The Kohn Lab UCLA
@Kohn_Lab
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Our mission is to treat patients suffering from primary immunodeficiency diseases with hematopoietic stem cell gene therapy.🩸🦠✂️🧬⛑: tweets from lab members.
Westwood, Los Angeles
Joined September 2020
New long-term follow study for the ADA-SCID lentiviral gene therapy is out!
nejm.org
Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a life-threatening inborn error of immunity for which lentiviral gene therapy has been investigated...
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
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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.
cbsnews.com
Alicia and Jon Langenhop's three children were each diagnosed with a rare disorder. A clinical trial was "a no-brainer."
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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
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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
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.
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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.
stemcell.ucla.edu
Days after their son was born in 2019, Andrea Fernandez and Kamil Guziak learned their new baby had a rare genetic disease and would be lucky to reach his second birthday.“You’re thinking, ‘How many...
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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.
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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.
stemcell.ucla.edu
Dr. Donald Kohn has been awarded a $14.7 million grant from the California Institute for Regenerative Medicine to develop a commercial manufacturing protocol for a groundbreaking gene therapy...
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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.
DNA Contamination Within Recombinant Adeno Associated Virus (AAV) Preparations Correlates with Decreased CD34+ Cell Clonogenic Potential https://t.co/Q2BKYpCikh
#MTMCD
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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
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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.
apnews.com
The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures.
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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
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
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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
nature.com
Nature Medicine - To correct the market failure around pediatric cell and gene therapies, the authors propose a new model to lead late-stage development and commercialize these therapies outside...
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Congratulations Chris! 🎉
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!
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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! 🍾
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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
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5th yr PhD candidate, Eva Segura, presenting our progress on lentivirus-based gene therapy for Alpha-Thalassemia. ##ASGCT2024
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@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
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Every year @ASGCTherapy reminds us how blessed we are to be under Dr. Donald B. Kohn’s mentorship. 🩸🧬🦠 ✂️ #ASGCT2024
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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'.🌟 @LeicsLieutenant
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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).🧬
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