Similar articles for PMID: 20384479

Year	Number of Results
1997	1
2000	2
2001	2
2003	1
2005	6
2006	1
2008	1
2009	7
2010	8
2011	18
2012	10
2013	7
2014	11
2015	9
2016	6
2017	7
2018	11
2019	9
2020	9
2021	10
2022	5
2023	6
2024	0

1

Replacement gene therapy with a human RPGRIP1 sequence slows photoreceptor degeneration in a murine model of Leber congenital amaurosis.

Pawlyk BS, Bulgakov OV, Liu X, Xu X, Adamian M, Sun X, Khani SC, Berson EL, Sandberg MA, Li T. Pawlyk BS, et al. Hum Gene Ther. 2010 Aug;21(8):993-1004. doi: 10.1089/hum.2009.218. Hum Gene Ther. 2010. PMID: 20384479 Free PMC article.

2

Selective loss of RPGRIP1-dependent ciliary targeting of NPHP4, RPGR and SDCCAG8 underlies the degeneration of photoreceptor neurons.

Patil H, Tserentsoodol N, Saha A, Hao Y, Webb M, Ferreira PA. Patil H, et al. Cell Death Dis. 2012 Jul 19;3(7):e355. doi: 10.1038/cddis.2012.96. Cell Death Dis. 2012. PMID: 22825473 Free PMC article.

3

Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.

Pawlyk BS, Smith AJ, Buch PK, Adamian M, Hong DH, Sandberg MA, Ali RR, Li T. Pawlyk BS, et al. Invest Ophthalmol Vis Sci. 2005 Sep;46(9):3039-45. doi: 10.1167/iovs.05-0371. Invest Ophthalmol Vis Sci. 2005. PMID: 16123399

4

Leber congenital amaurosis caused by mutations in RPGRIP1.

Li T. Li T. Cold Spring Harb Perspect Med. 2014 Nov 20;5(4):a017384. doi: 10.1101/cshperspect.a017384. Cold Spring Harb Perspect Med. 2014. PMID: 25414380 Free PMC article. Review.

5

Spata7 is a retinal ciliopathy gene critical for correct RPGRIP1 localization and protein trafficking in the retina.

Eblimit A, Nguyen TM, Chen Y, Esteve-Rudd J, Zhong H, Letteboer S, Van Reeuwijk J, Simons DL, Ding Q, Wu KM, Li Y, Van Beersum S, Moayedi Y, Xu H, Pickard P, Wang K, Gan L, Wu SM, Williams DS, Mardon G, Roepman R, Chen R. Eblimit A, et al. Hum Mol Genet. 2015 Mar 15;24(6):1584-601. doi: 10.1093/hmg/ddu573. Epub 2014 Nov 14. Hum Mol Genet. 2015. PMID: 25398945 Free PMC article.

6

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. Zhong H, et al. Gene Ther. 2015 Aug;22(8):619-27. doi: 10.1038/gt.2015.42. Epub 2015 May 12. Gene Ther. 2015. PMID: 25965394 Free PMC article.

7

AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.

Boye SL, Peshenko IV, Huang WC, Min SH, McDoom I, Kay CN, Liu X, Dyka FM, Foster TC, Umino Y, Karan S, Jacobson SG, Baehr W, Dizhoor A, Hauswirth WW, Boye SE. Boye SL, et al. Hum Gene Ther. 2013 Feb;24(2):189-202. doi: 10.1089/hum.2012.193. Hum Gene Ther. 2013. PMID: 23210611 Free PMC article.

8

RPGRIP1 is mutated in Leber congenital amaurosis: a mini-review.

Koenekoop RK. Koenekoop RK. Ophthalmic Genet. 2005 Dec;26(4):175-9. doi: 10.1080/13816810500374441. Ophthalmic Genet. 2005. PMID: 16352478 Review.

9

Rescue of cone function in cone-only Nphp5 knockout mouse model with Leber congenital amaurosis phenotype.

Hanke-Gogokhia C, Chiodo VA, Hauswirth WW, Frederick JM, Baehr W. Hanke-Gogokhia C, et al. Mol Vis. 2018 Dec 30;24:834-846. eCollection 2018. Mol Vis. 2018. PMID: 30713422 Free PMC article.

10

RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.

Molday LL, Djajadi H, Yan P, Szczygiel L, Boye SL, Chiodo VA, Gregory-Evans K, Sarunic MV, Hauswirth WW, Molday RS. Molday LL, et al. Hum Mol Genet. 2013 Oct 1;22(19):3894-905. doi: 10.1093/hmg/ddt244. Epub 2013 Jun 4. Hum Mol Genet. 2013. PMID: 23740938 Free PMC article.

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