MCW Ocular Gene Therapy Laboratory Publications

Recent Publications

• Human iPSC-derived photoreceptor transplantation in the cone dominant 13-lined ground squirrel.

  (Yu CT, Kandoi S, Periasamy R, Reddy LVK, Follett HM, Summerfelt P, Martinez C, Guillaume C, Bowie O, Connor TB, Lipinski DM, Allen KP, Merriman DK, Carroll J, Lamba DA.) Stem Cell Reports. 2024 Mar 12;19(3):331-342 PMID: 38335965 PMCID: PMC10937153 SCOPUS ID: 2-s2.0-85186065745 02/10/2024

• Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber.

  (Chern KJ, Issac KZ, Gumbs ZD, O'Connor ME, Lawrence MS, Lipinski DM.) Gene Ther. 2023 Sep;30(9):714-722 PMID: 37221271 SCOPUS ID: 2-s2.0-85160260133 05/24/2023

• Downregulation of lysosomal trafficking in ARPE19 cells leads to decreased transfection efficiency at high passage.

  (Hood EMS, Lipinski RAJ, Lipinski DM.) bioRxiv. 2023 Jul 26 PMID: 37546846 PMCID: PMC10402107 08/07/2023

• rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis.

  (Zhang H, Nagree MS, Liu H, Pan X, Medin JA, Lipinski DM.) Gene Ther. 2023 Apr;30(3-4):297-308 PMID: 35902747 SCOPUS ID: 2-s2.0-85149011321 07/29/2022

• Improving retinal vascular endothelial cell tropism through rational rAAV capsid design.

  (Periasamy R, Patel DD, Boye SL, Boye SE, Lipinski DM.) PLoS One. 2023;18(5):e0285370 PMID: 37167304 PMCID: PMC10174500 SCOPUS ID: 2-s2.0-85159655750 05/11/2023

• Prostaglandin-based rAAV-mediated glaucoma gene therapy in Brown Norway rats.

  (Chern KJ, Nettesheim ER, Reid CA, Li NW, Marcoe GJ, Lipinski DM.) Commun Biol. 2022 Nov 03;5(1):1169 PMID: 36329259 PMCID: PMC9633612 SCOPUS ID: 2-s2.0-85141145849 11/05/2022

• Identification of Novel Retinal Pericyte-Targeting rAAV Vectors Through Directed Evolution.

  (Patel DD, Marsic D, Periasamy R, Zolotukhin S, Lipinski DM.) Transl Vis Sci Technol. 2022 Aug 01;11(8):28 PMID: 36018583 PMCID: PMC9428359 SCOPUS ID: 2-s2.0-85136924759 08/27/2022

• Isolation, culture, and cryosectioning of primary porcine retinal pigment epithelium on transwell cell culture inserts

  (Hood EMS, Curcio CA, Lipinski D.) STAR Protocols. 16 December 2022;3(4) SCOPUS ID: 2-s2.0-85139727880 12/16/2022

• Pre-retinal delivery of recombinant adeno-associated virus vector significantly improves retinal transduction efficiency.

  (Zhang H, Sajdak BS, Merriman DK, Carroll J, Lipinski DM.) Mol Ther Methods Clin Dev. 2021 Sep 10;22:96-106 PMID: 34485598 PMCID: PMC8390453 09/07/2021

• Development of a Preclinical Laser Speckle Contrast Imaging Instrument for Assessing Systemic and Retinal Vascular Function in Small Rodents.

  (Patel DD, Dhalla AH, Viehland C, Connor TB, Lipinski DM.) Transl Vis Sci Technol. 2021 Aug 02;10(9):19 PMID: 34403474 PMCID: PMC8374978 SCOPUS ID: 2-s2.0-85114392221 08/18/2021

• Electroretinogram of the Cone-Dominant Thirteen-Lined Ground Squirrel during Euthermia and Hibernation in Comparison with the Rod-Dominant Brown Norway Rat.

  (Zhang H, Sajdak BS, Merriman DK, McCall MA, Carroll J, Lipinski DM.) Invest Ophthalmol Vis Sci. 2020 Jun 03;61(6):6 PMID: 32492111 PMCID: PMC7415905 SCOPUS ID: 2-s2.0-85085963234 06/04/2020

• Validating a low-cost laser speckle contrast imaging system as a quantitative tool for assessing retinal vascular function.

  (Patel DD, Lipinski DM.) Sci Rep. 2020 Apr 28;10(1):7177 PMID: 32346043 PMCID: PMC7188677 SCOPUS ID: 2-s2.0-85083998758 04/30/2020

• 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 Jun 11;27(11):3107-3116.e3 PMID: 31189098 PMCID: PMC6628893 SCOPUS ID: 2-s2.0-85066800714 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 Feb;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 SCOPUS ID: 2-s2.0-85077321194 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 Oct 01;59(12):4909-4920 PMID: 30347085 PMCID: PMC6181441 SCOPUS ID: 2-s2.0-85055194829 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 Aug 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 SCOPUS ID: 2-s2.0-85036634077 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 Dec 01;58(14):6429-6439 PMID: 29260200 PMCID: PMC5736327 SCOPUS ID: 2-s2.0-85038855464 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 Aug;24(8):462-469 PMID: 28617420 SCOPUS ID: 2-s2.0-85030779767 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 Nov;23(11):767-774 PMID: 27416076 PMCID: PMC5097463 SCOPUS ID: 2-s2.0-84981508189 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 SCOPUS ID: 2-s2.0-84946906911 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 SCOPUS ID: 2-s2.0-84938420570 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 SCOPUS ID: 2-s2.0-84892868349 10/16/2013