![Gene Therapy may Treat Progressive Blindness Gene Therapy may Treat Progressive Blindness](https://images.medindia.net/health-images/1200_1000/guidelines-for-heritable-human-genome-editing.jpg)
‘Novel OPA1-based gene therapy promises effective therapeutics in an eye disease that causes progressive visual loss and also neurological diseases like Alzheimer's and Parkinson's disease.’
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The scientists demonstrated a visual functional recovery in mice model of mitochondrial dysfunction – DOA. The OPA1-based gene therapy also enhanced the mitochondrial performance in human cells that had OPA1 gene mutations, thereby promising effective therapeutics in patients having these mutations.
"We used a clever lab technique that allows scientists to provide a specific gene to cells that need it using specially engineered non-harmful viruses. This allowed us to directly alter the functioning of the mitochondria in the cells we treated, boosting their ability to produce energy which in turn helps protects them from cell damage", says Dr. Maloney, Research Fellow, Trinity's School of Genetics and Microbiology
The novel gene therapy also signified benefits for a broad spectrum of diseases that involves mitochondrial dysfunction including neurological diseases like Alzheimer's and Parkinson's disease. This can help in formulating possible gene-based treatments to bring hope to the life of these patients.
Source-Medindia