A recent article from STAT news (1) relayed the story of Jaci, a young woman in Iowa dying from a rare form of ALS, and her parents heroic efforts to help her get access to an untested drug.
Interestingly, the drug is an antisense oligonucleotide. Antisense oligos are short pieces of DNA, that bind to complementary molecules of mRNA and target them for destruction. These types of drugs are interesting because they can prevent the production of toxic proteins.
In Jaci’s case, her form of ALS results from a toxic version of the FUS protein. Normally, FUS is... Read more