Gene Therapy is playing a major role in rare disease management. Gene therapy in rare diseases is a specialised and targeted treatment that works by inserting corrected genes into an individual's cells. Genetic aetiology accounts for approximately 80% of rare diseases. Gene Therapy for Rare Disease Market is thought to be proven, with the potential to transform the treatment of rare diseases. Gene therapy, with the advantage of single-dose treatment for rare diseases, confers effective lifelong improvement when compared to conventional treatment options. Gene therapy development for rare diseases includes a wide range of manufacturing technologies, including plug-and-play manufacturing processes for AAV (adeno-associated virus) serotypes, a compendium of standard analytical and bioanalytical methods, cell suspension, and cell potentiation technology.
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