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.
B) may need to repeat the treatment as the viral DNA is degraded.
You might also like to view...
While our personal genomes can identify genetic risk factors, disease risk is often greatly increased by (select all that apply)
A. diet. B. exercise. C. exposure to drugs and other chemicals in our environment. D. exposure to ultraviolet radiation. E. none of these
Which one of the following statements about the use of synthetic polynucleotides in cracking the genetic code is not true?
A. Poly(A) RNA directs the synthesis of only polylysine in vitro. B. Particular codons are generated in predictable ratios with random RNA polymers containing specific ratios of different nucleotides. C. Three different amino acids are incorporated into peptides when synthetic polymers of only one nucleotide are used to synthesize the RNA polymer. D. The codon table was completed by using RNA polymers of defined sequences.
Which of the following is NOT a function of bile salts?
A. aid the enzyme lipase in the digestion of fat molecules B. act as emulsifiers C. production of vitamin D D. needed for fat digestion
Because most birds travel through the air at high speeds, they must have
a) highly sensitive sensory systems. b) eggs and no placenta. c) a crop and a gizzard. d) complex songs. e) a three-chambered heart.