Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges

Abstract excerpt

INTRODUCTION: Therapeutic gene editing is becoming a viable biomedical tool with the emergence of the CRISPR/Cas9 system. CRISPR-based technologies have promise as a therapeutic platform for many human genetic diseases previously considered untreatable, providing a flexible approach to high-fidelity gene editing. For many diseases, such as sickle-cell disease and beta thalassemia, curative therapy may already be...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Biotechnology and Related Fields
• Share to CRISPR and Genetic Engineering
• Share to Virus-based gene therapy research