Câu trả lời ngắn gọn
A lipid nanoparticle is a microscopic bubble of fat with the genetic cargo tucked inside. Injected into the blood, it naturally ends up in the liver, where cells absorb it and release the contents. It is the same delivery technology used in mRNA COVID-19 vaccines, and it is what made editing a gene inside a living person practical.
Lipid nanoparticles comprise an ionisable lipid, a helper phospholipid, cholesterol and a PEG-lipid. After intravenous administration they acquire an ApoE corona and are taken up by hepatocytes via the LDL receptor, then escape the endosome as the ionisable lipid protonates. Cargo — typically Cas9 mRNA plus guide RNA — is expressed transiently, which limits editor exposure and off-target opportunity. Redosing is possible, unlike with AAV.
Advantages that matter clinically
Sources
- National Human Genome Research Institute
Talking Glossary of Genomic Terms ↗