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A baby named KJ, born with a rare genetic disorder called CPS1 deficiency, is thriving thanks to the world’s first personalized gene-editing treatment. 🧬✨ Doctors at @childrensphila.bsky.social used base editing—a tool invented by HHMI Investigator David Liu and his lab at Harvard.

💥🥳 At long last, our latest paper is out! Gag proteins of endogenous retroviruses are required for zebrafish development www.pnas.org/doi/10.1073/... Led heroically by Sylvia Chang & @jonowells.bsky.social A study which has changed the way I think of #transposons! No less! 🧵 1/n

TIM-3's role in microglia and Alzheimer's is unveiled! Found to influence neurodevelopment and neuroinflammation, affecting late-onset Alzheimer's risk. PMID:40205047, Nature 2025, @Nature https://doi.org/10.1038/s41586-025-08852-z #Medsky #Pharmsky #RNA #ASHG #ESHG 🧪

A new study raises the odds that a strategy already successful against some cancers may be deployed against Alzheimer’s. From @kuchroolab.bsky.social and colleagues, co-first authors Kimitoshi Kimura and @ayshwaryas.bsky.social news.harvard.edu/gazette/stor...

Microglia are the resident immune cells in the brain and have pivotal roles in neurodevelopment and neuroinflammation1,2. This study investigates the function of the immune-checkpoint molecule TIM-3 (encoded by HAVCR2) in microglia. TIM-3 was recently identified as a genetic risk factor for late-onset Alzheimer’s disease3, and it can induce T cell exhaustion4. However, its specific function in brain microglia remains unclear. We demonstrate in mouse models that TGFβ signalling induces TIM-3 expression in microglia. In turn, TIM-3 interacts with SMAD2 and TGFBR2 through its carboxy-terminal tail, which enhances TGFβ signalling by promoting TGFBR-mediated SMAD2 phosphorylation, and this process maintains microglial homeostasis. Genetic deletion of Havcr2 in microglia leads to increased phagocytic activity and a gene-expression profile consistent with the neurodegenerative microglial phenotype (MGnD), also referred to as disease-associated microglia (DAM). Furthermore, microglia-targ

Outstanding Barbara McClintock Life Sciences Lecture by @michellemonje.bsky.social @cornell. And honored to have her as the first official visitor to our newly renovated space, and adding her favorite OPCs to our cell type leaderboard 🧫 🧠

Breaking News: A baby with a rare disorder made medical history by receiving the first custom gene-editing treatment. The technique used has the potential to help people with thousands of other uncommon genetic diseases. nyti.ms/4j49xBy