Which of the following would provide the longest lasting treatment for Leber's congenital amaurosis II?
A)Replacing an enzyme that restores the function of T and B cells
B)Substrate reduction therapy and pharmacological chaperone therapy
C)Injecting adeno-associated virus carrying ornithine transcarbamylase (OTC)into T and B cells
D)Injecting adeno-associated virus carrying a wild type version of the RPE65 gene into affected cells of the retina

Question

Quizplus · Accepted Answer

Leber's congenital amaurosis II is caused by a mutation in the RPE65 gene, which regulates the production of a protein essential for vision. Injecting adeno-associated virus carrying a wild type version of the RPE65 gene into affected cells of the retina is a gene therapy that is safe and effective in treating LCA2, providing long-term benefits. The other options listed do not directly address the genetic mutation causing the disease and are not effective treatments for LCA2.

Which of the Following Would Provide the Longest Lasting Treatment