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    • 70. 发明申请
    • Therapeutic treatment of digestive organs with engineered stem cells
    • 用工程干细胞治疗消化器官
    • US20040071674A1
    • 2004-04-15
    • US10392377
    • 2003-03-19
    • Hiroshi MashimoSatish K. Singh
    • A61K048/00C12N005/06C12N005/08
    • A61K38/44C12Y114/13039
    • A method of directly implanting embryonic or adult somatic stem cells into a desired site in a mammal, e.g., into the adult digestive tract, for gene therapy and/or cell replacement is disclosed. Stem cells with their inherent pluripotency have been found to be capable of being implanted directly, e.g., in the digestive system where they become permanently engrafted. The advantage of cultured stem cells is that they can be expanded in vitro and engineered to produce cells that stably express a variety of gene products. Stem cells directly implanted in the gastrointestinal tract according to the method of the invention, have been shown to differentiate into specialized cells of the adult gut (such as nerves, muscles and epithelia). These cells remain at the site of injection and respect local tissue architecture while permanently expressing transfected genes, e.g., nitric oxide synthase, which catalyzes the production of nitric gas in vivo. This approach is particularly well suited for such a diffusible, unstable and transiently bioactive compound as nitric oxide. This technology is useful, e.g., in treating gastrointestinal disorders where key components of a normally functioning bowel are missing, and where local administration of the gene product is thought to be essential.
    • 公开了将胚胎或成体体细胞干细胞直接植入哺乳动物(例如成人消化道)中用于基因治疗和/或细胞置换的期望部位的方法。 已经发现具有其固有多能性的干细胞能够被直接植入,例如在其被永久植入的消化系统中。 培养的干细胞的优点是它们可以在体外扩增并被工程化以产生稳定表达多种基因产物的细胞。 已经显示根据本发明的方法直接植入胃肠道的干细胞分化成成年肠道(如神经,肌肉和上皮细胞)的特殊细胞。 这些细胞保留在注射部位并且尊重局部组织结构,同时永久表达转染的基因,例如催化体内硝酸生成的一氧化氮合酶。 这种方法特别适用于像一氧化氮这样的可扩散,不稳定和瞬时生物活性化合物。 该技术可用于例如在正常功能性肠道的关键成分缺失以及基因产物的局部给药被认为是必需的胃肠疾病中。