Gene Therapy In The Eye - Retinaresearchfnd

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Foresight for SightMarch 2018Issue Number 1 / Volume 40Retina Research Foundation Newsletter is published three times per year: Spring, Summer and Fall.Gene Therapy in the EyeGene therapy for inherited retinaldisease is showing much promisedue to several factors uniqueto the eye. The eye is a smallorgan that is easy to get to, soimplanting cells in them is mucheasier than elsewhere in the body.Importantly, the eye is what isimmune-privileged, which meansthat cells implanted there are lesslikely to be rejected as they mightbe in other parts of the body.According to Dr. Paul A. Sieving,Director of the National EyeInstitute, National Institutes ofHealth, what makes the eye “awonderful place to test therapyideas,” is that while it is obviouslyconnected to the rest of the bodyby blood and nerve tissue, “theeye is a separate compartment. Weonly need to deliver a microscopicquantity of gene vector in orderto try a treatment,” unlike othergene therapy experiments, inwhich the gene and the virus thatcarries it, known as a vector, cancirculate throughout the wholebody system.Another advantage is that in genetransfer trials involving the eye,outcomes will be crystal clear.“The visual system has veryprecise, quantifiable measures offunction, and even tiny measuresof success can be documented,”he ealth/Importance of Exercise in Preserving VisionIt has long been common knowledge that evenmoderate regular physical exercise is beneficial foryour heart and your well-being, but did you know thatit can also help protect your eyes? According to theAmerican Academy of Ophthalmology, two studieshave shown that people who exercise regularly wereless likely to develop serious, eye disease.GlaucomaIn one study, British researchers, led by Dr. Paul J. Fosterof the University College London (UCL) Institute ofOphthalmology, discovered that subjects who engagedin regular physical exercise over a 15-year periodwere less likely to develop glaucoma than people whowere inactive. In the study group of 5,650 British menand women over the age of 48, Dr. Foster reported a25 percent reduction in a key indicator of glaucomarisk. The study was published in InvestigativeOphthalmology & Visual Science (IOVS).For people who already have glaucoma, even abrisk walk three times a week was found to lowerintraocular pressure (IOP) and improve blood flowto the retina and optic nerve, but only as long as theexercise routine continued. “We cannot commenton the cause, but there is certainly an associationbetween a sedentary lifestyle and factors that increaseglaucoma risk,” said Dr. on page 2)

Importance of Exercise inPreserving Vision(continued from cover page)Paul Foster, MDMachelle Pardue, PhDAge-related macular degeneration (AMD)Researchers at Emory University School of Medicine in Atlanta, GA, andthe Atlanta Veterans Administration (VA) Medical Center in Decatur,GA, conducted studies that identify a relationship between level ofphysical activity and developing age-related macular degeneration(AMD) in mice. Brain-derived neurotrophic factor (BDNF) is a growthfactor that contributes to the health and well-being of neurons, whichare found in the retina aswell as other parts of thebody. Dr. Machelle Pardue,the senior author of the study,and Dr. Jeffrey Boatright, coauthor, found that exerciseraised levels of BDNF in themouse retina, potentiallyimproving retinal health.These experiments stronglysuggest that “exercise protectsvision, at least in mice, byincreasing BDNF in the retina,” said Dr. Boatright, an associateprofessor of ophthalmology at Emory. But whether exercise can preventmacular degeneration in human eyes is “impossible to know, basedon the data we have now,” said Dr. Pardue, a research scientist at theAtlanta VA. For now, those with a family history of retinal degenerationmight want to discuss an exercise program with their doctor. This studywas published in the Journal of Neuroscience.www.nytimes.comDiabetic RetinopathyMany eye diseases are linked to other health problems such as diabetesor high blood pressure. Controlling the underlying disease leadsto improved outcomes for your eyes. For example, in patients withdiabetes, exercise can help in disease management and therefore reducethe risk of developing complications, including diabetic retinopathy.Jeffrey Boatright, PhDThe best news about exercise and your vision is that you earn the benefitswith even a moderate regimen of physical activity, even 30 minutes aday, and the benefits continue as long as you maintain your more activelifestyle. Ask your doctor about the right exercise program for

Researchers Discover CriticalTrigger for InflammationA University of Virginia School of Medicine team of researchershas recently discovered an enzyme that plays a critical role incausing the inflammation that can ultimately lead to blindness.Dr. Jayakrishna Ambati, Professor & Vice Chair for Research ofOphthalmology, and Dr. Nagaraj Kerur, Assistant Professor, haveidentified that an enzyme called cGAS is the culprit. This enzymeplays an important role in the body’s immune response to infectionsby detecting foreign DNA, but now this molecule has been newlyidentified to also play a key role in the dry form of age-relatedmacular degeneration.“It’s really surprising that in macular degeneration, which, as faras we know, has nothing to do with viruses or bacteria, that cGASis activated, and that this alarm system is turned on,” Dr. Ambatisaid. “This is what leads to the killing of the cells in the retina, and,ultimately, vision loss.”Jayakrishna Ambati, MD“For the first time, we know in macular degeneration what is oneof the very first events that triggers the system to get alarmedand start, to use an anthropomorphic term, hyperventilating. Thisoverdrive of inflammation is what ultimately damages cells, and so,potentially, we have a way of interfering very early in the process,”said Dr. Ambati.The finding may lead to methods of halting the inflammation earlyon and potentially preserving sight. Researchers are already tryingto create drugs that could inhibit the function of this enzyme. TheUVA researchers expect that the development and extensive safetyand efficacy testing of any drug to inhibit cGAS will take years.The scientists also hope to develop a way to measure the levelsof the enzyme in patients’ eyes in order to determine when best toadminister a treatment that blocks cGAS. “If they have high levels ofthis enzyme in their eye, they might be a wonderful candidate for thissort of treatment,” Dr. Ambati said. “This is really precision medicineat the single-molecule level.”Nagaraj Kerur, Butler Boulevard, Houston, Texas 77030 (713) 797-1925e-mail: www.retinaresearchfnd.orgRetina Research Foundation is dedicated to the eradication ofretina disease through programs in research and education.3

Gillingham Pan-AmericanFellowship ReportIn the fall issue of RRF’s newsletter, we reported that Dr. AndreaElizabeth Arriola-López was completing her fellowship at BascomPalmer Eye Institute in Miami, FL. Bascom Palmer is one of the top eyehospitals in the country, and she had many opportunities while there toadvance her knowledge and expertise. Dr. Arriola has now returned toGuatemala City, Guatemala, to teach and treat patients. RRF is proud tosponsor this program in affiliation with the Pan-American Associationof Ophthalmology.The following is an excerpt from her report:Andrea Arriola-López, MDI attended the Bascom Palmer EyeInstitute in Miami, Florida, USA,for six months under the direction ofThomas Albini, MD, and EduardoAlfonso, MD, in the Uveitis Clinic;previously, I spent three years inMexico City; one year as a researchretina fellow and two years as aretina fellow.While at Bascom, I was able toobserve and partici e entire eye. This innovativetechnology could make eye examinations faster for patients byavoiding the need to undergo imaging with multiple instrumentsto look at different areas of the eye. Dr. Ireneusz Grulkowski atNicolaus Copernicus University in Poland and Dr. Pablo Artal at theUniversidad de Murcia in Spain collaborated to develop this newimaging system.“Diseases such as glaucoma affect both the frontand back portions of the eye,” said Dr. Grulkowski.“An instrument that can examine the whole eyewill improve the patient’s experience becausethey won’t have to go through imaging withdifferent devices.”In Optica, the journal of the Optical Society,these researchers show that their new opticalcoherence tomography (OCT) imaging systemis able to image in great detail both thefront and the back of the eye, and also the interfaces of the eye’svitreous gel with the retina and lens. This newimaging capability could allow scientists to betterunderstand how the vitreous gel that fills the eyeinteracts with the retina.“We also want to use our instrument to measureopacities in the eye’s crystal lens and the vitreousto better understand how various parts of theeye affect the deterioration of vision,” saidDr. Grulkowski. “We believe that the ability tomeasure these opacities and other properties ofthe eye that couldn’t be examined before willopen up many new ophthalmology applicationsfor OCT.” Artal, PhD7

FREE MATTERFOR THEBLIND ORHANDICAPPED1977 Butler Boulevard, Houston, Texas 77030 (713) 797-1925e-mail: www.retinaresearchfnd.orgEditor in Chief:Alice McPherson, M.D.Managing Editor: Margaret FareseRetina Research Foundation Is a Nonprofit Organization.CHANGE SERVICE REQUESTEDPlease indicate changes in boxes and make anycorrections needed next to your name and address,then clip and return entire address label in an envelope.Change name or address as shown on addressRemove name from mailing list.Special RemembrancesIN MEMORY OFMayde ButlerRhett Butler Charitable FoundationSid GilbertMary BermanAnthony MierzwaRhett Butler Charitable FoundationAlan KurtzSarah SalisburyWilliam and Susan BarrowBaird FoundationEMPLOYEE GIVINGYourCauseAT&T employee (anonymous)BenevityGoogle Inc.MicrosoftANNOUNCEMENT: RRF is a registered donor organization with AmazonSmileWhen you shop on Amazon, you are now able to automaticallysend a donation to Retina Research Foundation.How?1) Shop at and choose RRF as your charity of choice.2) You can also access the link through RRF’s website at retinaresearchfnd.org3) AmazonSmile Foundation will donate 0.5% of the purchase price of eligible productsto RRF. There is no additional cost to you - Amazon makes the donation on your behalf.4) After selecting RRF as your charity, every AmazonSmile purchase you make triggersan Amazon automatic donation to RRF. It’s that simple!RRF accepts credit cards for donations securely online at www.retinaresearchfnd.orgCall the office for more information: 713-797-1925Additional memorials received will appear in the next issue.

Palmer Eye Institute in Miami, FL. Bascom Palmer is one of the top eye hospitals in the country, and she had many opportunities while there to advance her knowledge and expertise. Dr. . including grand rounds

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