How ASO Research May Help Nano-Rare ALS Patients
There has been increasing interest in the research and development of antisense oligonucleotides (ASO) therapies for treating neurodegenerative disorders such as ALS. These novel oligonucleotide therapies may even be able to target “nano-rare” ALS patients — those who carry an extremely rare genetic mutation associated with the disease.
The proportion of ALS cases carrying a known genetic mutation is low. Thus, ASOs are particularly good news for ALS patients who need hyper-personalized therapies that aren’t commercially viable to produce since they’re only helpful to a handful of people.
What is Nano-Rare ALS?
Most cases of ALS are sporadic ALS (sALS), in which patients have no known family history of the disease. Inherited (or familial) ALS (fALS) accounts for just 5% to 15% of cases.
While gene therapies are under development, they aren’t typically relevant to nano-rare ALS patients because their genetic mutations are so uncommon that they may be one of up to just 30 people worldwide who carry it. In these cases, only highly targeted treatments can keep these mutations from causing disease.
Now, an initiative called Silence ALS — a cooperative venture between Columbia University and the n-Lorem Foundation, with a funding grant from Target ALS — addresses these rare gene mutations.1 Better yet, the goal is to find treatments for these patients before the onset of symptoms. And the key is expanding research on the class of therapeutic compounds called antisense oligonucleotides (ASOs).
What are ASOs?
Antisense oligonucleotides are short, single-stranded molecules of DNA. They’re designed to be absorbed into cells in order to attach to messenger RNA (mRNA).
