One reason gene editing is not widely used for genetic diseases is the difficulty in targeting specific cell types and tissues. Effective treatment requires precision in editing affected cells while minimizing side effects elsewhere.
A US-based research team successfully targeted cystic fibrosis by focusing on the lung tissue and stem cell populations producing mature lung cells, ensuring stable effects. The technique used lipid bubbles to deliver mRNA inside cells, harnessing technology similar to COVID-19 mRNA vaccines.
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