Figure 1.

Gene delivery technologies in lipid research and therapies. (A) AAV vectors are commonly used for overexpressing proteins. (B) ASOs and siRNAs are synthetic nucleic acids engineered to hybridize with a target mRNA or pre-mRNA to induce its degradation and silencing. (C) AAV vectors are also used for delivering the CRISPR/Cas9 genome editing system. A gRNA guides the Cas9 nuclease to a complementary genomic site in proximity to a Protospacer Adjacent Motif (PAM). Cas9 induces a DSB causing indel mutations, which can be used to permanently inactivate or knock out the target gene. AAV: adeno-associated virus; ASO: antisense oligonucleotides; siRNAs: small interfering RNAs; CRISPR/Cas9: Clustered Regularly Interspaced Short Palindromic Repeats; gRNA: guide RNA