Gene Therapy Reverses Childhood Blindness


One form of childhood blindness now has a cure called gene therapy. This type of gene therapy injects altered viruses into the patient's eyes. The altered viruses carry healthy genes into the retina, improving their sight.

After treatment, 90 percent of the people had improved vision. Patients described this opportunity as giving them an average life. Misty Lovelace, age 18, said, "I can honestly say my biggest dream came true when I got my sight. I would never give it up for anything. It was truly a miracle. "

While gene therapy is helpful, it has many risks. If the treatment goes wrong, the patient could have permanent damage to their sight. Another risk could be death; some people die during gene therapy attempts. Besides these risks, there is a 1 million dollar cost per person. There are very few resources to lower the treatment's cost. Because of this many people are not willing or able to undergo this treatment.

The Food and Drug Administration (FDA) had to make a choice whether or not to support gene therapy treatment. Although this form of childhood blindness now has a treatment, the FDA has to consider the advantages and disadvantages to it. After discussing, they decided to support gene therapy. Many scientists that were working on this were excited about gene therapy and are now looking forward to working with it.

While gene therapy treatment can significantly improve affected individuals' vision and lives, the decision to receive treatment is difficult due to these risks.

 

[Source: NPR]

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