Here’s an overview of a new potential treatment for transthyretin (ATTR) amyloidosis.
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Transthyretin (TTR) is expressed in liver, is secreted into blood, and functions as vitamin/hormone transporter.
ATTR amyloidosis is caused by abnormal aggregates of destabilized TTR monomers (normally forms tetramers)—either mutant or wild-type—damaging nerves and/or heart.
Jun 27, 2020 • 22 tweets • 15 min read
How to use #CRISPR gene editing to fight heart disease, the #1 killer worldwide:
We asked: are there more PCSK9’s to be discovered?
Nov 26, 2019 • 17 tweets • 8 min read
Lulu and Nana, humanity’s first gene-edited babies, both have some CRISPR edits in the CCR5 gene, as He Jiankui (JK) attempted. His goal was to confer HIV resistance.
Unfortunately, the editing turned out quite badly, as shown by *his own data* in *his own manuscript*. (thread)
This is a Sanger sequencing chromatogram of a PCR amplicon of the CCR5 target site, using DNA from cells extracted from Lulu’s embryo prior to pregnancy.
In the manuscript, JK claims there’s a wild-type allele and a 15bp deletion allele.
Nov 25, 2019 • 12 tweets • 10 min read
Today is the 1-year anniversary of @antonioregalado breaking the news about Chinese scientist He Jiankui (JK) using gene-edited human embryos to establish pregnancies.
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