🔥The 9th Round of Easy Loan, Earn $40 Reward is in progress❗️ ⏰ Promotion Period: January 15th - Feburary 15th, 2025 👉 Register now and check more details at gate.io/campaigns/358

We’re excited to share our work on developing a high-titer, bifunctional LV approach for Sickle Cell Disease #SCD Thank you to everyone involved especially our collaborators from David Williams’ group. #GeneTherapy #lentivirus

Congratulations Alyssa! 🏆 Exactly two years on from the day she began her life-changing clinical trial, Alyssa attended a special ceremony last night, sweeping not just one but two awards! 'Young Person of Courage' and the 'Young Person of the Year 2024'.🌟 Leics Lieutenancy

Baltimore here we come! We're excted to see everyone again & learn more science at #ASGCT2024. We invite you to stop by on Thursday where Christopher Luthers (4th PhD candidate) will share our progress on developing a gene editing strategy for X-linked agammaglobulinemia (XLA).🧬

Every year ASGCT reminds us how blessed we are to be under Dr. Donald B. Kohn’s mentorship. 🩸🧬🦠 ✂️ #ASGCT2024

Chris Luthers presents his work where he is investigating our site-specific gene integration strategy on X-linked agammaglobulinemia (XLA) using a murine XLA-disease model. #ASGCT2024

5th yr PhD candidate, Eva Segura, presenting our progress on lentivirus-based gene therapy for Alpha-Thalassemia. ##ASGCT2024

Annual ASGCT Kohn lab (past and present) dinner. It's always great catching up with previous members. Hearing their success stories and how they're leading their own group inspires to us even more to be great in this field. #ASGCT2024

We want to congratulate our 4th yr PhD candidate Chris Luthers for receiving this year's John W. Phillips Legacy Award & UCLA's Dissertation Year Award; & also our 2nd yr Phd student, Dylan Smock for being a recipient of this year's UCLA Broad Stem Cell (BSCRC) training grant! 🍾

Congratulations Chris! 🎉

To correct the market failures around pediatric #celltherapies and #genetherapies, Crystal Mackall & colleagues propose a new model to lead late-stage development and commercialize these outside of traditional routes in the US. @MackallLab Stanford Medicine nature.com/articles/s4159…

How do we take the shining example of Emily Whitehead, cured of her pediatric cancer, and scale it? Dr. Crystal Mackall @MackallLab, Dr Donald Kohn The Kohn Lab UCLA, and a team of fellow SMEs across all facets of the problem space offer an actionable path. #PlatformCures #HealthJustice

In the media: Dr. Donald Kohn The Kohn Lab UCLA spoke to The Associated Press about the financial disincentives that threaten to halt the progress of promising gene therapies. apnews.com/article/rare-d…

Scenes from a moustache party celebrating 5-year-old Jakob's gene therapy treatment for ADA-SCID in a clinical trial led by Dr. Moustache himself AKA Dr. Donald Kohn. Learn more about the @CIRMNews-funded therapy: capitolweekly.net/bubble-boy-tre…

Lead by 4th yr PhD student Chris Luthers , we utilized Single-Stranded Virus Seq to determine wide variability in DNA contamination across rAAV preps. We characterized the effects & showed that DNAse treatment on these preps improved our CD34+ HSPC gene editing results.

A $14.7M California Institute for Regenerative Medicine grant will help Dr. Donald Kohn The Kohn Lab UCLA advance a gene therapy for ADA-SCID toward FDA approval, making this life-saving therapy that has so far successfully treated 48 of 50 children in clinical trials, available to kids everywhere. stemcell.ucla.edu/news/donald-ko…

Thank you California Institute for Regenerative Medicine for your endless support. Facing some adversities, the ADA-SCID gene therapy was on hold. Every year, more parents wonder when their child will get the chance. CIRM is now giving us another opportunity to continue this trial and move towards FDA approval.

5-year-old Jakob Guziak born with ADA-SCID, a deadly immune disorder, received gene therapy developed by Dr. Donald Kohn The Kohn Lab UCLA. “It’s not just a new life for Jakob, but it's a new life for us as parents,” his mother Andrea Fernandez said. stemcell.ucla.edu/news/after-lif…

We’re extremely happy to see 9 children living symptom-free 2 years post-GT. It was a long journey but these LAD-I GT trials emphasize the importance of supporting therapies for rare diseases in our community. #raredisease #genetherapy

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

In the news: Five years after receiving #GeneTherapy in a clinical trial led by Dr. Donald Kohn The Kohn Lab UCLA for a life-threatening immune disorder, the Langenhop siblings are now thriving. The family shares their story with CBS News. cbsnews.com/news/kids-alwa…