The Kohn Lab UCLA

Our mission is to treat patients suffering from primary immunodeficiency diseases with hematopoietic stem cell gene therapy.🩸🦠✂️🧬⛑: tweets from lab members.

[Rocket Pharmaceuticals @RocketPharma]

#NEWS: Rocket Pharmaceuticals today announced the U.S. FDA approval of the first #GeneTherapy for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I).

More: https://t.co/YrJuWo1r3J

[The Kohn Lab UCLA @Kohn_Lab]

In collaboration with @RocketPharma , we celebrate the first FDA-approved gene therapy supported by @CIRMnews and CA taxpayer funding. #raredisease matters.

[UCLA Broad Stem Cell @UCLAstemcell]

The FDA has granted accelerated approval for KRESLADI, a gene therapy for LAD-I — a rare immune disorder that can be fatal in young children.
Led by @Kohn_Lab @UCLA, all 9 clinical trial patients survived with restored immune function.
🔗https://t.co/YRAIw99k5Y #GeneTherapy

[The Kohn Lab UCLA @Kohn_Lab]

New long-term follow study for the ADA-SCID lentiviral gene therapy is out! https://t.co/IKocp0yOQ0

[UCLA Broad Stem Cell @UCLAstemcell]

A blood stem cell gene therapy co-developed by @UCLA's Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications reported. #ResearchPowersProgress @Kohn_Lab
https://t.co/nDGhz1PkuH

[The Kohn Lab UCLA @Kohn_Lab]

@ASGCTherapy ‘s Founders Award recipient for 2025 #ASGCT2025

[UCLA Broad Stem Cell @UCLAstemcell]

In the news: Five years after receiving #GeneTherapy in a clinical trial led by Dr. Donald Kohn @Kohn_Lab for a life-threatening immune disorder, the Langenhop siblings are now thriving. The family shares their story with @CBSNews. https://t.co/GhiSYJ2l2a

[The Kohn Lab UCLA @Kohn_Lab]

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

[The Kohn Lab UCLA @Kohn_Lab]

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

[UCLA Broad Stem Cell @UCLAstemcell]

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 @Kohn_Lab @UCLA.

[UCLA Broad Stem Cell @UCLAstemcell]

5-year-old Jakob Guziak born with ADA-SCID, a deadly immune disorder, received gene therapy developed by Dr. Donald Kohn @Kohn_Lab. “It’s not just a new life for Jakob, but it's a new life for us as parents,” his mother Andrea Fernandez said. https://t.co/mrVLCn8ehn

[The Kohn Lab UCLA @Kohn_Lab]

Thank you @CIRMnews 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.

[UCLA Broad Stem Cell @UCLAstemcell]

A $14.7M @CIRMnews grant will help Dr. Donald Kohn @Kohn_Lab 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. https://t.co/UTWrMA3HY4

[UCLA Broad Stem Cell @UCLAstemcell]

A $14.7M @CIRMnews grant will help Dr. Donald Kohn @Kohn_Lab 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. https://t.co/UTWrMA3HY4

[The Kohn Lab UCLA @Kohn_Lab]

Lead by 4th yr PhD student @C_Luthers31 , 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.

[Molecular Therapy Family of Journals @MolTherapy]

DNA Contamination Within Recombinant Adeno Associated Virus (AAV) Preparations Correlates with Decreased CD34+ Cell Clonogenic Potential https://t.co/Q2BKYpCikh #MTMCD

[UCLA Broad Stem Cell @UCLAstemcell]

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: https://t.co/HQ2jX85ZsY

[UCLA Broad Stem Cell @UCLAstemcell]

In the media: Dr. Donald Kohn @Kohn_Lab spoke to @AP about the financial disincentives that threaten to halt the progress of promising gene therapies. https://t.co/IzpYrhwgiv

[Fyodor Urnov @UrnovFyodor]

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 @Kohn_Lab, and a team of fellow SMEs across all facets of the problem space offer an actionable path.
#PlatformCures
#HealthJustice

[Nature Medicine @NatureMedicine]

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 @StanfordMed
https://t.co/8nWJevprto

[The Kohn Lab UCLA @Kohn_Lab]

Congratulations Chris! 🎉

[UCLA Microbio, Immunology, & Molecular Genetics @MicrobioUcla]

We are thrilled to announce that the distinguished John W. Phillips Legacy Award has been granted to Christopher Luthers this year. Christopher Luthers is a rising 5th year Molecular Biology PhD student in the lab of Dr. Donald Kohn of MIMG. Congratulations, Chris!

[The Kohn Lab UCLA @Kohn_Lab]

We want to congratulate our 4th yr PhD candidate @C_Luthers31 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 @UCLAstemcell (BSCRC) training grant! 🍾

[The Kohn Lab UCLA @Kohn_Lab]

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

[The Kohn Lab UCLA @Kohn_Lab]

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

[The Kohn Lab UCLA @Kohn_Lab]

@C_Luthers31 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