Richard Voit

Pediatric hematology/oncology | Stem cell biology | AML | Gene therapy | CRISPR

[Richard Voit @RichardVoit]

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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[Richard Voit @RichardVoit]

Congrats Walter! Trainees and folks looking for a new and exciting challenge: don’t pass up this opportunity to work with Walter!

[Walter W. Chen, MD, PhD @WalterWChen1]

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

[Vijay Sankaran @bloodgenes]

❓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

[Sean @S_Gallagher23]

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

[Daniel Bauer @danielevanbauer]

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

[The V Foundation for Cancer Research @TheVFoundation]

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.

[Blood Journals Portfolio @BloodPortfolio]

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

[Richard Voit @RichardVoit]

Proud of you @tjflemin for your incredibly hard work on this. Now the next frontier: inhibiting this pathway in a clinically tractable way.

[Vijay Sankaran @bloodgenes]

Terrific to have our paper, led by @tjflemin, featured as a plenary article in @BloodPortfolio today: https://t.co/Bg0tX8YbJu

[Richard Voit @RichardVoit]

Congratulations @Mendell_lab. What an interesting mechanism!

[Joshua Mendell @Mendell_lab]

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

[Children’s Research Institute at UT Southwestern @CRI_UTSW]

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

[Richard Voit @RichardVoit]

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.

[UTSW Science @UTSWScience]

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

[Dr. Arnaldo Díaz @ADiaz_PhD]

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

[Elizabeth McKenna @ElizSMcKenna]

Catalytic inhibition of KAT6/KAT7 enhances the efficacy and overcomes primary and acquired resistance to Menin inhibitors in MLL leukaemia - by
@ShellainaGordon @florian_perner, @Armstrong_dfci, @MAF_Dawson @TheDawsonLab
et al. https://t.co/hdfSLRw7SU @PeterMacRes @DanaFarber

[Elizabeth McKenna @ElizSMcKenna]

KAT6A and KAT7 Histone Acetyltransferase Complexes Are Molecular Dependencies and Therapeutic Targets in NUP98-Rearranged Acute Myeloid Leukemia
@NMichmerhuizen, Emily Heikamp, @Armstrong_dfci, @CMullighan and colleagues https://t.co/orc4w45Iw8
@StJudeResearch @DanaFarber

[Elvin Wagenblast @Elvin_Wag]

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!

[Richard Voit @RichardVoit]

Congratulations @michaelpoeschla and @bloodgenes. Incredible work!

[Vijay Sankaran @bloodgenes]

Delighted to have our work on Polygenic Modifiers of #TelomereBiologyDisorders, led by @michaelpoeschla, along with @SashaGusevPosts, @mitchiela, @SharonSavageMD, @hemanth_tummala, and others, published as an advanced online paper in @jclinicalinvest today: https://t.co/pqfe7H1AOx

[Iannis Aifantis @iannisaifantis1]

Excited to share with all our latest work on the use of single cell tools in following IDH mutant AML patients during their treatment! Out today @CellStemCell !! An amazing collaboration with @AkEisfeld & @PapaemmanuilLab & @landau_lab !!! Driven by the impressive @MariaSirenko and @soobeomlee & Zhengxi Sun at @NYUGSOM_Path

[Richard Voit @RichardVoit]

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.

[Richard Voit @RichardVoit]

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

[Richard Voit @RichardVoit]

Congratulations Elvin and team. This is a really elegant way to probe an important clinical question underlying aggressive AMLs.

[Elvin Wagenblast @Elvin_Wag]

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

[Vijay Sankaran @bloodgenes]

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