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Rare Diseases: Passion, Mission, and Startups
The chapter explores the significance of rare diseases in gene therapy and the passion and mission-driven focus required to tackle them. It discusses the financial incentives and timeline for startups in the field, highlighting the importance of long-term goals rather than immediate profitability. The chapter also delves into specific genetic diseases, their variations, and the potential for gene therapy to address childhood diseases and prevent chronic lifestyle diseases.