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美国Somatix治疗公司已获得许可拥有耶鲁大学开发的、用腺病毒伴随病毒(AAV)载体在分裂和未分裂细胞中存放DNA的技术.公司计划用AAV载体将基因传递给消化道细胞,这些细胞在体内产生治疗性蛋白,直接把它释放到循环系统中.AAV不致病,但能感染消化道衬细胞,包括直接位于循环床上的细胞.此项技术可用于生产凝血因子Ⅷ和Ⅸ等蛋白,将上述蛋白基因插入AAV载体,被血友病患者摄入后,释放到胃肠道粘膜中.肠道毛细血管侧细胞就产生凝血因子,虽然有些凝血因子可能被消化酶破坏,但是应有足量凝血因子直接进入循环系统.由AAV载体传递的基因在靶细胞中的表达能持续4～12个月,意味着血友病患者每年仅需重复治疗数次即可保持增高的因子Ⅷ和Ⅸ水平.推测口服AAV可将肿瘤相关抗原(TAA)基因序列传递给肠道细胞,一旦这些细胞把特定的TAA分泌到血流中,这种抗原就可在病人体内引起对携有TAA的肿瘤的免疫应答.
US Somatix Therapeutics has licensed a technology developed by Yale University that uses adenovirus-associated virus (AAV) vectors to store DNA in both dividing and non-dividing cells The company plans to deliver the gene to the digestive tract cells using the AAV vector, Therapeutic protein is produced in the body and released directly into the circulatory system.AAV is not pathogenic but can infect lining cells of the digestive tract, including cells located directly on the circulating bed.This technique can be used to produce proteins such as Factor VIII and IX, The above-mentioned protein gene is inserted into the AAV vector and released into the mucosa of the gastrointestinal tract after ingestion by a patient with hemophilia. The intestinal capillary side cells produce coagulation factors. Although some coagulation factors may be destroyed by digestive enzymes, The amount of clotting factor directly into the circulatory system.The expression of the gene delivered by the AAV vector in target cells lasts 4 to 12 months, meaning that patients with hemophilia need only repeated treatment several times a year to maintain the elevated factor VIII and IX Levels. It is speculated that oral AAV can deliver the tumor associated antigen (TAA) gene sequence to gut cells, and once these cells have secreted the specific TAA into the bloodstream, this antigen can Causing the patient to carry a TAA tumor immune response.