Missed stand up for science because my daughter was getting her 2nd ACL surgery. She's an awesome soccer player. So hard to see her go through this again.

Great lunch to celebrate recent papers in Sci Trans Med and Molecular Therapy. Great job everyone!

If you want a competitive advantage over China we need to invest in our best and brightest... especially those who would not otherwise have access to science education. They come with a fierce motivation to prove themselves.

6/ Our approach increases FIX expression to therapeutic levels, and is safe and well-tolerated in 1 yr follow up.

5/ We show that Repair Drive not only increases the frequency of cells with correct edits, but also decreases off-target integrations.

4/ The selective pressure can be increased through manipulation of the protein content in the diet, improving the frequency of HDR repaired hepatocytes from 1% to >25%.

3/ Cells correctly repaired through HDR have a selective advantage as they express a re-coded version of the essential gene, in tandem with the therapeutic protein. In the image below you can see clonal expansion of targeted cells (red).

2/ We use siRNA to temporarily knockdown the essential gene Fah in hepatocytes. In doing so, we kill off unedited or incorrectly edited cells. Over time, correctly repaired cells divide and regenerate the liver.

1/ Very excited to share the outstanding work of Marco De Giorgi and colleagues in Science Translational Medicine! science.org/doi/10.1126/scit… Repair Drive increases the efficiency of precise gene editing in the liver, through selective expansion of corrected cells.

Congrats to Marco De Giorgi on making the cover of Science Translational Medicine! @BCMFromtheLabs @somaticediting @GETC_BCM