In this section you find information about the in's and out's of gene therapy.
The Promise of Gene Therapy
For some years now, gene therapy has been held up as having great promise for the treatment of genetic conditions. In particular, it has been seen as a route for the treatment of rare, single gene disorders.
Despite initial optimism, and some promising early results, gene therapy has not delivered a range of clinically available interventions that will treat, prevent or cure a growing number of these rare conditions.
Gene Therapy Keenest Advocate: the Patient
Despite this apparent lack of progress, patients and families continue to be amongst the keenest advocates for research and development in gene therapy. Why is this?
Research continuously produces new insights in the development and treatment options for inherited diseases. A growing number of clinical trials is under way. Gene therapy has the potential to make possible a gene medicine that has a generic application for monogenetic, rare disorders.
That potential is the drive for patients to advocate and raise funds for more research, as rare disorders often are the orphans in research programmes.
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