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New Approach to Deliver Hereditary Blindness Therapeutics

A technique for delivering messenger RNA into neural cells of the eye is described in Science Advances this week, marking a key step toward treating blindness associated with inherited retinal degenerations (IRDs). IRDs are a complex group of genetic disorders that arise from mutations in the more than 300 different genes necessary for photoreceptor and retinal pigment epithelium functioning. While a number of drugs for IRDs are under development and one has been approved in the US, they rely on adeno-associated viruses (AAVs) for the delivery of their therapeutic payloads, which have a number of limitations including immunogenicity and limited packaging capacity. In this week's study, a team led by scientists from Oregon State University looked to lipid nanoparticles (LNPs) as delivery vehicles, developing ones that can carry mRNA and use peptides to localize the drug to the neural retina, which had previously been inaccessible to LNPs. They demonstrate their approach in rodents and non-human primates, suggesting LNP-mRNA delivery could have clinical applications for IRDs

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.