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BioWorld Science, Neurology/psychiatric
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Genetic/Congenital

Base editing rescues spinal muscular atrophy in vivo

April 6, 2023
By Mar de Miguel
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
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Elderly hands holding broken brain structure
Neurology/Psychiatric

STIM1 overexpression restores synaptic plasticity in female mice modeling Alzheimer’s disease

April 5, 2023
Gliotransmitter release by astrocytes depends on calcium dynamics and is an essential regulator of brain functions. Alterations of calcium homeostasis have been observed in neurons and astrocytes from mouse models of Alzheimer’s disease (AD). A new longitudinal study in the PS2APP mouse model of AD suggests a possible link between calcium dynamics in astrocytes and memory loss.
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Elderly woman holding illustration of brain with missing puzzle piece
Neurology/Psychiatric

ARKD-104, a novel PGRN-enhancing compound to prevent frontotemporal dementia

April 4, 2023
Frontotemporal dementia (FTG-GRN) is a fatal neurodegenerative disease caused by heterozygous mutations in the GRN gene, encoding progranulin (PRGN). Researchers at Arkuda Therapeutics Inc. reported on ARKD-104, a novel potent orally bioavailable PGRN-enhancing compound that increased PRGN secretion and lysosomal granulins in multiple relevant cell lines.
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