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Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing
CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold tremendous potential to effectively treat disease-causing mutations with diverse cellular origin. However, it is currently impos...
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Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs
Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of medicines that directly reprogramme the central dogma of biology to prevent and treat disease. However, multiple biological barr...
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Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells
Therapeutic genome editing of haematopoietic stem cells (HSCs) would provide long-lasting treatments for multiple diseases. However, the in vivo delivery of genetic medicines to HSCs remains challenging, espec...