EP 240: Hijacking DNA repair machinery to treat Huntington’s disease with Vincent Dion of the UKDRI

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

01:48 Working on origins-of-life chemistry in Stanley Miller’s lab

04:13 Vincent’s path to genetics 

06:29 How somatic repeat expansion drives Huntington’s disease 

10:26 Therapeutic strategies for Huntington’s disease

16:53 Using gene editing to shrink repeat expansions

20:16 Optimizing CRISPR nickase delivery and expression for repeat expansion editing

27:32 Moving gene editing from academic research toward a first-in-human trial

29:55 Clinical trial challenges for Huntington’s gene therapies and uniQure’s early data

33:33 Epigenetic regulation of repeat instability in neurodegenerative disease

34:51 How basic science breakthroughs like CRISPR become transformative biology tools

37:52 How academic couples navigate the two-body problem when building research careers

43:02 Developing biomarkers to measure whether DNA-targeting therapies are working

45:54 Closing remarks

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