Safety of adeno-associated virus gene therapy vectors: a current evaluation.
Safety of adeno-associated virus gene therapy vectors: a current evaluation.
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DOI：
10.1517/14740338.1.1.79
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发表时间：
2002-05-01
影响因子：
3.1
通讯作者：
Sands, Mark S
中科院分区：
文献类型：
--
作者：
Monahan, Paul E;Jooss, Karin;Sands, Mark S
越来越多的用于疾病的分子治疗的策略依赖于腺相关病毒（AAV）作为治疗性基因递送载体。这种病毒DNA载体最吸引人的特征之一是AAV基因递送的感知安全性。最近在人体临床试验中的应用支持在临床前试验中建立的安全记录，有证据表明在没有细胞免疫反应或组织干扰的情况下进行基因转移。然而，野生型AAV及其衍生物的生物学的许多方面仍在探索中。虽然新型重组AAV治疗剂的治疗潜力似乎很有希望，但最近的见解表明，需要考虑其药代动力学，生物分布和毒性方面，以实现这些分子药物的最安全应用。
An increasing number of strategies for molecular treatment of disease rely on the adeno-associated virus (AAV) as a therapeutic gene delivery vector. One of the most attractive features of this viral DNA vector is the perceived safety of AAV gene delivery. Recent applications in human clinical trials support the safety record established in preclinical trials, with evidence of gene transfer in the absence of cellular immune responses or tissue disturbance. Nevertheless, many aspects of the biology of the wild type AAV and its derivatives are still being explored. While the therapeutic potential of novel recombinant AAV therapeutics appears promising, recent insights suggest aspects of their pharmacokinetics, biodistribution and toxicity that require consideration to achieve the safest application of these molecular medicines.