Proof-of-Concept Study Advances Potential New Way to Deliver Gene Therapy

Seth Blackshaw, PhD

Johns Hopkins Medicine researchers say they have successfully used a cell’s natural process for making proteins to “slide” genetic instructions into a cell and produce critical proteins missing from those cells. If further studies verify their proof-of-concept results, the scientists may have a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Such disorders include neurodegenerative diseases that affect the brain, including Alzheimer’s disease, forms of blindness, and some cancers. For those looking to develop treatments for diseases where cells lack a specific protein, it’s critical to precisely target the cell causing the disease in each structure, such as the brain, to safely kickstart the protein-making process of certain genes, says Seth Blackshaw, PhD, Professor of Neuroscience in the Sol Snyder Department of Neuroscience and member of the Institute for Cell Engineering at the Johns Hopkins University School of Medicine. Therapies that don’t precisely target diseased cells can have unintended effects in other healthy cells, he adds.

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