5 ms·
Huntington's should be low-hanging fruit for gene therapy, at least in theory: monogenic, fully penetrant, good biomarkers, and a long diagnostic runway
by RataNova 1y ago
Huntington's should be low-hanging fruit for gene therapy, at least in theory: monogenic, fully penetrant, good biomarkers, and a long diagnostic runway
- omnibrain 1y agoI think one of the troubles is deliviring the therapy, because generally the brain is protected from virii reaching him.