This week on The Genetics Podcast, Patrick is joined by Dr. Vincent Dion, Group Leader at the UK Dementia Research Institute at Cardiff University. They discuss how repeat expansions drive Huntington’s disease and other neurological disorders, why DNA repair can sometimes worsen these mutations over time, and how CRISPR nickase editing could offer a new way to shrink disease-causing repeats rather than simply silence them.
Show Notes
0:00 Intro to The Genetics Podcast
01:00 Welcome to Vincent
01:45 Working on origins-of-life chemistry in Stanley Miller’s lab
03:56 Vincent’s path to genetics
06:00 How somatic repeat expansion drives Huntington’s disease
09:40 Therapeutic strategies for Huntington’s disease
15:29 Using gene editing to shrink repeat expansions
18:31 Optimizing CRISPR nickase delivery and expression for repeat expansion editing
25:15 Moving gene editing from academic research toward a first-in-human trial
27:31 Clinical trial challenges for Huntington’s gene therapies and uniQure’s early data
30:47 Epigenetic regulation of repeat instability in neurodegenerative disease
33:14 How basic science breakthroughs like CRISPR become transformative biology tools
34:55 How academic couples navigate the two-body problem when building research careers
39:39 Developing biomarkers to measure whether DNA-targeting therapies are working
42:17 Closing remarks
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