UCLA Broad Stem Cell Research Center

This is why rare disease research matters. 🧬

Ahead of #RareDiseaseDay, read how a blood stem cell gene therapy co-developed by UCLA's Dr. Donald B. Kohn restored immune function in 59 children born with ADA-SCID, including Eliana: bit.ly/4kVdxXC

Considerations for the future of in vitro gametogenesis in fertility care - @hannahlandecker.bsky.social @uclacrshe.bsky.social @uclamcdb.bsky.social @uclastemcell.bsky.social go.nature.com/4l2e9en

A BSCRC Stem Cell Seminar Series poster advertising that Harvard University's Dr. Clifford Tabin will present, "The evolutionary changes in the timing of limb formation" at 12pm in CHS 13-105. Please see post for registration link.

Mark your calendars for Thursday, March 5 as
@harvard.edu's Dr. Clifford Tabin presents, "The evolutionary changes in the timing of limb formation" at 12pm in CHS 13-105.

The seminar is open to all UCLA faculty, students and staff! 🐻

Register today: bit.ly/4l3bTmX

Stylized valentine card with light blue background and dark blue elements and image of three human hearts in a row, with text: "UC heals broken [hearts image]. A newborn baby born with holes in her heart is alive today thanks to pediatric cardiologists at UCLA Health."

A baby born with holes in her heart is alive today thanks to pediatric cardiologists at @uclahealth 🫀 💙 💛 #ValentinesDay #HeartHealth https://bit.ly/406Qgs7

A young girl with glasses and a hat is smiling outdoors in a park setting. The image text reads: "Gene therapy offers kids with rare immune disorder a new chance. A gene therapy developed by UCLA offers young patients a new chance by editing the gene causing their rare immune disorder, LAD-1." Several people and trees are visible in the blurred background.

A @uclastemcell.bsky.social and Rocket Pharma gene therapy is transforming lives for kids with LAD-1. Marley, diagnosed at 8, is now healthy and enjoying life thanks to a CIRM-funded trial correcting her own stem cells. Read her inspiring story: https://bit.ly/4aBvHZT #RareDisease #GeneTherapy

Nanoparticle-based gene editing could expand treatment options for cystic fibrosis UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

UCLA scientists have shown that lipid nanoparticles — the same tiny particles used to deliver mRNA vaccines — can insert an entire healthy gene into human airway cells, restoring key function in a lab model of cystic fibrosis. ➡️ bit.ly/4rVYWh2

American Heart Month reminder: Do what makes your heart happy! 💙💛

#AmericanHeartMonth #RegenerativeMedicine

Join us on Tuesday, Feb. 17 as Luca Danelli @natcomms.nature.com @natcancer.nature.com presents, "Understanding the editorial process at Nature journals" at 12:30pm in Boyer 130. The seminar is open to all UCLA faculty, students and staff! 🐻

UCLA scientists identify optimal design for off-the-shelf immunotherapy targeting solid tumors FindingsA UCLA research team has identified the best design for a promising new type of immunotherapy that could be mass-produced to treat multiple solid tumors. The study focused on engineered invari...

A UCLA research team has identified the best design for a promising new type of immunotherapy that could be mass-produced to treat multiple solid tumors. ➡️ bit.ly/4s0hmNR

#ResearchPowersProgress

... and we're doing it again THIS FRIDAY, February 6! Join us for our Annual Stem Cell Symposium, featuring an outstanding lineup of speakers who will present cutting-edge advancements across the full spectrum of stem cell research. Register now: stemcell.ucla.edu/events/22nd-...

UCLA scientists discovered that muscle stem cells in aging mice accumulate a protein that extends the cells’ lifespan at the cost of slower tissue repair. This finding could lead to therapies for healthier aging that balance stem cell activation with survival. ➡️ stemcell.ucla.edu/news/muscle-...

Lab-grown organoids reveal how glioblastoma outsmarts treatment UCLA scientists have developed advanced miniature 3D tumor organoid models that make it possible to study glioblastoma tumors in a setting that closely mirrors the human brain, shedding light on how t...

UCLA scientists have developed mini 3D tumor organoid models that make it possible to study glioblastoma tumors in a setting that closely mimics the human brain, shedding new light on how the aggressive cancer outsmarts treatment.

stemcell.ucla.edu/news/lab-gro...

Trump one year on: How six US researchers plan to protect science amid chaos and cuts From education to pandemic preparedness and public health, the past year has seen huge stress put on US science.

New in @nature.com: Center member Amander Clark shares how higher education could be reimagined to better serve the public good:

www.nature.com/articles/d41...

Scientists are inventing treatments for devastating diseases. There’s just one problem. Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

Therapies that fix genetic illnesses at their root are already here. But many patients are stuck waiting for treatment because "getting to the end zone of an approved drug is very challenging," said Dr. Donald Kohn, who co-developed a gene therapy for ADA-SCID. www.washingtonpost.com/health/2026/...

Restoring the healthy form of a protein could revive blood vessel growth in premature infants’ lungs FINDINGSA UCLA-led research team has discovered a molecular switch that determines whether tiny blood vessels in premature infants’ lungs can regenerate after injury. A failure of this repair process ...

New in @cp-cellstemcell.bsky.social: A UCLA-led research team has discovered a molecular switch that determines whether blood vessels in premature infants' lungs can regenerate after injury, offering key insight into bronchopulmonary dysplasia, a serious lung disease. ➡️ bit.ly/3LwCcVi

Rebalancing NTRK2 isoforms promotes vascular regeneration in bronchopulmonary dysplasia Zhang et al. uncover a pathological NTRK2 isoform switch in gCaps that impairs vascular regeneration in bronchopulmonary dysplasia. Restoring full-length NTRK2 reverses vascular and alveolar defects in hyperoxic lung injury. These findings highlight isoform-specific NTRK2 targeting as a promising regenerative strategy.

Online Now! Rebalancing NTRK2 isoforms promotes vascular regeneration in bronchopulmonary dysplasia #stemcells

Mark your calendars for Thursday, January 8 as UCLA's Dr. Sophie Deng presents, "Stem cell-based therapy for corneal diseases" at 12pm in CHS 13-105.

The seminar is open to all UCLA faculty, students and staff! 🐻

Register today: bit.ly/4jbXhAR

YouTube video by UCLA Broad Stem Cell Research Center From Isolation to Inspiration: Evangelina's Journey with ADA-SCID

Stories like Evangelina's highlight why CIRM funds research. Supporting the scientific path to clinical trials helps us achieve life-changing treatments and cures. Watch her inspiring speech: https://youtu.be/SofJVAi19_Y?si=6dU-dHyrbramdtM6

🎥 : @uclastemcell.bsky.social

Experimental gene therapy treatment created at UCLA gives 'bubble girl' a new life An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

In the news via @abc7.bsky.social: A girl born without a functioning immune system is thriving as a healthy teenager today — thanks to an experimental gene therapy treatment developed at UCLA.

abc7.com/post/experim...

David Baltimore (1938–2025) - Nature Biotechnology Nature Biotechnology - David Baltimore (1938–2025)

"Among David’s greatest legacies is not a single discovery or institution, but a community the constellation of people he trained, inspired and bound together through six decades of mentorship."

Read the tribute by Drs. Lili Yang, Antoni Ribas and Owen Witte: www.nature.com/articles/s41...

Meet Dr. Mingxia Gu, a stem cell biologist who engineers miniature human organs like hearts and lungs to develop personalized treatments.

Read our Q&A to learn about her mission to reverse aging, how her father inspired her and the silver lining to living with face blindness. tinyurl.com/MingxiaGu

UCLA scientists develop one-product-fits-all immunotherapy for pancreatic cancer Pancreatic cancer is among the deadliest forms of cancer, with most patients diagnosed after the disease has already spread throughout the body. The five-year survival rate for metastatic cases hovers...

UCLA researchers have developed a new “off-the-shelf” cell therapy approach that could expand access to immunotherapy for pancreatic cancer.

“Our goal is to create a therapy that’s ready when patients need it,” says lead scientist Dr. Lili Yang.

stemcell.ucla.edu/news/ucla-sc...

Game changer for kidney transplant patients: UCLA clinical trial could eliminate need for lifelong immunosuppressive drugs When Karina Ledesma’s kidneys failed in high school, she was told she’d need a transplant — and a lifetime of immunosuppressive drugs to keep it working. But thanks to an experimental UCLA clinical tr...

A $6.7 million grant from @cirmnews.bsky.social will support UCLA's clinical trial of a stem cell-based therapy designed to help kidney transplant patients safely stop taking lifelong immunosuppressive drugs. ➡️ bit.ly/4rkHUcW

Love science that makes a real difference? We share the latest in stem cell research and regenerative medicine — from groundbreaking discoveries to the patients they help. Follow along! 👋🧬

Join us on Thursday, December 4 as Dr. Darrell Kotton of Boston University presents, “Pluripotent stem cells to model and treat lung disease” at 12pm in CHS 13-105.

The seminar is open to all UCLA faculty, students & staff! 🐻

Register today: bit.ly/4pg3b5E

El #CNIOStopCancer acoge el encuentro internacional #CFM_MolCellAging en el que 20 ponentes están exponiendo los últimos avances en el estudio de las bases biológicas del envejecimiento. Abordan el papel de las células madre, los telómeros, el metabolismo o el sistema inmunitario

Lab tours. Live demos. Real talk with scientists. Students from South Gate High School got an inside look at UCLA's breakthrough research in medicine and disease treatment. Their curiosity and questions brought so much energy to our labs! 🔬🧬🧫

The race is on to turn your body into a GLP-1 factory | CNN People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories that churn out a protein that is essentially the acti...

Gene therapy pioneer Dr. Donald Kohn talks to CNN about companies developing gene therapies to help the body make more GLP-1: “There is promise, but much more data would be needed to consider human clinical trials, as it likely would be irreversible,” he said.

www.cnn.com/2025/11/04/h...

What a fantastic way to kick off #CSStemCells2025—delegates and speakers enjoyed a scenic hike to the Hollywood Sign before diving into the latest in stem cell research! #WelcometoLA CedarsSinai @uclastemcell.bsky.social @cp-cellstemcell.bsky.social @cp-cellrepmed.bsky.social

Making clones in the lab this Halloween - what can be spookier?! Happy Halloween from the BSCRC! 🎃