Publications by authors named "Kenia Guzman"
Prion disease is a fatal neurodegenerative disease caused by the misfolding of prion protein (PrP) encoded by the PRNP gene. While there is currently no cure for the disease, depleting PrP in the brain is an established strategy to prevent or stall templated misfolding of PrP. Here we developed in vivo cytosine and adenine base strategies delivered by adeno-associated viruses to permanently modify the PRNP locus to achieve PrP knockdown in the mouse brain.
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- Prion diseases happen when a special protein in the brain gets twisted wrong, which can cause serious memory problems and even death.
- Researchers created something called CHARM, a tool that can change how our DNA works to stop these bad proteins from causing harm.
- In tests with mice, CHARM successfully turned off the harmful protein in their brains and could help treat other brain diseases too!
Neurofilament light (NfL) concentration in cerebrospinal fluid (CSF) and blood serves as an important biomarker in neurology drug development. Changes in NfL are generally assumed to reflect changes in neuronal damage, while little is known about the clearance of NfL from biofluids. We observed an NfL increase of 3.
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