【摘要】 腺相关病毒(AAV)是细小病毒家族一员,为无包膜的线形单链DNA病毒。由于AAV有长期潜伏于人体而不具有任何致病性等优点,人们对AAV作为基因治疗载体,基因打靶载体等方面的应用给予了很大的希望。AAV载体的安全性能已经用于帕金森病和囊性纤维病一期的治疗。目前通过不断的发展、改造病毒载体的基因结构,扩大其载体容量,提高病毒产率和转染效率,以使腺相关病毒载体更加符合实际需要。更多还原

【Abstract】 AAV is a member of parvovirus.Becuase of its long incubation and non-pathogenicity,people focus on AAV in gene therapy,gene targeting and so on.Perhaps the most important attribute of AAV vectors is their safety profile in phase I clinical trials ranging from CF to Parkinson’s disease,which is discussed in this paper.At present,AAV genetic strucure was reconstructed and its vec- tor capacity was enlarged,enhancing virus yield and transfection efficiency,to make the AAV to be consistent with practical requirement.更多还原