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Nov 11, 2021

Surprise! Our Bodies Have Been Hiding a Trojan Horse for Gene Therapy

Posted by in categories: biotech/medical, genetics

The problem? Our bodies aren’t big fans of foreign substances—particularly ones that trigger an undesirable immune response. What’s more, these delivery systems aren’t great with biological zip codes, often swarming the entire body instead of focusing on the treatment area. These “delivery problems” are half the battle for effective genetic medicine with few side effects.

“The biomedical community has been developing powerful molecular therapeutics, but delivering them to cells in a precise and efficient way is challenging,” said Zhang at the Broad Institute, the McGovern Institute, and MIT.

Enter SEND. The new delivery platform, described in Science, dazzles with its sheer ingenuity. Rather than relying on foreign carriers, SEND (selective e ndogenous e n capsidation for cellular delivery) commandeers human proteins to make delivery vehicles that shuttle in new genetic elements. In a series of tests, the team embedded RNA cargo and CRISPR components inside cultured cells in a dish. The cells, acting as packing factories, used human proteins to encapsulate the genetic material, forming tiny balloon-like vessels that can be collected as a treatment.

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