NeuroGenetics
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Gene and Molecular Therapies in Neurogenetics
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Question 1
Which statement correctly distinguishes gene addition therapy from gene editing for neurological disorders?
A
Gene addition integrates into the genome; gene editing uses episomal vectors
B
Gene addition delivers a functional gene copy that remains episomal in neurons; gene editing modifies the genomic DNA sequence at a specific locus
C
Gene editing is suitable for recessive disorders; gene addition is used only for dominant conditions
D
Both gene addition (AAV) and CRISPR gene editing permanently modify the patient's germline
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