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Gene therapy in autoimmune diseases
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Introduction |
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Collectively, autoimmune diseases constitute a major, unmet, clinical challenge. Although no single autoimmune disorder is highly prevalent, there are over 80 of them, and 20% of the population is affected; approximately 75% of patients are women. Because these diseases are generally incurable and difficult to manage, there is a pressing need for novel approaches to their treatment. For reasons detailed below, we have proposed that gene therapy merits investigation in this regard.1
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Why genes? |
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Traditional pharmacological approaches to treatment entail the
synthesis of small, diffusable compounds given orally or by injection.
These approaches have yet to provide ideal agents for use in autoimmune
diseases. Recent research, however, has identified a number of proteins
with the potential to improve treatment, but these are difficult to
administer long term. Gene transfer provides the opportunity to deliver
protein products, as well as therapeutic species of nucleic acids, such
as antisense RNA, much more efficiently than traditional
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