What a decade of gene therapy research for sickle cell disease taught UCLA scientists When Dr. Donald Kohn began performing bone marrow transplants in 1987, he saw firsthand the promise and limitations of available treatments for life-threatening blood disorders. That experience plante...

A clinical trial for sickle cell disease that UCLA's Dr. Donald B. Kohn led over nearly a decade yields hard-won insights — and a roadmap for better clinical trial design for blood diseases.

Read more ➡️ bit.ly/4vHEX8y

#ResearchPowersProgress #SickleCellDisease #GeneTherapy

About 1 in 3 Angelenos has fatty liver disease. UCLA scientists may have just found a new way to treat it by targeting dysfunctional immune cells dubbed "zombie cells." 🧟🧫

#ResearchPowersProgress

🔗 https://ow.ly/1gKx50YKAuB

Ahead of #WorldParkinsonsDay 🧠, we’re highlighting the work of Dr. Avi Samelson, who seeks to uncover the mechanisms that drive protein aggregation — a hallmark of neurodegenerative diseases like Parkinson’s. Learn more about his research: stemcell.ucla.edu/member-directory/avi-sam...

All 3 Langenhop kids were born with a rare, often-fatal immune disorder called LAD-I. After joining a UCLA #GeneTherapy trial, they're now "normal, happy kids" with functional immune systems.
Their trial participation helped make last week’s FDA accelerated approval possible.
💙 bit.ly/4sUCztg

A photograph of UCLA's Dr. Donald Kohn wearing a blue shirt and tie with a white lab coat. There is a quote overwriting the image. The quote reads, "I saw a marked improvement across all patients in terms of restored immune function. Witnessing that these patients no longer battle life-threatening infections has been incredibly meaningful." Attribution is: Distinguished professor of microbiology, immunology and molecular genetics Dr. Donald Kohn

How does the FDA-approved LAD-I gene therapy work?

Doctors take the patient's OWN stem cells, add a healthy copy of the mutated gene, and return them. No donor needed. No transplant rejection risk.

All 9 trial patients: restored immune systems.

🔗 lnkd.in/gDJzjgvx

#GeneTherapy #StemCells

The FDA granted accelerated approval for KRESLADI, a gene therapy for a rare pediatric immune disorder called LAD-I.

Dr. Donald Kohn at UCLA has spent 30+ years developing therapies like this. All 9 trial patients have restored immune function.

This is what science is for. 🔬
bit.ly/4uVBcfg

UCLA researchers receive $13.9 million CIRM grant to identify drug targets for autism and schizophrenia using human stem cell models A team of researchers led by UCLA’s Dr. Daniel Geschwind has received a $13.9 million grant from the California Institute for Regenerative Medicine to use human stem cell-based models to uncover the m...

UCLA researchers led by Dr. Daniel Geschwind have been awarded a $13.9M grant from @cirmnews.bsky.social to use stem cell models to identify drug targets for autism and schizophrenia — conditions that affect tens of millions of people in the U.S.

🔗 bit.ly/4tg4zY9

#ResearchPowersProgress

Can you #GuessTheCells? Zoom in. Look closer. 🔬 This microscopy image shows a portion of an organ. Let us know in the comments what you think it is! 👇

📷: Kevin Shih/Pearl Quijada Lab

Overcoming T cell tolerance to tumor self-antigens through catch-bond engineering T cells are often weakly responsive to tumor self-antigens because of central tolerance, constraining their ability to eliminate tumors. We exploited mechanical force to engineer a weakly reactive T c...

Engineering #TCR #catch #bonds for #tumor #immunity.

@science.org

Chris Garcia @stanforduniversity.bsky.social @hhmi-science.bsky.social

Owen Witte @uclastemcell.bsky.social

Brian Evavold @utah.edu

www.science.org/doi/10.1126/...

Mark your calendars for Thurs., April 2 as @drshrutinaik.bsky.social of the Icahn School of Medicine at Mount Sinai presents, "Mechanisms of Tissue Adaptation and Maladaptation” at 12pm in CHS 13-105.

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

Register today: bit.ly/4rLfVC9

Universal, ready-to-use immunotherapy detects and destroys endometrial cancer Endometrial cancer is the most common gynecologic cancer in the United States and is one of the few cancers in which survival rates have steadily declined over the last few decades. The most aggressiv...

UCLA scientists develop a universal, ready-to-use immunotherapy that detects and destroys endometrial cancer, the most common gynecologic cancer in the United States.

stemcell.ucla.edu/news/univers...

#ResearchPowersProgress

Fat cells burn energy to make heat – making them the next frontier of weight loss therapies GLP-1 drugs like Ozempic and Wegovy aid in weight loss by suppressing appetite. Researchers are looking next at how the body expends energy – including through fat.

UCLA scientist Claudio Villanueva explains why the next frontier of weight loss therapies will center around fat cells + how fat is far more than just a storage for excess calories in @us.theconversation.com ➡️ bit.ly/4us58PZ

Congratulations to center member Kathrin Plath on this distinction from @isscr.org! 🎉

February 17, 2026 | Nanoparticle-based gene editing could expand treatment options for cystic fibrosis - California NanoSystems Institute FEATURED NEWS California NanoSystems Institute > Faculty News > February 17, 2026 | Nanoparticle-based gene editing could expand treatment options for cystic fibrosis Nanoparticle-based gene editing c...

UCLA researchers led by @cnsiatucla.bsky.social member Steven Jonas (@uclastemcell.bsky.social) developed lipid nanoparticles that deliver a full CFTR gene into airway cells, restoring function in a cystic fibrosis model. CNSI’s EICN and ALMS supported this study:
cnsi.ucla.edu/project/febr...

Scenes from our 22nd Annual Stem Cell Symposium! 📸🧬🧫

See more photos from the day on our Flickr: lnkd.in/grCgteT3

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