Multiple Choice
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
Correct Answer:

Verified
Correct Answer:
Verified
Q14: Tyrone has Gaucher disease.He receives recombinant glucocerebrosidase
Q15: A nasal spray for cystic fibrosis patients,which
Q16: Unlike cholesterol checks and chest x-rays,the results
Q17: Performing gene therapy on somatic cells removed
Q18: The defect in Canavan disease that causes
Q20: The Genetic Testing Registry of the U.S.National
Q21: "Preconception comprehensive carrier screening" is a test
Q22: A genetic counselor might discuss assisted reproductive
Q23: Which is an example of a germline
Q24: A test offered on the Web by