Innovations in Immune Deficiency Treatments

This chapter explores the complexities and advancements in treating severe immune deficiencies, with a focus on bubble boy disease and the limitations of existing therapies. It highlights the evolution of gene therapy, the role of CRISPR, and the entrepreneurial journey of developing CAR-T cell therapies, emphasizing collaboration and the need for scalable solutions. The discussion underscores the challenges of translating personalized treatments into effective, accessible therapies for pediatric malignancies.