Gene transfer to the intestine

Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai

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424 931, 4351723, A61K 4800

Patent

active

057863407

ABSTRACT:
Method for in vivo introduction of a nucleic acid cassette into stem cells of intestinal epithelium. The nucleic acid cassette is introduced via vector solution. The vector solution can be delivered via the intestinal lumen in a variety of ways, including through an insertion device such as an endoscope, through catheters, through ligating and clamping the intestine after laparotomy or through slow release capsules. The vector solution once introduced into the intestinal epithelium is allowed to contact the stem cells for sufficient time for incorporation, usually between 1 and 48 hours. After sufficient incorporation, the insertion device and/or clamping and ligation procedure blockage are removed. Preferably, the procedure includes sufficient fluid to distend the intestine and provide additional access to the stem cells and the crypts. The procedure is useful in treating a variety of diseases including metabolic disorders, endocrine disorders, circulatory disorders, coagulation disorders, cancer, and gastrointestinal disease.

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Orkin, S. H et al. Report and Recommendations of the Panel to Assess the N.I.H. Investment in Research on Gene Therapy (see pp. 8-11).

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