During M J, Xu R, Young D, Kaplitt M G, Sherwin R S, Leone P
Department of Molecular Medicine, University of Auckland School of Medicine, New Zealand.
Nat Med. 1998 Oct;4(10):1131-5. doi: 10.1038/2625.
Gene therapy is usually reserved for severe and medically refractory disorders because of the toxicity, potential long-term risks and invasiveness of most gene transfer protocols. Here we show that an orally administered adeno-associated viral vector leads to persistent expression of a beta-galactosidase transgene in both gut epithelial and lamina propria cells, and that this approach results in long-term phenotypic recovery in an animal model of lactose intolerance. A gene 'pill' associated with highly efficient and stable gene expression might be a practical and cost-effective strategy for even relatively mild disorders, such as lactase deficiency.
由于大多数基因转移方案具有毒性、潜在的长期风险和侵入性，基因治疗通常仅用于严重的、医学上难以治疗的疾病。在这里，我们表明口服给药的腺相关病毒载体可导致β-半乳糖苷酶转基因在肠道上皮细胞和固有层细胞中持续表达，并且这种方法可使乳糖不耐受动物模型实现长期表型恢复。对于甚至相对较轻的疾病，如乳糖酶缺乏症，与高效稳定基因表达相关的基因“药丸”可能是一种实用且具有成本效益的策略。
