8min chapter

What's Your Problem? cover image

Using Gene Therapy to Help the Blind See

What's Your Problem?

CHAPTER

Advancements in Gene Therapy

This chapter explores the evolution of gene therapy, particularly in addressing the rare genetic condition LCA1, which causes severe visual impairment. It discusses innovative research methods using chickens as a model to test gene therapy techniques and the challenges of embryo survival. The chapter also highlights the crucial role of viral vectors, particularly adeno-associated virus (AAV), in the development of effective gene therapy solutions.

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