CRISPR-Cas9 & Viral Vectors in Pompe Disease Therapy

This coursework provides a comprehensive study of Pompe Disease, starting with an introduction to its genetic basis and clinical challenges. It explores current treatments such as enzyme replacement therapy and examines new advancements like CRISPR-Cas9 and viral vector technologies for gene therapy. Ethical and practical considerations are addressed, along with safety and efficiency concerns. The material concludes with insights into future research and emphasizes the value of...