A Treatment for Henry
While there historically has not been a treatment for AHC, Henry’s parents have discovered a treatment for Henry with the support of his medical team at Boston Children’s Hospital. After developing this treatment, called an ASO, doctors would be able to use it as a blueprint to treat others with AHC and other rare neurological diseases.
Antisense oligonucleotides, or ASOs, are short strings of DNA or RNA letters that can bind to the mRNA to 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.
We believe a knockdown ASO will be most useful to treat Henry as they may be particularly useful for mutations that are dominant negative or which make a toxic protein which adversely affect the wild type (or healthy gene) product in the same cell. These ASOs 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.