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Harvard Team Report One-Time Base Editing Treatment for Motor Neuron Disease in Mice

Using base editing, a team led by Harvard University scientists has treated spinal muscular atrophy (SMA) in mice, opening the door to a potential one-time treatment for the progressive motor neuron disease in humans. SMA is caused by homozygous loss or mutation of the essential survival motor neuron 1 (SMN1) gene, which encodes a protein called SMN. Approved therapies for the condition can increase SMN levels, but don't fix the underlying problem, require repeat dosing, and many wane in effectiveness. In a study appearing in Science this week, the researchers focused on SMN2, a gene closely related to SMN1 that harbors a mutation preventing it from regulating the SMN protein. They developed base editors to modify SMN2 into an active form and administered them to mouse models of SMA, bringing the animals' SMN levels to normal while improving their motor function and extending their lifespans with a single treatment. Importantly, the intervention did not alter endogenous regulatory mechanisms. "As such, a future base editing therapeutic approach could offer substantial benefits over existing SMA therapies," the study's authors write.

The Scan

ChatGPT Does As Well As Humans Answering Genetics Questions, Study Finds

Researchers in the European Journal of Human Genetics had ChatGPT answer genetics-related questions, finding it was about 68 percent accurate, but sometimes gave different answers to the same question.

Sequencing Analysis Examines Gene Regulatory Networks of Honeybee Soldier, Forager Brains

Researchers in Nature Ecology & Evolution find gene regulatory network differences between soldiers and foragers, suggesting bees can take on either role.

Analysis of Ashkenazi Jewish Cohort Uncovers New Genetic Loci Linked to Alzheimer's Disease

The study in Alzheimer's & Dementia highlighted known genes, but also novel ones with biological ties to Alzheimer's disease.

Tara Pacific Expedition Project Team Finds High Diversity Within Coral Reef Microbiome

In papers appearing in Nature Communications and elsewhere, the team reports on findings from the two-year excursion examining coral reefs.