Sort by
Items per page

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 29


Advances in Gene Therapy for Diseases of the Eye.

Petit L, Khanna H, Punzo C.

Hum Gene Ther. 2016 Aug;27(8):563-79. doi: 10.1089/hum.2016.040. Epub 2016 Jun 13.


Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.

Beltran WA, Cideciyan AV, Iwabe S, Swider M, Kosyk MS, McDaid K, Martynyuk I, Ying GS, Shaffer J, Deng WT, Boye SL, Lewin AS, Hauswirth WW, Jacobson SG, Aguirre GD.

Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5844-53. doi: 10.1073/pnas.1509914112. Epub 2015 Oct 12.


Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.

Mookherjee S, Hiriyanna S, Kaneshiro K, Li L, Li Y, Li W, Qian H, Li T, Khanna H, Colosi P, Swaroop A, Wu Z.

Hum Mol Genet. 2015 Nov 15;24(22):6446-58. doi: 10.1093/hmg/ddv354. Epub 2015 Sep 10.


Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa.

Pawlyk BS, Bulgakov OV, Sun X, Adamian M, Shu X, Smith AJ, Berson EL, Ali RR, Khani S, Wright AF, Sandberg MA, Li T.

Gene Ther. 2016 Feb;23(2):196-204. doi: 10.1038/gt.2015.93. Epub 2015 Sep 8.


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. doi: 10.1038/gt.2015.42. Epub 2015 May 12.


Protective effect of high concentration of BN52021 on retinal contusion in cat eyes.

Huang JF, Zhao HP, Yang YF, Huang HM, Yao Y, Wang ZJ.

BMC Ophthalmol. 2015 May 9;15:50. doi: 10.1186/s12886-015-0030-2.


Leber congenital amaurosis caused by mutations in RPGRIP1.

Li T.

Cold Spring Harb Perspect Med. 2014 Nov 20;5(4). pii: a017384. doi: 10.1101/cshperspect.a017384. Review.


Leber congenital amaurosis caused by mutations in GUCY2D.

Boye SE.

Cold Spring Harb Perspect Med. 2014 Sep 25;5(1):a017350. doi: 10.1101/cshperspect.a017350. Review.


Loss of Raf-1 kinase inhibitory protein delays early-onset severe retinal ciliopathy in Cep290rd16 mouse.

Subramanian B, Anand M, Khan NW, Khanna H.

Invest Ophthalmol Vis Sci. 2014 Aug 14;55(9):5788-94. doi: 10.1167/iovs.14-14954.


Insights gained from gene therapy in animal models of retGC1 deficiency.

Boye SE.

Front Mol Neurosci. 2014 May 14;7:43. doi: 10.3389/fnmol.2014.00043. eCollection 2014. Review.


Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.

Lhériteau E, Petit L, Weber M, Le Meur G, Deschamps JY, Libeau L, Mendes-Madeira A, Guihal C, François A, Guyon R, Provost N, Lemoine F, Papal S, El-Amraoui A, Colle MA, Moullier P, Rolling F.

Mol Ther. 2014 Feb;22(2):265-77. doi: 10.1038/mt.2013.232. Epub 2013 Oct 4.


Combined rod and cone transduction by adeno-associated virus 2/8.

Manfredi A, Marrocco E, Puppo A, Cesi G, Sommella A, Della Corte M, Rossi S, Giunti M, Craft CM, Bacci ML, Simonelli F, Surace EM, Auricchio A.

Hum Gene Ther. 2013 Dec;24(12):982-92. doi: 10.1089/hum.2013.154. Epub 2013 Oct 30.


Immunology of AAV-Mediated Gene Transfer in the Eye.

Willett K, Bennett J.

Front Immunol. 2013 Aug 30;4:261. doi: 10.3389/fimmu.2013.00261. eCollection 2013. Review.


Smelling the roses and seeing the light: gene therapy for ciliopathies.

McIntyre JC, Williams CL, Martens JR.

Trends Biotechnol. 2013 Jun;31(6):355-63. doi: 10.1016/j.tibtech.2013.03.005. Epub 2013 Apr 17. Review.


Prospectives for gene therapy of retinal degenerations.

Thumann G.

Curr Genomics. 2012 Aug;13(5):350-62. doi: 10.2174/138920212801619214.


Photoreceptor sensory cilia and ciliopathies: focus on CEP290, RPGR and their interacting proteins.

Rachel RA, Li T, Swaroop A.

Cilia. 2012 Dec 3;1(1):22. doi: 10.1186/2046-2530-1-22.


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 5;110(6):E517-25. doi: 10.1073/pnas.1218933110. Epub 2013 Jan 22.


Gene therapy for retinal disease.

McClements ME, MacLaren RE.

Transl Res. 2013 Apr;161(4):241-54. doi: 10.1016/j.trsl.2012.12.007. Epub 2013 Jan 8. Review.


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.

Hum Gene Ther. 2013 Feb;24(2):189-202. doi: 10.1089/hum.2012.193.

Supplemental Content

Support Center