Gene Therapy to Prevent BlindnessBy: Ashley Nicole and Alex Gadda
Eye diseases such as diabetic retinopathy and age-related macular degeneration are among the leading causes of irreversible vision loss and blindness worldwide. Currently, gene therapy can be administered to treat these conditions — but this requires an injection. Now researchers report in the journal ACS Applied Materials & Interfaces a new way to deliver the treatment topically, without a needle. Eye diseases that can cause blindness can be hard to treat, but a new therapy via eye drop could be on the way.
Scientist found that loss in blindness is caused by a mitochondrial Disease. They have their own DNA, and its mutations within this mitochondrial DNA (mtDNA) that lead to LHON, Leber hereditary optic neuropathy, as well as a host of other diseases. University of Miami Miller School of Medicine laboratory is among the first to develop an approach that can target mtDNA in living mice and people. The global impact of LHON is unknown. In England, the estimated prevalence is about 1 in 30,000. The trial is recruiting LHON patients who fit into three categories — those with chronic vision loss in both eyes, with recent-onset vision loss in both eyes, or with recent-onset vision loss in one eye but no signs of abnormal vision in the other eye.
Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed onto future generations. This approach is known as germline gene therapy. germline gene therapy could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. people who would be affected by germline gene therapy are not yet born, they can’t choose whether to have the treatment.
The impact Gene Therapy has on us, is that it helps people to see, read, and write. gene therapy has helped people see things with color too. A man said that trees and flowers seemed more vivid. Other patients said that they were able to see more light , read more letters and numbers, and see stars at night. The patients also became more optimistic. Gene therapy has helped many people and will continue to help people.
Our prediction is that if Gene Therapy would become more widespread, people that have blindness would have more opportunities. They would be able to do things that they were not able to do. They could also see the things that they thought they could never see. Others might be able to see other humans beings for the firs time. If this type of therapy were known everywhere, people could have more opportunities, and could benefit from it.