Genetic Engineering (non-infectious disease)

Question 1

What does CRISPR treat?

Answer 1

Can treat non-infectious diseases that are the result of a single gene mutation.

Question 2

What does CRISPR do?

Answer 2

Has the ability to precisely cut out the infected gene and replace it with a working copy.
For some diseases the damaged gene could be replaced and the symptoms eliminated if enough healthy cells are produced.

Question 3

What does Luxturna treat?

Answer 3

Treats people with vision mutations in both copies of the RPE65 gene (which makes proteins for normal vision).

Question 4

What does Luxturna do?

Answer 4

Using gene therapy, it delivers the functional gene (RPE65) to produce protein to restore normal vision.
It can slow the progression of vision loss.