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Dec 23, 2014
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Gene Therapy: The “Promise” to cure Gastrointestinal disorders!
Gastrointestinal disorders refer to diseases involving the gastrointestinal tract, namely the esophagus, stomach, small intestine, large intestine and rectum, and the accessory organs of digestions, the liver, gallbladder, and pancreas. Gastrointestinal diseases which might benefit from medical care are very common, affecting around millions of Americans each year. The rapid progress in our understanding of some of the molecular mechanisms involved in the pathogenesis of cancer and metabolic disorders, coupled with the development of gene delivery vector technology, have urged us to consider novel genetic approaches to gastrointestinal diseases.
Gene therapy consists of the introduction of genetic material into cells for a therapeutic purpose. A wide range of gene therapy vectors have been developed and used for applications in the field of gastrointestinal disorders. In this field gene therapy has produced considerable expectation as a potential tool in the management of conditions that lack effective therapy including non-resectable neoplasms of the liver, pancreas and gastrointestinal tract, chronic viral hepatitis unresponsive to interferon therapy, liver cirrhosis, and inflammatory bowel disease. Moreover, the intestinal tract has many features that make it an attractive target for therapeutic gene transfer like: (a) easy accessibility via the intestinal lumen; (b) large surface area of the epithelium; (c) the possibility of in situ gene transfer by endoscopy; (d) known location of stem cells within the intestinal crypt, (e) intestinal cells can secrete foreign protein into the circulation which could act as a promising strategy for the treatment of systemic diseases, such as haemophilia.
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Low disease awareness, poor diagnosis rates, combined with a lack of pharmaceutical industry involvement, has kept gastrointestinal diseases away from the limelight. But this is changing as evidenced by companies such as Digna Biotech, Sylentis S.A. and many more which are operating in the field of gastrointestinal disorders in the gene therapy domain. Many companies have started investing in and researching on gene therapy as it has the potential to become a lucrative market. Moreover, animal models of human diseases and pilot clinical studies clearly show that there is a future for genes to be used as curative drugs.
DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for gastrointestinal disorders including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners in this therapeutic area. It also identifies the potential market movers and future regulatory landscape.
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