Targeted VP1 amino acid substitutions in AAV capsids improve intracellular trafficking and ocular cell transduction while preserving a human AAV backbone.
NR5A1 and RUNX overexpression drives pluripotent stem cells into granulosa-like cells in 5-7 days with robust markers and hormonal function.
Small alphaherpesvirus LAP promoters help AAV vectors avoid transcriptional repression and sustain chronic transgene expression.
Peptide insertions in AAV capsid variable regions IV or VIII improve eye-cell targeting and transduction for ocular gene delivery.
Self-splicing introns and homology arms circularize RNA only in target cells, improving expression stability while avoiding genomic integration.
A U-rich 3' UTR placed after the stop codon boosts mRNA stability and translation, enabling lower therapeutic doses and cost.
Segmented poly(A) blocks with non-A spacers stabilize mRNA tail length and improve translation consistency for RNA therapeutics.
Localized nucleotide modification in pre-crRNA boosts Cas3 editing efficiency and supports allele-specific editing with clearer pattern analysis.
Bacterial sterol-degrading enzymes let modified macrophages break down cholesterol, addressing foam-cell buildup linked to atherosclerosis.
Inactive recombinase halves dimerize only after induction, reducing cell toxicity while enabling precise gene expression control.
TOP-derived 5′UTR elements and histone stem-loops help RNA resist RNase degradation while boosting translation and protein expression.
Conditionally activated MM transgenes in immunocompetent mice capture disease progression and tumor-immune interactions for drug screening.
An asymmetric AAV ITR design boosts DNA packaging and transgene expression while reducing host genome integration and genotoxicity.
Specific plant promoter sequences and GATCTG motif editing improve constitutive gene expression stability and reduce variation in transgenic plants.
Podocyte-specific AAV vectors use NPHS1 or NPHS2 promoters to restore functional gene expression and reverse monogenic nephrotic syndrome.
Reduced-CpG AAV vectors use methylation, CAG promoter control, and WPRE to sustain payload expression while limiting immune activation.
MicroRNA target sites in therapeutic polynucleotides enable cell-selective expression, reducing off-target effects in mRNA and gene editing.
Engineered AAV capsids use DNA shuffling and in vivo screening to boost liver-specific transduction while limiting off-target gene transfer.
NR5A1 and RUNX overexpression drives fast, serum-free iPSC differentiation into granulosa-like cells with proper markers and hormonal function.
End-adapted linear DNA with an appended NLS improves nuclear entry in non-dividing cells while resisting exonuclease digestion.