The disease Leber congenital amaurosis (LCA) , caused by loss-of-function mutation of a gene called RPE65, has been treated with gene therapy by injecting recombinant AAV vectors containing a normal copy of the RPE65 gene into their retinal epithelial cells.Patients receiving this therapy
A) may acquire detrimental mutations due to integration of the virus.
B) may need to repeat the treatment as the viral DNA is degraded.
C) will pass the mutation to their offspring.
D) now have the normal RPE65 gene in all cells.
Correct Answer:
Verified
Q18: A trait unique to embryonic stem cells
Q19: Although bacteria can be used to produce
Q20: A researcher has a laboratory strain of
Q21: Fragile X syndrome is a human disease
Q22: Using the CRISPR/Cas9 system, a genetically-engineered sgRNA
Q23: To generate a knockin mouse, a scientist
Q24: A scientist might create a conditional knockout
Q25: Huntington disease is caused by expansion of
Q27: To create a knockout mouse, after introducing
Q28: The FDA-approved GM Atlantic salmon have a
Unlock this Answer For Free Now!
View this answer and more for free by performing one of the following actions
Scan the QR code to install the App and get 2 free unlocks
Unlock quizzes for free by uploading documents