Ledley F D
Gene Medicine Inc, Houston.
Curr Opin Biotechnol. 1994 Dec;5(6):626-36. doi: 10.1016/0958-1669(94)90085-x.
Non-viral gene therapies are currently under development that employ drug-delivery methods for targeting genes to selected cells in the body, where they express therapeutic gene products. Various methods have been described for non-viral gene therapy, ranging from the direct intramuscular injection of purified DNA to the systemic administration of formulations comprising DNA and lipids, proteins, peptides, or polymers. Products for non-viral gene therapies are designed both for direct administration to patients by conventional routes and for expression of a therapeutic product over a finite period of time in a manner similar to conventional medicines. Initial preclinical and clinical studies indicate that non-viral gene delivery methods exhibit safety profiles similar to conventional pharmaceutical or biological products. Clinical trials have been proposed, or are currently under way, to assess the applicability of non-viral gene therapy for a variety of disorders, including cystic fibrosis, cancer, and peripheral vascular disease. Non-viral techniques may soon allow gene therapy to be applied in clinical practice alongside conventional medicines for the treatment of common diseases.
目前正在研发非病毒基因疗法，该疗法采用药物递送方法将基因靶向输送到体内特定细胞，使其表达治疗性基因产物。已描述了多种非病毒基因治疗方法，从直接肌肉注射纯化的DNA到全身给药包含DNA和脂质、蛋白质、肽或聚合物的制剂。非病毒基因治疗产品的设计既可以通过传统途径直接给予患者，也可以在有限时间内以类似于传统药物的方式表达治疗性产品。最初的临床前和临床研究表明，非病毒基因递送方法的安全性与传统药物或生物制品相似。已提出或正在进行临床试验，以评估非病毒基因疗法对包括囊性纤维化、癌症和外周血管疾病在内的多种疾病的适用性。非病毒技术可能很快使基因疗法能够与传统药物一起应用于临床实践，用于治疗常见疾病。
