Self-complementary AAV bypasses cellular second-strand synthesis to increase β-sarcoglycan expression in LGMD2E.
A compact enhancer–core promoter hybrid enables selective expression of larger genes in podocytes within AAV size constraints.
The TDP-43 RNA-binding domain is fused to RAVER1 to restore splicing repression while limiting cytoplasmic aggregation in motor neuron models.
T7 RNA polymerase is fused with NP868R to add 5′ caps during transcription, enabling functional capped RNA production in eukaryotic cells.
Inducible viral-gene expression and helper functions address low rAAV output, increasing production by 1.5–2-fold.
By isolating essential promoter elements, 123–252 bp nucleic acids retain activity while shortening expression cassettes for hepatocytic cells.
Conventional promoters can limit cultured-cell productivity; Chinese hamster Eno1 promoters raise foreign protein expression while retaining mammalian cell quality.
Modified HBB2 introns and codon-optimized UGT1A1 in AAV vectors target liver cells to lower bilirubin and reduce immune response.
Modified bagworm Fib H lets transgenic silkworms produce hybrid silk at scale, avoiding contaminated natural nests and costly cleanup.
Compact enhancer elements linked to minimal promoters preserve liver-specific expression while leaving vector space for large effector genes.
DNA shuffling and mouse-liver screening select synthetic AAV capsids for liver gene transfer while limiting off-target transduction.