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在构建安全有效的方式以输入基因至病人的细胞时,有人试图用“解除武装的腺病毒”(disarmed adenovirus,AV)将活性基因输至人体细胞中。至目前为止,一般使用含有RNA的逆转录病毒作人类基因治疗的临床试验,但此类病毒的生物学特性涉及安全问题,因其具有引起突变或癌症的潜在可能性。此外,逆转录病毒的基因容积也限制了载入及输入基因物质的含量。另一基因输入系统,即分子偶合物,虽能克服上述困难,但其所输入人体细胞或实验动物的肝细胞中的异体DNA在细胞内囊泡中仍为双股,因而降低了此法的效率。
When building a safe and effective way to enter genes into the cells of a patient, someone tried to transport the gene into human cells using a “disarmed adenovirus” (AV). To date, clinical trials of human gene therapy with RNA-containing retroviruses have generally been used, but the biological properties of such viruses involve safety concerns as they have the potential to cause mutations or cancer. In addition, the gene capacity of retroviruses also limits the amount of genetic material loaded and imported. Although the other gene input system, molecular conjugate, can overcome the above difficulties, allogeneic DNA in the hepatocytes of human cells or experimental animals that are input into the system remains double-stranded in the intracellular vesicles, effectiveness.