Medical College of Wisconsin Ocular Gene Therapy Laboratory 2018 News

2018 News

	^{November 26, 2018} Congratulations – Kristina Ertel passes her qualifying exam Kristina Ertel, a second year graduate in the Interdisciplinary Doctoral Program (IDP), successfully passed her qualifying exam in front of a committee consisting of Daniel M. Lipinski, DPhil, Brian A. Link, PhD, Ross Collery, PhD and Elena Semina, PhD. Kristina will continue to work on developing a long-acting, single use gene therapy treatment for primary open angle glaucoma (POAG), a debilitating disease that causes vision loss in approximately 70 million people worldwide.
	^{October 22, 2018} Welcome – Gavin Marcoe joins OGTL as a Research Technologist Gavin Marcoe recently completed bachelor’s degrees (BS) in both Cell Biology and Chemistry from the University of Wisconsin Green Bay and will join OGTL as a research Technologist. Gavin will be responsible for assisting the everyday running of the lab, in addition to conducting his own research project focused on the design of novel viral vectors with large coding capacity for retinal gene delivery.
	^{October 5, 2018} Farewell– Emily Nettesheim heads to industry Emily Nettesheim, a research technologist within the OGTL, leaves today to pursue an exciting opportunity at Tenaya Therapeutics, a San Francisco-based biopharmaceutical company focused on developing therapeutics for heart failure. Everyone in the OGTL wishes Emily the best of luck in her new endeavor!
	^{October 1, 2018} Publication – OGTL collaborative research published in IOVS In collaboration with Dr. John Hulleman, study leader and Assistant Professor of Ophthalmology at UT Southwestern, OGTL researchers helped evaluate whether incorporation of a dihydrofolate reductase (DHFR) destabilising protein domain into a rAAV-expression cassette is able to effectively mediate changes in steady state protein levels within the retina. The study demonstrated that oral administration of the activating drug, trimethoprim (TMP), is able to rapidly and reversibly mediate changes in retinal protein expression following rAAV delivery without adverse events, making it potentially potent tool for the future development of inducible gene therapy treatments. Read the full article
	^{September 21, 2018} Grant – National Eye Institute Dr. Daniel Lipinski and the Ocular Gene Therapy Laboratory have been awarded a Research Project Grant (R01) from the National Eye Institute entitled ‘Vascular gene delivery and early disease biomarkers in diabetic retinopathy’. This five-year, $1.25M award focuses on developing and assessing technologies that enable gene transfer to cells of the retinal vasculature, dysfunction of which underlies the progression of diabetic retinopathy and vision loss in patients with diabetes mellitus.
	^{August 06, 2018} Publication – OGTL research published in Scientific Reports In an article published today, Chris Reid, PhD, a recent graduate from the Lipinski Laboratory, describes the design of an inducible recombinant adeno-associated virus (rAAV)-based gene therapy treatment to prevent choroidal neovascularization (CNV) in age-related macular degeneration (AMD). Traditional pharmacological approaches to control CNV formation in AMD necessitate the monthly intraocular injection of compounds to inhibit vessel growth, which are associated with numerous side effects, including cataract formation, eye infections and accelerated retinal atrophy. In this publication, we describe an alternate treatment strategy wherein expression of a vascular endothelial growth factor (VEGF) antagonist from an intravitreally injected recombinant adeno-associated virus vector can be induced on-demand, leading to effective prevention of CNV formation. If developed further, this strategy would represent a paradigm shift in the clinical management of AMD by eliminating the need for repetitive intravitreal injections whilst simultaneously increasing flexibility of dosing, leading to a fully personalized gene therapy treatment. Read the full article
	^{June 12, 2018} Congratulations – Dwani Patel receives Vision Research Trainee Grant The McPherson Eye Research Institute announced today that Dwani Patel, a second year PhD student in the OGTL, has been awarded one of only two Vision Research Trainee Grants for 2018. The $3,000 award will be used to help fund Dwani’s research into the development of laser speckle contrast imaging (LSCI) technologies to assess retinal vascular health in diabetes.
	^{June 01, 2018} Donation – Robert A. Brandt Macular Degeneration Fund The OGTL recently received a generous philanthropic donation of $50,000 from the Robert A. Brandt Macular Degeneration Fund to support the development of a gene therapy treatment to prevent vision loss in patients with age-related macular degeneration (AMD).
	^{May 11, 2018} Welcome back – Kristina Ertel joins the OGTL. Kristina Ertel, formerly a research technologist in the Lipinski lab, re-joins the OGTL as a graduate student. Kristina is a member of the Interdisciplinary doctoral program (IDP) whose research will focus on the development of a long-acting gene therapy treatment for primary open-angle glaucoma (POAG), a blinding disease that affects an estimated 65 million people worldwide.
	^{April 05, 2018} Grant – Foundation for Fighting Blindness. Dr. Daniel Lipinski and the Ocular Gene Therapy Laboratory have been awarded a Foundation for Fighting Blindness Individual Investigator Research Grant entitled ‘Maintaining proteostasis to prevent photoreceptor degeneration in retinal disease’. This award is intended to support on going research efforts to evaluate whether inhibiting proteolysis prevents the secondary degeneration of cone photoreceptors, which are essential for daytime and color vision, in the disease retinitis pigmentosa (RP). It is expected that this research will further the development of a broadly applicable gene therapy treatment to prevent vision loss in diseases like RP, where the underlying genetics are incompletely understood.
	^{April 03, 2018} Congratulations – Chris Reid receives the Meritorious Abstract Travel Award to attend the American Society of Gene and Cell Therapy 21st Annual Meeting. Dr. Chris Reid, a post - doctoral fellow, has been awarded a prestigious travel grant to attend this year’s American Society for Gene and Cell therapy Annual Meeting, to be held in Chicago, May 16-19, 2018. Chris will be presenting a paper on the ‘Effect of inducible VEGF - trap expression on CNV formation in a murine model of wet AMD following intravitreal administration of a capsid mutant rAAV - riboswitch vector’.
	^{March 27, 2018} Congratulations – Hanmeng Zhang passes her qualifying exam. Hanmeng Zhang, a second year graduate in the Neuroscience Doctoral Program NDP), successfully passed her qualifying exam in front of a committee consisting of Daniel M. Lipinski, DPhil, Brian A. Link, PhD, Matt Scaglione, PhD and Margaret Wong-Riley, PhD. Hanmeng will be continuing her research looking at the role of proteolysis in photoreceptor degeneration and the development of a broadly applicable treatment for retinitis pigmentosa, a severe degenerative disease that causes blindness in approximately 1 in 4000 people worldwide.
	^{March 08, 2018} Congratulations – Dwani Patel passes qualifying exam and thesis proposal. Dwani Patel, a first year graduate student in the Medical Scientist Training Program MSTP), successfully passed his qualifying exam defended his thesis proposal, which focuses on the development of imaging and gene transfer technologies to enable a gene therapy treatment for diabetic retinopathy. Dwani was examined by a five-member committee, consisting of Daniel M. Lipinski, DPhil, Joseph Carroll, PhD, Iris Kassem, MD, PhD, Judy Kim, MD and John Corbett, PhD.
	^{March 06, 2018} Congratulations – Chris Reid successfully defends his PhD thesis. In a public seminar Chris Reid highlighted some of his major research accomplishments before successfully defending his thesis, entitled ‘The Development of rAAV Technologies for Ocular Gene Therapy Applications’ to a committee consisting of Drs. Daniel Lipinski, Joseph Carroll, Iris Kassem and Jeffery Medin. In doing so, Dr Reid becomes the first student to graduate from the Ocular Gene Therapy Laboratory, completing an outstanding period of graduate research exemplified by the publication of four peer-review journal articles and book chapters, including three as lead author. We are delighted to announce that Dr Reid will remain in the OGTL as a Postdoctoral Fellow, allowing him to continue his ground-breaking research focusing on the development of novel gene therapy treatments for age-related macular degeneration and glaucoma.
	^{January 01, 2018} Publication – Chris Reid has a chapter published in the Methods in Molecular Biology Springer Protocols book series. In his upcoming book chapter, Chris Reid, a final year graduate student in the Cell Biology program, describes an optimized methodology for producing and purifying recombinant adeno-associated virus (rAAV) vectors on two different scales, including a micro-scale preparation that can be used for high-throughput screening of novel serotypes and expression constructs. Hardback copies of Methods in Molecular Biology Retinal Gene Therapy are expected to ship in early 2018 and the article is currently available online.