Piotr Hadaczek
University of California-Berkeley, CA·Neuroscience
Huntington's disease and C9orf72-linked amyotrophic lateral sclerosis (C9-ALS) are inherited brain diseases with no disease-modifying treatments. A multi-project program called CASPER aims to develop CRISPR gene-editing therapies that permanently correct the faulty genes.
- Fields
Neuroscience
Biomedical Engineering
Medicine & Clinical Research
- Topics
gene editing
gene therapy
Huntington's disease
- Grants
- 1 known grant, through Apr 2028
What they work on
- Deliver CRISPR gene-editing vectors into the mouse brain using convection-enhanced delivery surgery, finding the dosing and spread that work best for each CASPER project.
- Measure whether gene editing rescues disease-related behavior in mouse models of Huntington's disease and C9orf72 ALS, using standardized behavioral tests.
- Collect tissues from treated animals and supply them to the Genomics and Biological Outcomes Cores to evaluate editing efficiency and biological effects.
More about the research
Before such therapies can move toward human trials, researchers must show in animals that the editing tools reach the right brain areas, edit the gene, and improve symptoms.
Why it matters
For people with Huntington's disease and C9orf72-associated ALS, durable CRISPR gene-editing therapies could one day slow or modify disease. Consistent animal data on delivery, editing and behavioral benefit may shorten the path from preclinical studies to an investigational new drug application for human testing.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
X. Shawn Liu
Columbia University, NY·Neuroscience
Test whether reactivating the healthy MECP2 copy on the silenced X chromosome, by removing promoter methyl tags with dCas9-Tet1, rescues Rett syndrome symptoms in female mouse models.
Bruce Armitage
Carnegie Mellon University, PA·Chemistry
Design fluorescent peptide nucleic acid (PNA) probes that bind unmodified C9orf72 repeat RNAs, so these RNAs can be seen in biomolecular condensates by microscopy.
Jailson Brito Querido
University of Michigan, MI·Biochemistry
Determine how expanded GGGGCC repeats in C9orf72 mRNA physically engage the ribosome to trigger non-AUG start site selection, using cryo-electron microscopy with biochemical and genetic approaches.
The school
University of California-Berkeley: 46 labs on PI Pages, $1.1B a year on research, 825 research doctorates a year.
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