Breaking barriers in brain gene therapy Researchers are developing new delivery technologies to bring gene therapies to the central nervous system safely and precisely. Written byAlejandra Manjarrez, PhD

Breaking barriers in brain gene therapy Researchers are developing new delivery technologies to bring gene therapies to the central nervous system safely and precisely. Written byAlejandra Manjarrez, PhD https://www.drugdiscoverynews.com/breaking-barriers-in-brain-gene-therapy-17537?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8NXoSueeDn6O6K_9XiFDwPNnhdy5h5vHqR1gfCQr_c0cAAMIZXcNhlIZ30u_45mxTL_Unra24blayXGq5HSNhc2FcOOw&_hsmi=444434988&utm_content=444434988&utm_source=hs_email By correcting, replacing, or silencing disease-causing genes, gene therapies offer the potential to treat disorders at their source rather than simply managing symptoms. Several gene therapies have already reached the clinic for inherited diseases, demonstrating the promise of genetic medicines. Yet, despite a few landmark approvals for central nervous system (CNS) disorders, widespread and precise delivery to the brain remains one of the field’s greatest challenges. The problem is often not the therapeutic gene itself, but how to deliver it. The brain is protected by multiple biological barriers that prevent most circulating molecules from entering brain tissue and reaching the intended targets. For Jingjing Gao, a biomedical engineer at the University of Massachusetts Amherst, tackling this challenge became a natural extension of her scientific journey. Trained as a chemical engineer, Gao’s doctoral research focused on designing novel materials capable of controlled drug release.