Help Support Research to Cure Blindness and Vision Loss
There has never been a more exciting time in eye research than today. The hope and promise of developing gene therapies to restore vision is no longer science fiction. It is no longer a question of if these therapies will be developed, but when. As the Center where patients from all over the world turn for hope and answers, Mass. Eye and Ear and the Ocular Genomics Institute are urgently committed to accelerating gene therapy research toward cures.
But we cannot do it alone: We need your help. Cutting edge gene therapy research is very expensive, and the pace of progress will directly relate to the investment in research. Every dollar raised will help to move the field forward in bringing sight-restoring treatments to the children and adults who await them.
You can help. To support the Ocular Genomics Institute’s research, please visit Mass. Eye and Ear’s giving page: https://www.masseyeandear.org/online-donation-form. Note: Please select Other in the Gift Designation drop-down menu, and then enter Ocular Genomics Institute in the Donation Options Comments section, to make a donation.
Two million people worldwide suffer with vision loss and blindness from inherited retinal disease. Retinitis Pigmentosa (RP) is the most common of these diseases. RP progresses over decades, with patients first experiencing night blindness, then peripheral vision loss, and eventually loss of central vision, resulting in complete blindness. Leber congential amaurosis (LCA) is the severest of all inherited retinal diseases. LCA presents in the first year of life with profound blindess. These are just two types of conditions on the spectrum of inherited blinding diseases. They all have one thing in common – each results from a single gene defect – just one bad gene.
If one bad gene causes blindness, why don’t we replace that gene with a healthy one? This is precisely the goal of gene therapy. There are over 250 known genes that cause inherited retinal disease. An abnormality in any one of these genes may cause vision loss and blindness. Gene therapy is designed to improve vision by delivering a new healthy gene to override the faulty one. To date, experimental human trials in gene therapy have proven to be safe and effective and have improved vision in patients.
Currently, the most effective way of achieving this is to use a vector or virus that is engineered to safely deliver a correct copy of a gene to the particular cell of interest. Vectors are continuously improved to be safer for clinical use, target more cells, be made more efficient in order to treat diseases better, and to be made bigger, so they can transport larger genes, as many disease genes exceed the capacity of current vector technologies.
Please consider joining the Ocular Genomics Institute and Mass. Eye and Ear in the efforts to cure and prevent blindness and give a gift today.