Zolgensma Rewrites Your DNA: Here's How
Although Zolgensma is sometimes described as “rewriting DNA,” that phrase is not scientifically exact. Zolgensma, also known as onasemnogene abeparvovec-xioi, is a gene replacement therapy that delivers a new working copy of a gene encoding SMN protein rather than editing or repairing the patient’s original SMN1 gene. The FDA describes Zolgensma as an AAV9-based gene therapy designed to deliver a copy of the gene encoding human SMN protein, allowing treated cells to produce the protein that is deficient in SMA.
Zolgensma works by placing a functional SMN gene inside a modified adeno-associated virus called AAV9, which acts as a delivery vehicle. The viral DNA normally responsible for the virus is removed, and the therapeutic SMN gene is placed inside instead. After Zolgensma is given as a one-time intravenous infusion, the AAV9 vector travels through the body and delivers the working gene to cells, including motor neurons. Those cells can then use the added genetic instructions to produce more SMN protein, helping support motor neuron survival and muscle function. Official Zolgensma information explains that the therapy replaces the function of the missing or nonworking SMN1 gene rather than changing the defective gene already present in the person's chromosomes.
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