Gene therapy went through a ~20 year "winter"
Then this in < 1 month
@theLancet @NEJM @Naturepic.twitter.com/r0qpjnbD6y
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and lenti-SCT - remarkable effects in ALD and MLD: http://www.nejm.org/doi/full/10.1056/NEJMoa1700554 … and https://www.ncbi.nlm.nih.gov/pubmed/27289174
That’s awesome!
Might add the $ions and $alny TTR #amyloidosis drugs to this list
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