How safe is in utero stem cell transplantation? This Systematic Rev ISPD journal Prenatal Diagnosis found 66 procedures in 52 fetuses 1967-2023. Acute 48h fetal procedural complication rate =4.5%; all were in cases <1994
UCL-IfWH Karolinska Institutet @UCLH
obgyn.onlinelibrary.wiley.com/doi/10.1002/pd…

Magnificent advance for the field of N=1 CRISPR cures.

Technical correction - for the aficionados - this is epigenome editing, not genome editing. No targeted genetic alteration will take place here.

Revolutionary Crispr gene editing speeds from lab to treatment room ft.com/content/e3c121…

What a privilege to mentor Lexi this summer in William Peranteau lab. Her scientific curiosity, positive attitude, and love for Insomnia cookies made for a fun + productive few months. I am so excited to see what this superstar future bioengineer will accomplish in college and beyond!

Mike Mitchell opens the day with brilliant talk on LNP therapies for treating disease before birth 👏

And he is hiring! What are you waiting for ?! 👇

Controlled Release Society #crs2022

Edits to a cholesterol gene could stop the biggest killer on earth technologyreview.com/2022/07/12/105…

House Democrats are seeking to pass a wide-ranging slate of gun reforms this week as a swift response to the growing number of mass shootings. nytv.to/8BEQl

Excited to announce the first paper from the lab is out today (on the cover!!) Science Translational Medicine

science.org/doi/10.1126/sc…

Exciting day for Andrea Toth (MD/PhD student in our group and general superstar) and I, along with our co-authors.

Last night was an abomination in our city. Our community is dying. Where is the outrage…from everyone?

👏👏👏👏👏 I am with you Sen McMorrow! Mallory McMorrow I am pushing the rock up the hill with you. We will get it there.

If you haven’t watched this speech by Michigan state Senator Mallory McMorrow on school shootings & gun violence, you’re welcome.

Lipid nanoparticles incorporating a GalNAc ligand enable in vivo liver ANGPTL3 editing in wild-type and somatic LDLR knockout non-human primates biorxiv.org/content/10.110…

Daniel J. Rader, M.D., FAHA, of the University of Pennsylvania, to receive 2021 Research Achievement Award, the American Heart Association’s highest scientific award spr.ly/6014JnPLn #AHA21

The 1st randomized trial of a booster (3rd) shot in 10,000 people, placebo-controlled, shows 95.6% efficacy, with 5 cases (Pfizer vaccine) vs 109 in placebo group, Delta variant, broad benefit across age groups ft.com/content/d4e58d…

In addition to moonlighting in William Peranteau’s lab on in utero gene therapies, Mike Mitchell Penn Bioengineering PhD student Maggie Billingsley, PhD’s day job is doing killer CAR T cell research. Check out her latest on mRNA LNPs for CAR T cell engineering at poster 73! #BMES2021 Biomedical Engineering Society

Incredible talk by Maggie Billingsley, PhD’s protégé and Mike Mitchell Penn Bioengineering undergrad Savan Patel on how hydroxycholesterol incorporation into LNPs enhances mRNA delivery to T cells! #BMES2021 Biomedical Engineering Society

Batting leadoff in front of a packed house is Mike Mitchell PhD student Maggie Billingsley, PhD with her latest on in utero mRNA LNP delivery - come join the fun in the women’s health tech session chaired by Prof. Brian Aguado in Grand Ballroom 3-4! #BMES2021 Biomedical Engineering Society

Another step towards therapeutic in vivo gene editing:

Treatment of patients with Leber congenital amaurosis type 10 (LCA10), announced by Dr. Mark Pennesi OHSU Casey Eye and Editas Medicine today.

Here’s the background & overview of the new results.

(thread)

twitter.com/kiranmusunuru/…

👋👋👋Congrats to Katalin Kariko @ Drew Weissman Penn Medicine for being awarded the #Lasker Prize - 'based on 20-plus years of work . . . and work by hundreds if not thousands of other scientists'
nytimes.com/2021/09/24/hea…

If you can’t get vaccinated for your family or your friends or neighbors, how about get vaccinated for the babies and toddlers!