Annual Ataxia Conference – Day 2

Day two was really great! I don’t have the strength to write everything, but many more scientific as well as ordinary life issues were raised. Finding a cure for ataxia comes down to two approaches – looking for the drug, the substance, and/or helpful genetic therapy that could modify the abnormal genome. Dr. Marie Davis’s speech was extremely interesting because, using wonderful analogies, she explained what the leading trends in genetic therapies are all about. Basically there are three: Antisense oligonucleotides (ASO) Viral (AAV – mediated gene replacement CRISPR Cas 9 Genome Editing I suspect that someday I will write