Huntington's Disease: A New Beginning for the Story

There is some hope in the form of an exciting gene therapy technique being pioneered by Professor Sarah Tabrizi at UCL. She's been running small clinical trials of antisense oligonucleotides, short genetic messages that cancel out the repetitive Huntington's gene so that the faulty protein doesn't get made. Other researchers are investigating whether new gene editing techniques like CRISPR could be used to snip out the extra repeats. Getting gene therapy and CRISPR tools into enough nerve cells in the brain to make a difference is a big challenge, but it seems to be the best approach on the table right now.