5min chapter

FYI - For Your Innovation cover image

Therapeutic Human Gene Editing with Dr. David Liu

FYI - For Your Innovation

CHAPTER

Do Double Stranded Breaks Really Work?

There are more than 75 thousand human geane variants that are associated with genetic diseases. If you make a double stranded cut in d and a, you can stimulate a process called homology directed repair or h d r. It was originally considered an approach that might allow a precisely replace an original dana sequence in a human patient. But we now know a few things about it that have really limited its therapetic relevance. And just as your intuition might tell you, if you're trying to fix a specific deana sequence into a specific correctitina sequence, simply cutting the initial sequence doesn't actually fix the problem. The other example that's already shown promising clinical results is the crisper therapeutic

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