Wu G Y
Division of Gastroenterology-Hepatology, University of Connecticut School of Medicine, Farmington 06030, USA.
Zhonghua Min Guo Xiao Er Ke Yi Xue Hui Za Zhi. 1998 Sep-Oct;39(5):297-300.
Gene therapy toward the digestive organs has made substantial progress. The strategies applied include ex vivo and in vivo delivery. The ex vivo pathway does not require a tissue-specific vector while the in vivo pathway is advantageous for tissues not obtainable or hard to culture. The specific vectors of gene delivery for the liver and gastrointestinal tract include (1) viral vectors: retrovirus, adenovirus, and adeno-associated virus are the three most common currently used. (2) liposomes: when cationic substances are incorporated, DNA can be coated on the surface and carried into cells. (3) receptor-mediated gene therapy: natural and physiological and tissue-specific ligands were incorporated and modified to carry foreign DNA into the cells. They are tissue-specific and relatively nontoxic. The innovations focus on the prolongation of gene expression and a more efficient gene delivery.
针对消化器官的基因治疗已取得显著进展。应用的策略包括体外和体内递送。体外途径不需要组织特异性载体，而体内途径对于无法获取或难以培养的组织具有优势。肝脏和胃肠道基因递送的特定载体包括：(1) 病毒载体：逆转录病毒、腺病毒和腺相关病毒是目前最常用的三种。(2) 脂质体：当掺入阳离子物质时，DNA 可包被在表面并带入细胞。(3) 受体介导的基因治疗：掺入并修饰天然、生理和组织特异性配体以将外源 DNA 带入细胞。它们具有组织特异性且相对无毒。创新集中在基因表达的延长和更有效的基因递送。
