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Exploring Gene Therapy Approaches and Challenges
The chapter delves into in vivo and ex vivo gene therapy approaches, highlighting a case where a girl regained her hearing through targeted otofurlin gene therapy. Discussions include the use of viral vectors to treat hearing loss, concerns about immune responses, preclinical testing, and the challenges of funding expensive gene therapies like those for sickle cell disease and severe combined immune deficiency. The chapter also considers the affordability and long-term benefits of gene therapies, expressing optimism about their potential revolution in healthcare with the advancement of safer techniques.