
[TBL] [Abstract][Full Text] [Related]
56. 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]
57. 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]
58. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.
Simonelli F; Maguire AM; Testa F; Pierce EA; Mingozzi F; Bennicelli JL; Rossi S; Marshall K; Banfi S; Surace EM; Sun J; Redmond TM; Zhu X; Shindler KS; Ying GS; Ziviello C; Acerra C; Wright JF; McDonnell JW; High KA; Bennett J; Auricchio A
Mol Ther; 2010 Mar; 18(3):643-50. PubMed ID: 19953081
[TBL] [Abstract][Full Text] [Related]
59. Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel.
Banin E; Bandah-Rozenfeld D; Obolensky A; Cideciyan AV; Aleman TS; Marks-Ohana D; Sela M; Boye S; Sumaroka A; Roman AJ; Schwartz SB; Hauswirth WW; Jacobson SG; Hemo I; Sharon D
Hum Gene Ther; 2010 Dec; 21(12):1749-57. PubMed ID: 20604683
[TBL] [Abstract][Full Text] [Related]
60. 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]
[Previous] [Next] [New Search]