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
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The Kohn Lab UCLA retweeted
#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: bit.ly/3NRnGsh
The Kohn Lab UCLA retweeted
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/news/kids-always…
The Kohn Lab UCLA retweeted
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/news/after…
The Kohn Lab UCLA retweeted
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/news/donal…
The Kohn Lab UCLA retweeted
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…
The Kohn Lab UCLA retweeted
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/article/rare-dise…
The Kohn Lab UCLA retweeted
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
nature.com/articles/s41591-0…
The Kohn Lab UCLA retweeted
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
nature.com/articles/s41591-0…
The Kohn Lab UCLA retweeted
@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
The Kohn Lab UCLA retweeted
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
ALT Alyssa at the award ceremony holding her award. She's wearing a denim jacket, with a pale green dress and shoulder length hair. Photo by 📸 Dave Morcom
The Kohn Lab UCLA retweeted
For readers interested in genome editing, this comprehensive review covers drug delivery systems for CRISPR-based genome editors, with a focus on AAV vectors and lipid nanoparticles bit.ly/3tTMzZY rdcu.be/dAhRO
The Kohn Lab UCLA retweeted
ASGCT recently contributed to positive changes in @US_FDA's regulation of two #genetherapy modalities: Human gene therapy products incorporating human #genomeediting + considerations for the development of #CART cell products.
Read more on our blog. bit.ly/3P7vHHf
The Kohn Lab UCLA retweeted
Why is #acceleratedapproval important for the treatment of #rarediseases? @FDACBER Director Peter Marks, MD, PhD, shares his opinion in our latest Community Quick Takes module.
Learn more from the full video on our #PatientEducation site. bit.ly/48E2MkP
The Kohn Lab UCLA retweeted
“How can we accelerate the path from gene to therapy? - Dr. Flotte (@UMassChan) is on Capitol Hill for #RareDiseaseDay sharing the current treatment landscape for #CGTs. #ASGCTadvocacy
The Kohn Lab UCLA retweeted
#SickleCellDisease (#SCD) will be the first focus of the Cell and #GeneTherapy (CGT) Access Model, the Biden-Harris Administration announced today. Read more from @CMSGov. go.cms.gov/49bOKYm
ALT Sickle cell patient
The Kohn Lab UCLA retweeted
Doudna Institute aims to use #CRISPR technology to cure hundreds of rare diseases, pushing boundaries for potential revolutionary health solutions 🧬💊 Find out more: statnews.com/2024/01/09/doud… #CRISPR
The Kohn Lab UCLA retweeted
Excited to lead the Artemis-SCID project team for the new @igisci @DanaherCorp collaboration to advance CRISPR therapies for rare diseases @UCSFChildrens!
@doudna_lab @UrnovFyodor @Kohn_Lab @ucsf @UCSFPediatrics
Here's the non-paywall IGI press release
This Post is from an account that no longer exists. Learn more
The Kohn Lab UCLA retweeted
CIRM applauds the @US_FDA for this landmark decision! 👏🎉 #SickleCell #CRISPR
nbcnews.com/health/health-ne…
The Kohn Lab UCLA retweeted
On this historic day for #SCD patients & families, thank you to @HPKiem (@FredHutch), Matt Porteus (@StanfordMed), Drew Campbell (@ChildrensNatl), Clark Paramore (@bluebirdbio), & SCD warrior Nasir & mom SueAnn, for presenting at our Senate briefing last summer! #ASGCTAdvocacy
The Kohn Lab UCLA retweeted
.@US_FDA approved two #genetherapies, #Casgevy and #Lyfgenia, to treat #sicklecell disease (#SCD) in patients 12 years & older. These are the first GTs approved for SCD in the U.S., & Casgevy is the first approved therapy in the U.S. using #CRISPR. bit.ly/3taZOpk
