IVR researchers recently published an article presenting the development and evaluation of a gene therapy vector for RPGR, the 3rd most common cause of all inherited retinal disease. This gene is a challenging therapeutic target, because gene therapy vectors containing wild-type RPGR sequence, which includes the highly repetitive low complexity region ORF15, are extremely unstable. They exhibit accumulations of genomic changes during the process of propagating the viral plasmid.
Development of a Gene Therapy Vector for the Treatment of RPGR-associated X-linked Retinitis PigmentosaMay 20th, 2019
May 6th, 2019
A group of IVR researchers have published the first single-cell investigation of central macula versus peripheral retina, from three human donors.
April 5th, 2019
A team of IVR researchers published their successful correction of disease-causing mutations in patient-derived induced pluripotent stem cells (iPSCs). Enhanced S-cone syndrome (ESCS) is caused by recessive mutations in the photoreceptor cell transcription factor NR2E3. The loss of functional NR2E3 results in reduced populations of rod photoreceptors, due to a large increase in the population of photoreceptors tuned to identify short-wavelength (blue) light.
January 16th, 2019
The University of Iowa and the UI Institute for Vision Research announced today that Alice and John Butler of Dubuque, Iowa, have made a $5 million donation to accelerate research and treatments for blinding eye diseases that affect millions of people worldwide. Their gift will establish the Alice L. and John E. Butler Vision Research Fund, which will support gene- and stem-cell based treatments for degenerative retinal diseases.
August 9th, 2018
Monday night, at Sam’s Scramble for Sight in Grand Rapids Michigan, the Advisory Board of the Institute for Vision Research announced the creation of an annual “Heart of Our Vision Award” to recognize someone in our organization who has made an extraordinary contribution to the cure of heritable blindness. This year, Dr. Budd Tucker was chosen as the first recipient of the award and was presented with a Fender Telecaster guitar autographed by Bruce Springsteen to commemorate the event.
July 13th, 2018
The Kimberling Usher Research Laboratory in the Institute for Vision Research is pleased to announce an increase in our campaign goal to $10 million. This increase is possible because addiitonal major donors have joined the "challenge side" of the matching effort so that we can now match every gift for Usher Syndrome Research, dollar for dollar, until $10 million is raised.
December 27th, 2017
The pharmaceutical company Spark Therapeutics recently announced FDA approval1,2 of a gene therapy known as Luxturna, which has been shown to improve vision in patients with an extremely rare inherited eye disease caused by mutations in the gene RPE65. Scientists estimate this disease to occur in less than one in 500,000 people – or about 560 people total in the United States3,4.
The company has priced the drug at $425,000 per eye ($850,000 per patient), which does not include the cost of the surgery5.
November 30th, 2017
Scientists at the WIVR recently published a report in which they tested the efficiency and retinal cell type targeted by 7 different serotypes of adeno-associated virus (AAV) in the human retina. This paper is significant as most testing of AAVs is performed in animal models such as mice, rats, pigs and monkeys. In this report, WIVR investigators developed a protocol for culture of human donor retinas that allowed for the assessment of AAVs ex vivo.
October 24th, 2017
A new publication from the laboratory of Val Sheffield, M.D., Ph.D., focuses on the role of a specific protein complex found in photoreceptor cells and its role in retinal degeneration in a blinding disorder known as Bardet-Biedl syndrome (BBS). This protein complex is called the BBSome. Ying Hsu and Janelle Garrison, a graduate student and postdoctoral fellow respectively, who performed much of the work, demonstrated that the BBSome is indispensable for the initial formation and continued maintenance of the outer segment, the light sensing component of photoreceptor cells.
October 5th, 2017