Gene Transfer Method of EPO Improves Cognition in Mouse Study

Administration of the hormone erythropoietin (EPO) using a gene transfer method improved spatial cognition, stimulated the branching of nerve stem cells, and preserved the brain region known as the hippocampus in a mouse model of…

Gene Therapy Approach Reported to Block Mutant Huntington’s Gene in Preclinical Studies

uniQure recently published results from preclinical studies of its gene therapy program, AMT-130, indicating that a one-time administration of AAV5-delivered therapy into the central nervous system can block the mutant HTT gene that causes Huntington’s disease (HD). These findings, in the article “Design, Characterization, and Lead Selection of Therapeutic miRNAs…

New Virus Variant May Advance Gene Therapy for Diseases Like Huntington’s

A research team successfully developed a virus variant that is more efficient than the current standard at crossing the blood-brain barrier and delivering genes to the central nervous system (CNS), making it a promising gene therapy approach to treating neurodegenerative diseases such as Huntington’s disease. The blood-brain barrier, made up of specialized…