Briand P, Kahn A
Institut Cochin de Génétique Moléculaire, Paris, France.
Pathol Biol (Paris). 1993 Oct;41(8):663-71.
Review of the therapeutic use of DNA transfer to treat a certain number of hereditary diseases (such as adenosine deaminase deficiency) or acquired diseases. The strategies (ex vivo manipulation or direct in vivo transfer of the corrective gene), vectors (retrovirus, adenovirus, nonviral vectors), and diseases which can benefit from gene therapy are considered and discussed together with an evaluation of the risk of gene therapy.
对通过DNA转移治疗若干遗传性疾病(如腺苷脱氨酶缺乏症)或后天性疾病的治疗用途进行综述。文中考虑并讨论了相关策略(矫正基因的体外操作或直接体内转移)、载体(逆转录病毒、腺病毒、非病毒载体)以及可从基因治疗中获益的疾病,并对基因治疗的风险进行了评估。