Jul 25, 12:00 PM

Prime Editing Corrects Alternating Hemiplegia Mutations With 48% Genomic Correction in Mice, Published in Cell

Researchers from the Broad Institute, Jackson Laboratory, and the rare disease group RAREHope have demonstrated the first successful use of prime genome editing, also known as CRISPR 3.0, to correct mutations causing alternating hemiplegia of childhood (AHC), a rare neurodevelopmental disorder. This breakthrough was achieved through in vivo brain editing in mouse models, resulting in up to 48% genomic correction and 73% mRNA correction in the cortex, which reduced symptoms and extended lifespan. The findings were published on July 21, 2025, in the journal Cell. Separately, Japanese scientists have used CRISPR-Cas9 technology to remove the extra chromosome responsible for Down syndrome in affected cells in laboratory settings. Additional research has highlighted the neuroinvasive nature of SARS-CoV-2, with studies showing viral persistence in the brainstem of hamsters 80 days post-infection, contributing to neurodegenerative processes linked to long COVID. In another development, scientists have engineered mosquitoes by editing the FREP1 gene to carry a protective allele that blocks multiple malaria parasite species. This gene drive is expected to spread naturally through mosquito populations, potentially transforming them into vectors incapable of transmitting malaria, offering a promising strategy for malaria control.

#Broad Institute #Jackson Laboratory #Cell #Japanese #Down syndrome

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