Final Video review for Genes as medicine

Learn about gene therapy for blindness, from viral vectors to animal models, in this science review.

Soon after a gene associated with childhood blindness was discovered in the 1990s, Drs. Bennett and Maguire were able to offer a gene therapy treatment for children. true false In the film you met Molly, who has a form of childhood blindness called Leber amaurosis (also called Leber congenital amaurosis, or LCA). Her blindness is caused by _ early exposure to intensely bright light an eye injury she received at a young age. mutations in a gene that is necessary for maintaining sight. a viral disease in her nervous system that moved into her eyes. For many genetic diseases, children inherit the disease-causing mutations from their parents. What was the likely inheritance pattern in the case of Molly’s form of childhood blindness? _ Only females can have Leber amaurosis, so Molly must have inherited the genetic mutation only from her mother. Molly became blind from new mutations that occurred in her eye as a baby. Molly ended up with childhood blindness because both of her parents were blind when they were children. Molly inherited a mutation in the same gene from each of her parents. Which statements are reasons that eyes are a good target for clinical trials related to genetic medicine? I. Eyes are easy to access.II. Eyes are the least important of the five senses.III. Eyes in humans are identical to eyes of other species used in animal models.IV. One eye can be treated while the other can act as an experimental control. I and II only II and III only III and IV only I and IV only The gene therapy technique designed by Drs. Bennett and Maguire to cure Molly’s blindness involved using a virus as a vector to deliver the corrective gene into one of her eyes. What necessary step must be taken before viruses are used in this way? _ Patients must first take flu medicine to help them avoid any flulike symptoms that the virus causes. The virus’s harmful genes and the ones the virus needs to replicate must be removed Patients like Molly must first receive a vaccine for the virus so that they don’t get the disease that the virus carries Doctors must first snip out the defective gene from a patient’s photoreceptor cells before using the virus to deliver the corrective gene. As a final test of their technique to cure Leber amaurosis, Drs. Bennett and Maguire needed a large animal model that was similar to humans. What animal did they choose? Cat Dog Mouse Elephant Some reasons that this animal was a good model for testing a cure for childhood blindness is because Dog eyes are large and about the same size as human eyes Just like humans, the Briard shepherd can suffer from Leber amaurosis caused by mutations in the RPE65 gene, similar to humans Dog eyes and human eyes have many of the same anatomical features. Dogs are visual animals like humans, and testing their ability to see requires little more than watching their behavior. Mutations to the RPE65 gene can cause Leber amaurosis. Why is it a mistake to call RPE65 “the Leber amaurosis gene”? It's not a pair of blue genes it's not wrong to call it the Leber amaurosis gene RPE65 codes for a protein that is missing in an individual that shows Leber amaurosis