214 related articles for article (PubMed ID: 36589710)
1. Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing.
Jo DH; Jang HK; Cho CS; Han JH; Ryu G; Jung Y; Bae S; Kim JH
Mol Ther Nucleic Acids; 2023 Mar; 31():16-27. PubMed ID: 36589710
[TBL] [Abstract][Full Text] [Related]
2. CRISPR-Cas9-mediated therapeutic editing of
Jo DH; Song DW; Cho CS; Kim UG; Lee KJ; Lee K; Park SW; Kim D; Kim JH; Kim JS; Kim S; Kim JH; Lee JM
Sci Adv; 2019 Oct; 5(10):eaax1210. PubMed ID: 31692906
[TBL] [Abstract][Full Text] [Related]
3. Intraperitoneal chromophore injections delay early-onset and rapid retinal cone degeneration in a mouse model of Leber congenital amaurosis.
Dai X; Jin X; Ye Q; Huang H; Duo L; Lu C; Bao J; Chen H
Exp Eye Res; 2021 Nov; 212():108776. PubMed ID: 34582935
[TBL] [Abstract][Full Text] [Related]
4. In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration.
Choi EH; Suh S; Foik AT; Leinonen H; Newby GA; Gao XD; Banskota S; Hoang T; Du SW; Dong Z; Raguram A; Kohli S; Blackshaw S; Lyon DC; Liu DR; Palczewski K
Nat Commun; 2022 Apr; 13(1):1830. PubMed ID: 35383196
[TBL] [Abstract][Full Text] [Related]
5. Dual-AAV split prime editor corrects the mutation and phenotype in mice with inherited retinal degeneration.
She K; Liu Y; Zhao Q; Jin X; Yang Y; Su J; Li R; Song L; Xiao J; Yao S; Lu F; Wei Y; Yang Y
Signal Transduct Target Ther; 2023 Feb; 8(1):57. PubMed ID: 36740702
[TBL] [Abstract][Full Text] [Related]
6. Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).
Pang JJ; Chang B; Hawes NL; Hurd RE; Davisson MT; Li J; Noorwez SM; Malhotra R; McDowell JH; Kaushal S; Hauswirth WW; Nusinowitz S; Thompson DA; Heckenlively JR
Mol Vis; 2005 Feb; 11():152-62. PubMed ID: 15765048
[TBL] [Abstract][Full Text] [Related]
7. Pharmacological Amelioration of Cone Survival and Vision in a Mouse Model for Leber Congenital Amaurosis.
Li S; Samardzija M; Yang Z; Grimm C; Jin M
J Neurosci; 2016 May; 36(21):5808-19. PubMed ID: 27225770
[TBL] [Abstract][Full Text] [Related]
8. 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]
9. 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]
10. Unlocking therapeutic potential: dual gene therapy for ameliorating the disease phenotypes in a mouse model of
Liu Y; Tai J; Yu C; Xu D; Xiao D; Pang J
Front Med (Lausanne); 2023; 10():1291795. PubMed ID: 38264046
[TBL] [Abstract][Full Text] [Related]
11. Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing.
Suh S; Choi EH; Leinonen H; Foik AT; Newby GA; Yeh WH; Dong Z; Kiser PD; Lyon DC; Liu DR; Palczewski K
Nat Biomed Eng; 2021 Feb; 5(2):169-178. PubMed ID: 33077938
[TBL] [Abstract][Full Text] [Related]
12. Ablation of fatty acid transport protein-4 enhances cone survival, M-cone vision, and synthesis of cone-tropic 9-
Li S; Jin M
J Neurosci; 2024 May; ():. PubMed ID: 38811164
[TBL] [Abstract][Full Text] [Related]
13. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis.
Pang JJ; Chang B; Kumar A; Nusinowitz S; Noorwez SM; Li J; Rani A; Foster TC; Chiodo VA; Doyle T; Li H; Malhotra R; Teusner JT; McDowell JH; Min SH; Li Q; Kaushal S; Hauswirth WW
Mol Ther; 2006 Mar; 13(3):565-72. PubMed ID: 16223604
[TBL] [Abstract][Full Text] [Related]
14. Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis.
Bemelmans AP; Kostic C; Crippa SV; Hauswirth WW; Lem J; Munier FL; Seeliger MW; Wenzel A; Arsenijevic Y
PLoS Med; 2006 Oct; 3(10):e347. PubMed ID: 17032058
[TBL] [Abstract][Full Text] [Related]
15. Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.
Roman AJ; Boye SL; Aleman TS; Pang JJ; McDowell JH; Boye SE; Cideciyan AV; Jacobson SG; Hauswirth WW
Mol Vis; 2007 Sep; 13():1701-10. PubMed ID: 17960108
[TBL] [Abstract][Full Text] [Related]
16. Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy.
Cideciyan AV
Prog Retin Eye Res; 2010 Sep; 29(5):398-427. PubMed ID: 20399883
[TBL] [Abstract][Full Text] [Related]
17. 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]
18. Novel gene variants in Polish patients with Leber congenital amaurosis (LCA).
Skorczyk-Werner A; Niedziela Z; Stopa M; Krawczyński MR
Orphanet J Rare Dis; 2020 Dec; 15(1):345. PubMed ID: 33308271
[TBL] [Abstract][Full Text] [Related]
19. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.
Li W; Kong F; Li X; Dai X; Liu X; Zheng Q; Wu R; Zhou X; Lü F; Chang B; Li Q; Hauswirth WW; Qu J; Pang JJ
Mol Vis; 2009; 15():267-75. PubMed ID: 19190735
[TBL] [Abstract][Full Text] [Related]
20.
; ; . PubMed ID:
[No Abstract] [Full Text] [Related]
[Next] [New Search]