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Gene therapy targeting connexin 43 shows promise across inherited heart diseases
University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...
New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.
Getting therapeutic drugs past the blood-brain barrier has long been a major challenge in treating brain diseases. Now, researchers have explored how cholesterol-modified heteroduplex oligonucleotides ...
Gene editing in plants remains challenging, with the traditional non-homologous end-joining (cNHEJ) repair pathway often hindering precision. In this study, researchers advanced CRISPR-Cas-based gene ...
This study revealed that, by binding to lipoproteins, cholesterol-conjugated heteroduplex oligonucleotides (Chol-HDOs) can remain in the bloodstream for longer and reach brain tissue much more ...
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