Ocular Gene Therapy Laboratory

Publications from the Ocular Gene Therapy Laboratory 

• Presynaptic Expression of LRIT3 Transsynaptically Organizes the Postsynaptic Glutamate Signaling Complex Containing TRPM1.

  (Hasan N, Pangeni G, Cobb CA, Ray TA, Nettesheim ER, Ertel KJ, Lipinski DM, McCall MA, Gregg RG.) Cell Rep. 2019 06 11;27(11):3107-3116.e3 PMID: 31189098 PMCID: PMC6628893 06/13/2019

• Acid Ceramidase Deficiency in Mice Leads to Severe Ocular Pathology and Visual Impairment.

  (Yu FPS, Sajdak BS, Sikora J, Salmon AE, Nagree MS, Gurka J, Kassem IS, Lipinski DM, Carroll J, Medin JA.) Am J Pathol. 2019 02;189(2):320-338 PMID: 30472209 PMCID: PMC6412726 SCOPUS ID: 2-s2.0-85060099082 11/26/2018

• A Comparison of Inducible Gene Expression Platforms: Implications for Recombinant Adeno-Associated Virus (rAAV) Vector-Mediated Ocular Gene Therapy.

  (Lipinski DM.) Adv Exp Med Biol. 2019;1185:79-83 PMID: 31884592 12/31/2019

• A Destabilizing Domain Allows for Fast, Noninvasive, Conditional Control of Protein Abundance in the Mouse Eye - Implications for Ocular Gene Therapy.

  (Datta S, Renwick M, Chau VQ, Zhang F, Nettesheim ER, Lipinski DM, Hulleman JD.) Invest Ophthalmol Vis Sci. 2018 10 01;59(12):4909-4920 PMID: 30347085 PMCID: PMC6181441 10/23/2018

• Development of an inducible anti-VEGF rAAV gene therapy strategy for the treatment of wet AMD.

  (Reid CA, Nettesheim ER, Connor TB, Lipinski DM.) Sci Rep. 2018 08 06;8(1):11763 PMID: 30082848 PMCID: PMC6079038 SCOPUS ID: 2-s2.0-85051247613 08/08/2018

• Small and Micro-Scale Recombinant Adeno-Associated Virus Production and Purification for Ocular Gene Therapy Applications.

  (Reid CA, Lipinski DM.) Methods Mol Biol. 2018;1715:19-31 PMID: 29188503 12/01/2017

• Improvement of Photoreceptor Targeting via Intravitreal Delivery in Mouse and Human Retina Using Combinatory rAAV2 Capsid Mutant Vectors.

  (Reid CA, Ertel KJ, Lipinski DM.) Invest Ophthalmol Vis Sci. 2017 12 01;58(14):6429-6439 PMID: 29260200 PMCID: PMC5736327 12/21/2017

• miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity.

  (Reid CA, Boye SL, Hauswirth WW, Lipinski DM.) Gene Ther. 2017 08;24(8):462-469 PMID: 28617420 06/16/2017

• Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4 mouse and bipolar cells in the rd1 mouse and human retina ex vivo.

  (De Silva SR, Charbel Issa P, Singh MS, Lipinski DM, Barnea-Cramer AO, Walker NJ, Barnard AR, Hankins MW, MacLaren RE.) Gene Ther. 2016 11;23(11):767-774 PMID: 27416076 PMCID: PMC5097463 11/04/2016

• Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.

  (Lipinski DM, Reid CA, Boye SL, Peterson JJ, Qi X, Boye SE, Boulton ME, Hauswirth WW.) Hum Gene Ther. 2015 Nov;26(11):767-76 PMID: 26359319 PMCID: PMC4651034 09/12/2015

• CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa.

  (Lipinski DM, Barnard AR, Singh MS, Martin C, Lee EJ, Davies WIL, MacLaren RE.) Mol Ther. 2015 Aug;23(8):1308-1319 PMID: 25896245 PMCID: PMC4539573 04/22/2015

• Vesicular stomatitis virus glycoprotein- and Venezuelan equine encephalitis virus-derived glycoprotein-pseudotyped lentivirus vectors differentially transduce corneal endothelium, trabecular meshwork, and human photoreceptors.

  (Lipinski DM, Barnard AR, Charbel Issa P, Singh MS, De Silva SR, Trabalza A, Eleftheriadou I, Ellison SM, Mazarakis ND, MacLaren RE.) Hum Gene Ther. 2014 Jan;25(1):50-62 PMID: 24125177 10/16/2013

• Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo.

  (Tolmachova T, Tolmachov OE, Barnard AR, de Silva SR, Lipinski DM, Walker NJ, Maclaren RE, Seabra MC.) J Mol Med (Berl). 2013 Jul;91(7):825-37 PMID: 23756766 PMCID: PMC3695676 06/13/2013

• Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation.

  (Singh MS, Charbel Issa P, Butler R, Martin C, Lipinski DM, Sekaran S, Barnard AR, MacLaren RE.) Proc Natl Acad Sci U S A. 2013 Jan 15;110(3):1101-6 PMID: 23288902 PMCID: PMC3549087 01/05/2013

• Assessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina.

  (Charbel Issa P, De Silva SR, Lipinski DM, Singh MS, Mouravlev A, You Q, Barnard AR, Hankins MW, During MJ, Maclaren RE.) PLoS One. 2013;8(4):e60361 PMID: 23593201 PMCID: PMC3621895 04/18/2013

• Clinical applications of retinal gene therapy.

  (Lipinski DM, Thake M, MacLaren RE.) Prog Retin Eye Res. 2013 Jan;32:22-47 PMID: 22995954 09/22/2012

• Optimization of in vivo confocal autofluorescence imaging of the ocular fundus in mice and its application to models of human retinal degeneration.

  (Charbel Issa P, Singh MS, Lipinski DM, Chong NV, Delori FC, Barnard AR, MacLaren RE.) Invest Ophthalmol Vis Sci. 2012 Feb;53(2):1066-75 PMID: 22169101 PMCID: PMC3317405 12/16/2011

• AAV-mediated gene transfer of human X-linked inhibitor of apoptosis protects against oxidative cell death in human RPE cells.

  (Shan H, Ji D, Barnard AR, Lipinski DM, You Q, Lee EJ, Kamalden TA, Sun X, MacLaren RE.) Invest Ophthalmol Vis Sci. 2011 Dec 20;52(13):9591-7 PMID: 22039248 11/01/2011

• Combining M-FISH and Quantum Dot technology for fast chromosomal assignment of transgenic insertions.

  (Yusuf M, Bauer DL, Lipinski DM, MacLaren RE, Wade-Martins R, Mir KU, Volpi EV.) BMC Biotechnol. 2011 Dec 13;11:121 PMID: 22165957 PMCID: PMC3266222 12/15/2011

• Assessment of cone survival in response to CNTF, GDNF, and VEGF165b in a novel ex vivo model of end-stage retinitis pigmentosa.

  (Lipinski DM, Singh MS, MacLaren RE.) Invest Ophthalmol Vis Sci. 2011 Sep 21;52(10):7340-6 PMID: 21873685 08/30/2011

• Characterization of a dominant cone degeneration in a green fluorescent protein-reporter mouse with disruption of Loci associated with human dominant retinal dystrophy.

  (Lipinski DM, Yusuf M, Barnard AR, Damant C, Charbel Issa P, Singh MS, Lee E, Davies WL, Volpi EV, MacLaren RE.) Invest Ophthalmol Vis Sci. 2011 Aug 22;52(9):6617-23 PMID: 21705682 PMCID: PMC3175985 06/28/2011