466. Long-Term Rapamycin Control of Transgene Expression in Mouse Salivary Glands in a Single AAV Vector
Molecular Therapy(2005)
摘要
Background: Salivary glands are a promising gene transfer platform for secretory therapeutic protein delivery (Baum et al., Trends Mol Med 2004; Zufferey and Aebischer, Gene Ther 2004). For ideal clinical application, therapeutic protein levels need to be controlled. We have shown, as proof-of-concept, that the rapamycin-inducible dimerizer system can regulate transgene expression from salivary glands after delivery with adenoviral vectors (Wang et al., Gene Ther 2004). To achieve long-term transgene expression from salivary glands, we have used serotype 2 adeno-associated viral (AAV2) vectors (Voutetakis et al., PNAS 2004).
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mt, INSERT KEY WORDS HERE, pharmacology
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