Unlocking the potential: advancements and applications of gene therapy in severe disorders

Abstract excerpt

INTRODUCTION: Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health. BACKGROUND: Over the past few decades, researchers have been working on gene therapies, and currently, terrible dreams have come true. To date, the Food and Drug...

Topics

Join the communities discussing this publication.

• CRISPR and Genetic Engineering
• Neurogenetic and Muscular Disorders Research
• Virus-based gene therapy research