Question

why the progross of gene therapy has been dissapointly slow , give actual examples of failure gene therapy trails

Answer 1

The gene therapy is the replacement of the cells by the introduction of new cells in place of the defective ones. This is done to correct genetic disorders. Major advantages have been observed because of this therapy. However, the precise and sensitiveness of the method has been a reason for its slow progress.

The gene replacement is a highly specific and sensitive therapy. The insertion of the specific gene is difficult and requires expertise. One of the main reasons of the slow progress is the availability of limited vectors for the gene therapy. Intravenous injections are neutralized within a few millimeter of entering. Manufacture of synthetic vectors to overcome this problem have been carried out. This has also been one of the failure in the gene therapy.

Some other factors that cause the failure of gene therapy are adaptive and innate immune responses, genotoxicity, unwanted persistence of the genetically modified cells, and others.

Investigators and scientists are still trying to reverse the heart failure and experiments have been failing since. For example, in New York, a team led by Roger Hajjar, tested the therapy with an intention to reverse heart failure. 13 pigs suffered from severe heart failure and were given gene therapy. However, the therapy was capable of controlling 25% of the heart failure in the left ventricle and 20% in the left atrium.