Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso-occlusive crises at 12 month #bmtsm Markus Y. Mapara 🇺🇦 Stephan Grupp MD PhD Akshay Sharma MD @akshaysharma.bsky.social nejm.org/doi/full/10.10…

No words. Only feelings of gratitude to Emily, her family, the Penn Medicine team Stephan Grupp MD PhD Carl June Bruce Levine, Ph.D. 🇺🇦 who saved her life - and created an entire new class of medicines. And a reminder - Emily almost died. The physicians' skill saved her - now we have 35,000 patients!

How bigs are the cells? Microorganisms in perspective 🦠 [🎞️ MetaBallStudios]

Ok this clip about navigating the American Healthcare System from the recent South Park ozempic special (which was brilliant) needs to be the opening of every healthcare conference panel

Congrats to 2024's recipients of the Children's Hospital Award for Excellence in #Mentoring Research Trainees! Drs. Amelia J. Eisch, Chén C. Kenyon, & Frederick "Chris" Bennett are recognized for their support of the next generation of scientists. Learn more: ms.spr.ly/6012YiCEa

Cellectis Receives Orphan Drug Designation for UCART22, its Allogeneic CAR T Product for Patients with Acute Lymphoblastic Leukemia> cellectis.com/en/press/celle… #allogeneic #celltherapy #genetherapy

Looking for the most effective ways to CRISPR edit human T cells? Look no further! Along with the team Penn we identified the engineering steps that control product function. Nearly 10 years of troubleshooting with Cas9 and novel base editors distilled down into 4 figures! (1/4)

“Our community is grieving and heartbroken over the loss of an extraordinary doctor…We call on the Mayor and City Council to honor her life and the lives of our patients and families we tirelessly care for, by investing in safe streets for Philadelphians” inquirer.com/opinion/commen…

To Uber, With Love Great Spencer for Hire reference: "“Shut up,” I explained." nytimes.com/2024/07/27/sty…

Abby D. Phillip Rachel Scott Kadia Goba If only they'd done the first debate

CIRP is collaborating with areas across Children's Hospital & Penn to examine how medical conditions affect driving, including the learning to drive process and crash risk to help teens and young adults learn to drive safely: ow.ly/IYZ050SPEno

Listen, if we're going to have a president whose last name ends in an s we all need to get on the same page right now. The possessive version ends in 's (Harris's). Not just a simple apostrophe (Harris'). I will not abide four to eight years of grammatical nonsense.

Somebody is gonna have to explain this one to me

Excited to share 👇 “A major limitation of hemophilia A gene therapy is progressive loss of expression. Here the authors demonstrate that low-level expression of a gain-of-function factor VIII variant may safely restore normal hemostasis and permit durability of expression.”

At the height of One Million Checkboxes's popularity I thought I'd been hacked. A few hours later I was tearing up, extraordinarily proud of some brilliant teens. A thread about my favorite story from running OMCB....

This is amazing

A brilliant long form article discussing the insanity of pennies Bonus use of the word trochilidine! NYT gift article nytimes.com/2024/09/01/mag… via @NYTimes

Congrats on 10K CAR T The EBMT