Gene therapy is transforming how we treat genetic disorders, but what if mutations in more than 30 genes can cause similar symptoms?
Now published in @CellStemCell (https://t.co/Ub72yvUo0c), we report the development of a universal gene therapy for Diamond Blackfan anemia.
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Thrilled to share that I’ll be starting my lab as an Assistant Professor and Endowed Scholar in @UTSWMedCenter Molecular Biology Dept this Fall! 🥳 We’ll study #peroxisomes#mitochondria#metabolism via the lens of neonatology👶🏻, a very exciting frontier in medicine! #neotwitter
❓A one-time, durable, and even reversible way to prevent thrombosis? In this preprint, led by @lrbzldz, we show that epigenome editing of 🩸 #StemCells can durably reprogram platelet function: https://t.co/bioSZhRFuG
FDA just approved Kresladi (marnetegragene autotemcel) for severe LAD-I — a disease so rare there are ~25 new cases a year in the U.S. The pivotal data: 9 patients. 100% overall survival. All endpoints met. This approval is a masterclass in how to think about evidence standards when mechanism is unambiguous. https://t.co/7NgGdQnepv
In this piece, my mentor Stu Orkin and I (1) marvel at prime editing—discovery to disease correction for 2 patients after just 6 yr—wow Liu, Anzalone, Gori, Malech et al! & (2) consider the challenges to deliver genetic medicine’s promise for 2^n patients https://t.co/ni2hs4btt9
We fund researchers like @kennethschen, one of the brilliant minds that is helping to shape the future of cancer research. V Foundation researchers, throughout their careers, have generated $22 billion in additional cancer research funding. We truly fund the best of the best.
MECOM promotes malignant stem cell–like states in aggressive AMLs by directly repressing prodifferentiation gene regulatory programs. Read the plenary paper in Blood: https://t.co/LaUTyMPrmh #BloodJournal#PlenaryPaper#AML
Colliding ribosomes are potent signals of cellular stress. But do cells use ‘programmed’ ribosome collisions to regulate gene expression? I’m excited to present a new story led by Frederick Rehfeld revealing that the answer is YES! Read on to find out how. https://t.co/nnftTDxL7E
Congrats! 👏👏👏 Two CRI scientists named 2025 Pew-Stewart & Pew Scholars — @JaviGBermudez & @HijaiShin — to advance cancer, biomedical research. Read more about their #relentlessdiscovery ⬇️ https://t.co/MAFSU4zUqs @pewtrusts
Looking forward to the #NFRF2025@UTSWScience in October. https://t.co/mTjnLXxZzM. If you are an undifferentiated trainee @UTSWMedCenter, come check out highlights from new labs on campus.
Committed to advancing treatments for children with blood disorders, @RichardVoit is exploring the complexities of hematopoietic stem cells to enhance targeted leukemia therapies. Discover more: https://t.co/w3e9TvHjaK #NFRF2025#NFRF
Inspiring piece in @NatureCancer, "An unexpected career in cancer metabolism," highlights Dr. Ralph DeBerardinis & the pivotal moments that prompted him to pursue a career in cancer metabolism. @CRI_UTSW @HHMINEWS https://t.co/G92oSIn3mm
Be part of the Wagenblast Lab! We are hiring a Lab Manager for leukemia & blood stem cell research at @IcahnMountSinai in NYC, starting July 2025. See ad to apply!
Kids like KK motivate everything we do in the lab @utswcancer@CRI_UTSW @UTSW_PedsHemOnc. We are now hiring so if you are looking for the next step in your scientific journey or want to learn more about what we do, please reach out.
We at https://t.co/h1JtA2Hva3 are grateful to @TexasCCF for supporting our work. Thank you also to @dallascowboys@dak, @Jalen8Tolbert, @tonyromo, @TroyAikman for using their platform to raise awareness and funds for pediatric cancer research. https://t.co/8PTFZcyYqX
Thrilled to share the first pre-print from our lab! We tackled a big question: Why are some pediatric leukemias extremely aggressive? We explored the developmental origins of fusion-driven acute myeloid leukemia (AML). Let's dive in! #AML#NUP98 n/13
Delighted to have our Perturb-multiome paper, led by @jmartinrufino and #AlexisCaulier from our lab, published today in @ScienceMagazine. Please check out this powerful approach to decipher gene regulatory networks enriched for genetic variation: https://t.co/TOhYCqOvO1