CRISPR for SMA: The Next Breakthrough in Treatment
Current disease-modifying treatments have dramatically changed the natural history of SMA, but researchers are still searching for approaches that could address the condition more permanently at the genetic level. One of the most exciting areas of SMA research is CRISPR-based gene editing, including newer techniques known as base editing. Instead of repeatedly increasing SMN protein or supplying an additional working gene, these experimental approaches aim to make precise changes to DNA that could allow the body to produce more functional SMN protein over the long term.
One major CRISPR strategy being studied for Spinal Muscular Atrophy focuses on repairing the backup SMN2 gene. SMN2 is very similar to SMN1, but a small genetic difference causes most SMN2 transcripts to exclude exon 7, resulting in much less functional SMN protein. Researchers have used CRISPR-derived base editors to correct this difference in SMA patient cells, with one study reporting up to 99% intended editing and increased production of functional SMN protein. Other preclinical research has shown that base editing of SMN2 in SMA mice improved motor function and extended lifespan, including stronger results when the experimental gene-editing approach was combined with nusinersen. These findings are promising because they suggest gene editing may someday offer a more durable way to correct the underlying biology of SMA rather than only compensating for it.
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