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A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice | Gene Therapy
Abstract Genetic switches that allow for precise control over transgene expression timing or levels may improve the safety and expand the use of adeno-associated viral (AAV) vector-based gene therapy technologies. We previously engineered an efficient RNA switch system that comprises a novel self-cleaving ribozyme (T3H38) and an octaguanidine dendrimer-conjugated morpholino oligonucleotide (v-M8) complementary to the ribozyme.
Investigation of the Machined Surface Integrity of WC-High-Entropy Alloy Cemented Carbide
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Metabolite annotation from knowns to unknowns through knowledge-guided multi-layer metabolic network
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