Researchers find key to cure blindness

test It has been led by Dr. David Gamm and has been published in the American Journal of Human Genetics and this line of work could lead to healing an incurable disease. “data-reactid =” 12 “> A team of researchers from the University of Wisconsin-Madison have published a paper trial to cure blindness from hereditary macular degeneration, a condition that was not curable to date. The trial has been led by Dr. David Gamm and has It has been published in the American Journal of Human Genetics and this line of work could give way to cure an incurable disease.

blindness total over the course of a few decades. This disease has a clear hereditary component and is caused by more than 2,000 mutations of the BEST1 gene. “Data-reactid =” 24 “> Specifically, the team has focused on vitelliform macular dystrophy, also known as BEST disease , and which causes a gradual loss of vision from childhood to total blindness over the course of a few decades.This disease has a clear hereditary component and is caused by more than two thousand mutations of the BEST1 gene.

mutations that have been tested, but not in all, as the team itself highlights. Specifically, the work has been based on the application of the CRISPR-Cas9 gene in the mutated genes. “Data-reactid =” 25 “> Researchers have approached this incurable disease in a different way from those proposed to date: they have corrected degeneration by placating the mutated genes with functional copies of BEST1. This approach has been successful in most, but not all, of the mutations that have been tested. Specifically, the work has been based on the application of the CRISPR-Cas9 gene in the mutated genes.

genetics requires high precision when substituting faulty genes for functional ones, as highlighted in the trial. “data-reactid =” 26 “> What is the origin of Best’s disease? The BEST1 gene contains a protein that It affects a layer of the retina known as RPE, so that by inheriting this defective gene from one of the parents, the person will be affected by this disease and will be doomed to blindness. This gene therapy requires high precision to when substituting faulty genes for functional ones, as highlighted in the trial.

Are we close to the cure for Best’s disease? The short answer is no, but the hope is that, in theory, a new avenue has been opened to apply healing to real patients. “We were able to reduce disease across the gene lines,” confirms Gamm, and the good news does not end here: This approach to the problem could be applied to other diseases caused by genetic mutations that affect tissues.

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