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

The novel genetic engineering approach, tested in mice and laboratory-grown nerve and light-receiving cells, will initially have research applications.

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. Continue reading here and check out the original work here.

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