Antisense oligonucleotide or ASOs are short oligonucleotides that can modify gene expression in the nervous system. ASOs can knockdown (reduce) or upregulate (increase) protein expression, depending on the functional consequence of the mutation and how to correct it.
ASOs may be particularly useful for mutations that are dominant negative which adversely affect the wild type (or healthy gene) product in the same cell as they can “knockdown” such mutations and eliminate the adverse effects. To date, the FDA has approved about ten ASO treatments for genetic disorders and there is a precedent for such treatments to go from idea to injection in under one year.
This means Henry could be treated with an ASO in the next 12-18 months, changing his life forever.
We are hopeful that an ASO is not only the most targeted and fastest treatment we could develop for Henry due to his specific mutation, but that it could later be combined with additional therapies as they are discovered. In addition, we plan to use the research we gain in developing this ASO for Henry to develop ASO’s for all those affected by AHC.
Please help us blaze this path in our race to treat our baby boy before his brain and life are impacted forever.