Question

SPENT 100 POINTS, NEED HELP ASAP!!!

My assigned technology is GENE THERAPY!

Include a real-life scenario or “case” to explain how the technology has or is currently being used. For example, for GMOs you might include the scenario of Golden Rice Project to increase vitamin A in communities exhibiting vitamin A deficiency, or for cloning, you might describe the first successful cloning of Dolly the sheep. Your case should include the following:


· When did the scenario/case happen?

· Where did the scenario/case happen?

· Explain the background/details of the case/scenario.

· How was the technology involved in solving the problem or case?

· 2-3 related pictures

* Info should be pretty short but enough to probably fit on one slide.

Answer 1

Scenario: First FDA-approved gene therapy for inherited blindness

When: 2017

Where: United States

Background: Leber congenital amaurosis (LCA) is a rare inherited retinal disease that typically leads to complete blindness in early childhood. In 2017, the FDA approved Luxturna, the first gene therapy to treat inherited vision loss caused by mutations in both copies of the RPE65 gene, which is associated with LCA.

How was gene therapy involved: Luxturna works by replacing the mutated RPE65 gene with a healthy version, using a harmless virus as a vector to deliver the gene to the retina. The treatment involves a one-time injection directly into the eye. The therapy has been shown to improve vision in clinical trials, with some patients gaining the ability to navigate obstacles and even read lines on an eye chart.

Related pictures:

Image of a child with Leber congenital amaurosis before and after treatment with Luxturna.

Schematic representation of the mechanism of gene therapy using a viral vector to deliver the healthy RPE65 gene to the retina.

Photograph of the Luxturna injection being administered to a patient's eye.