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Sickle Cell Disease Mutation Repaired in Patient Cells With Prime Editing

In Nature Biomedical Engineering, investigators with the Broad Institute's Merkin Institute of Transformative Technologies in Healthcare, Harvard University, and other centers present findings from an ex vivo prime editing study focused on repairing a sickle cell disease (SCD)-related mutation in the beta-globulin-coding gene HBB. After achieving prime editing frequencies of between 15 percent and 41 percent in hematopoietic stem cells or progenitor cells from four SCD-affected individuals, the researchers transplanted cells into immunodeficient mice, tracking edited cell engraftment, wild-type HBB levels, potential off-target edits, and response to low oxygen conditions after 17 weeks. "Taken together, our results establish an early example of therapeutic prime editing in human HSCs and demonstrate a potential strategy for a one-time autologous SCD treatment that directly corrects the sickle globin allele back to wild-type HBB without requiring [double-strand breaks] or donor DNA templates," the authors suggest.

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.