Chapter 19 – Utility of Adenoviral Vectors in Animal Models of Human Disease II: Genetic Disease
作者： Raymond John Pickles
DOI: 10.1016/B978-012199504-1/50020-1
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摘要: Publisher Summary This chapter discusses the utility of adenoviral vectors in animal models genetic diseases. It is mainly focused on therapeutic uses cystic fibrosis (CF) lung disease. CF disease has been considered as a prototypic state for "proof-of-concept" gene-therapy strategies. The cloning gene responsible pathophysiology, two groundbreaking observations made therapy appear imminent. First, isolated epithelial cells cultured from airway epithelium patients could be phenotypically "corrected" by transferring into cDNA corresponding to gene. Second, (Ad) engineered express were administered airways experimental animals and transgene expression observed that require "correction." These findings will not only beneficial treatment but also other states, which are continually being brought closer perhaps cure this new exciting biomedical technology.
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