A research article about a targeted lipid nanoparticle strategy for delivering genome‑editing cargos to human blood stem cells in vivo, highlighting experimental approaches and potential translational value.
Key Takeaways
- Enabling the sustained long-term reactivation of fetal haemoglobin (HbF)
- In a humanized neutropaenia model harbouring an ELANE mutation
- 5: In vivo genome editing via CD34/LNP DP delivery in humanized mice