A new study in NEJM reports that an investigational gene therapy restored immune function in 9 children with LAD-I, a rare & life-threatening immune disorder. Those children are now living symptom-free 2 years after treatment in clinical trials co-led by The Kohn Lab UCLA UCLA.

For #CysticFibrosisAwarenessMonth, we're spotlighting the efforts of Drs. Brigitte Gomperts, The Kohn Lab UCLA & Steven Jonas, who are developing an innovative, inhalable gene-editing platform to correct the root cause of this life-threatening disease. ➡️ ucla.in/4jJNuRU

This week, the Kohn lab is coming to New Orleans for #ASGCT2025 ! Another special year for us because our PI is this year’s ASGCT Founders Award recipient. Don’t miss the Founder’s Keynote tomorrow afternoon! Look out for the 👨🏻#genetherapy #PIDD #raredisease

ASGCT ‘s Founders Award recipient for 2025 #ASGCT2025

Incredible story that we learned about today at #ASGCT2025!

A few weeks ago we introduced you to KJ, who made medical history as the first patient to receive a personalized gene editing therapy. After almost a year at CHOP, KJ is now at home with his family. 🎓 Tune in to NBC's TODAY tomorrow morning to hear from his family!

Aldrovandi was honored with the 2025 Douglas K. Richardson Award for Perinatal and Pediatric Healthcare Research. David Geffen School of Medicine at UCLA newsroom.ucla.edu/dept/faculty/d…

UCLA Samueli 2025 graduate commencement: The National Anthem, sung by ChemE PhD student Julie Zhu, accompanied by CS Prof Todd Millstein #UCLA2025 #engineerchange

A gene therapy trial for rare immune disorder LAD-I, developed by Rocket Pharmaceuticals and co-led by UCLA Broad Stem Cell and backed by a $5.8M CIRM grant, restored immune function in all 9 children treated—marking a major step forward in #GeneTherapy. #RareDisease stemcell.ucla.edu/news/gene-ther…

The guilt is real

We kicked off our season of giving right here in our own backyard at CHOC! Here’s to more hope, more healing, and new beginnings. ✨

Congrats to Yu Huang, professor of UCLA Materials Science and Engineering UCLA Chem & Biochem & a CNSI at UCLA member, on receiving the 2025 Global Energy Prize for her work in catalyst development — the 1st UCLA faculty & 1st woman to win in the non-conventional energy category. University of California samueli.ucla.edu/ucla-materials…

A new Los Angeles Times piece highlights the sweeping impact of the proposed $18 billion NIH budget cut. Center member Dr. Antoni Ribas weighs in with a stark warning: "The cuts in academia will lead to less positions being offered." UCLA Jonsson Comprehensive Cancer Center latimes.com/science/story/…

Since 2006, CIRM has committed $447M to fund training for 4,600+ #biomedical students in regenerative medicine. These programs help prepare the workforce that will turn today’s scientific discoveries into tomorrow’s treatments. blog.cirm.ca.gov/2025/07/21/20-…

With support from NIH, UCLA researcher Dr. Mingxia Gu & her team have created the first mini-lungs with specialized blood vessels—offering insights into how lungs develop and function, and unlocking new approaches to tackling pulmonary vascular disease. nih.gov/news-events/ni…

CNSI member Steven J. Jonas (UCLA Health) has received a $400K @HyundaiHope grant to develop nanotech “liquid biopsy” tools for more precise, less invasive childhood cancer care. Nearly $2.8M awarded to UCLA to date. #ResearchPowersProgress cnsi.ucla.edu/august-7-2025-…

Professor Michael Jung helped develop two FDA-approved drugs — Xtandi and Erleada — that have extended and improved the lives of thousands of men with prostate cancer. Learn more about how UCLA #ResearchPowersProgress: ucla.edu/research

Congratulations to pediatric cancer researcher Dr. Steven Jonas (Steven Jonas) on receiving a $400K Hope On Wheels grant to advance nanotech-based liquid biopsy tools for more precise, less invasive monitoring of childhood cancers. Read more: bit.ly/45t49Dd

This colorful handprint ceremony returned last week, thanks to Hope On Wheels. Dr. Steven Jonas was awarded a $400,000 grant to support his lab, seeking to design diagnostic tests that offer a "liquid biopsy" of underlying cancer. Read more: ucla.in/40ZaaX3

At UCLA Broad Stem Cell, Dr. Brigitte Gomperts and her team built a 3D stem cell-based lung model to study idiopathic pulmonary fibrosis — bringing hope to those with this deadly disease. Learn more about how @ucla #ResearchPowersProgress: ucla.edu/research