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Researchers show safer, more targeted way to deliver CRISPR gene therapy

Researchers have found that liposomes can be triggered by light to release their payload in specific parts of the body. Currently CRISPR gene therapy technology uses a guide RNA to find the faulty gene sequences and a Cas9 protein to correct it. The CRISPR molecules are introduced into viruses to get into their target cells. While this is revolutionary, using a virus to deliver anything can have potential adverse reactions. Researchers have now shown that liposomes (cargo vesicles compose of lipids) can carry the CRISPR molecules to where it needs to go in the body. This can be a safer delivery method compare to viruses. The liposomes can be “turned on” by light stimulation and release the molecules inside and thus, repair the gene of interest. If the liposome has to go into deep-seated areas, the researchers believe they can use X-rays to trigger the liposomes into releasing their contents. 

What this means for Usher syndrome: Researchers have already started looking at CRISPR therapy for Usher syndrome. This new innovation in using liposomes to deliver CRISPR molecules could be an improvement to using viruses by preventing potential adverse reactions individuals may have. 

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