Treatment for Rare Diseases

As 80% of rare diseases are caused by a single gene and gene therapy has the capacity to heal cardinal genetic flaws, it makes sense to employ it to treat rare genetic disorders. Gene therapy can restore a single gene defect by introducing a "right" gene. A single dose may also be necessary for successful gene therapy to provide improvements that last a lifetime. Another method for treating uncommon disorders that uses RNA-based drug delivery is antisense therapies. The alternative method for treating rare disorders is called cell therapy, in which the patient receives an injection of live cells that have therapeutic effects.

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