Harness Therapeutics Publishes HTT CAG Repeat Sizing Methodology Code on GitHub
At Harness Therapeutics we are passionate about driving innovation in neurodegeneration. As part of our Huntington Disease (HD) program we are pursuing a therapeutic approach aimed at slowing somatic expansion of CAG repeats in the HTT gene, which is widely recognized as the underlying driver of the disease.
A key challenge for those working to develop therapies to slow down somatic expansion is recapitulating this complex process in vitro, and establishing assay systems to accurately quantify it. As part of our work to develop MISBATM (MicroRNA Steric Blocking ASOs) constructs to upregulate FAN1 and slow somatic expansion, we have developed a number of iPSC-derived neuronal models (including organoids) to allow us to determine the effectiveness of our drug candidates in slowing down the DNA expansion process. We then apply a unique methodology that calculates the number of CAG triplet repeats in the HTT repeat tract. This technique utilises nanopore sequencing together with a proprietary bioinformatics platform to enable fast and accurate determination of CAG length in neurons.
In the spirit of collaboration, we are pleased to make the code for our HTT CAG repeat sizing methodology publicly available on GitHub. By doing this, we aim to support and accelerate progress across the HD research community.
We look forward to seeing how fellow researchers apply this cutting-edge technique to further our collective understanding of HD and contribute to the development of transformative therapies.
The code is available here.