Researchers at leading biotech firms apply CRISPR-Cas9, base editing, and prime editing to modify genes tied to cellular aging, such as SIRT1 and FOXO3. They leverage both ex vivo stem cell approaches and lipid nanoparticle delivery in vivo to develop potential one-time therapies against cardiovascular and neurodegenerative conditions.
Key points
- Ex vivo CRISPR-Cas9 editing of HSCs targets longevity genes (SIRT1, FOXO3) with HDR precision.
- Base and prime editing platforms reduce off-target effects compared to standard Cas9, enhancing specificity.
- Lipid nanoparticle delivery achieves >80% in vivo gene disruption of PCSK9 and ANGPTL3 in mouse liver.
Why it matters: This breakthrough signifies a paradigm shift in anti-aging therapeutics, enabling precise genetic interventions to enhance healthspan over traditional drug approaches.
Q&A
- What is prime editing?
- How are CRISPR therapies delivered in vivo?
- What distinguishes healthspan from lifespan?