How an ASO works

Like all of us, Ivy has two copies of the GRIA2 (glutamate receptor) gene. One of her copies is completely healthy and working fine. The other copy has the harmful, toxic mutation. Because of how essential GRIA2 is for brain function and development, Ivy needs an “allele-specific” ASO that will just turn down the toxic GRIA2 copy and let the healthy copy do its job. This ASO is not a one-time repair of the typo in Ivy’s genetic code. It is a treatment that she will get every few months for the rest of her life to keep the toxic GRIA2 copy in check.