Ancient Viruses Might Improve Gene Therapy
<p>Gene therapy is one of those advances that holds great promise, but fulfills that promise in small steps.</p>
<p>There have been a few recent successes. For example, <a href="https://medium.com/predict/crispr-based-therapy-treats-two-blood-disorders-b750b15e99d4" rel="noopener">CRISPR-based gene therapy for sickle cell disease and β-thalassemia</a>. And a <a href="https://medium.com/predict/send-in-the-mrna-213596312349" rel="noopener">new delivery system to transport custom mRNA into cells</a> and so potentially improve gene therapy is under development.</p>
<p>That last one illustrates a double sore spot for gene therapy: targeting and delivery. You need to get the therapeutic molecules to the right place (targeting) and you need a way to get them there and into the cells (delivery). If the targeting isn’t great, you’ll get off-target effects that may have bad consequences. If the delivery isn’t quite there, the therapeutic effects we’re after will be reduced.</p>
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