Cameron Glasscock
Rice University, TX·Biochemistry
Some stretches of DNA consist of a short sequence repeated over and over, and in more than 60 inherited disorders these repeats grow abnormally long. Huntington's disease, myotonic dystrophy and spinocerebellar ataxia are among them, and they cause neurological and other serious health problems.
- Fields
Biochemistry
Genetics
Biomedical Engineering
- Topics
repeat expansion disorders
Huntington's disease
myotonic dystrophy
- Grants
- 1 known grant, through Jul 2029
What they work on
- Use AI-based computational protein design to create DNA-binding proteins that selectively recognize the hairpin and bulge structures formed at slip-outs of pathogenic expanded repeats, rather than linear sequence.
- Fuse these designed binders to strand-selective nickases, enzymes that cut a single DNA strand, to test the hypothesis that slip-out selective nicking induces contraction of expanded repeats.
- Engineer slip-out selective cytosine base editors from cytidine deaminase enzymes that act on single-stranded DNA, to introduce repeat interruptions that stabilize the duplex and slow somatic instability.
- Test whether the slip-out selective tools induce repeat contractions or slow somatic instability in vivo in a Huntington's disease knock-in mouse model.
More about the research
A feature shared by many of these repeat expansion disorders (REDs) is somatic instability: the repeat keeps lengthening in a person's cells during life, and in Huntington's disease symptoms appear to begin once repeat length crosses a toxic threshold. Stopping that ongoing expansion is therefore an attractive therapeutic strategy, but few approaches can do so without also shutting down DNA repair enzymes that cells need for normal functions.
Why it matters
Patients with Huntington's disease, myotonic dystrophy, spinocerebellar ataxia and dozens of other repeat expansion disorders currently have no treatment that addresses the ongoing growth of the repeat itself. Tools that recognize the distorted DNA structures these repeats form could offer a gene therapy strategy applicable across many of these conditions, and would more broadly give researchers a way to bind aberrant DNA structures that sequence-based technologies cannot reach.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Xiao Wang
University of Pennsylvania, PA·Genetics
Test whether adenine base editors, which chemically rewrite a single DNA base without cutting both strands, can correct the PAH R408W variant in cultured human liver cells and restore phenylalanine hydroxylase activity.
Caroline Dias
University of Colorado Denver/Anschutz Medical Campus, CO·Genetics
Profile cell-type-specific gene expression in post-mortem brain tissue from individuals with Fragile X syndrome, using single-cell sequencing.
Qinglan Ling
University of Massachusetts Chan Medical School, MA·Genetics·Early-career
Develop and optimize a mitochondria-targeting base editor, a tool that changes single DNA letters, to create a truncating mutation in the mouse MTATP6 gene and generate a conditional mouse model.
The school
Rice University: 24 labs on PI Pages, $278M a year on research, 267 research doctorates a year.
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