Statistical Topics in Gene Therapy
The Effective Statistician - in association with PSI
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Exploring Gene Therapy Advances
This chapter examines the evolution of gene therapy from its origins to recent breakthroughs like Kimriah and Zolgensma. It discusses the mechanisms of gene therapy, including gene transfer and editing technologies like CRISPR, while also addressing the ethical and regulatory challenges of developing treatments for both rare and common diseases. The conversation emphasizes the potential of gene therapies as permanent solutions and the complexities involved in proving their long-term efficacy.
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