Pediatric hematology/oncology | Stem cell biology | AML | Gene therapy | CRISPR
UT Southwestern
Joined December 2021
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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 (cell.com/cell-stem-cell/full…), we report the development of a universal gene therapy for Diamond Blackfan anemia.
1/n
Richard Voit retweeted
Inspired by Gibson cloning, we describe prime assembly, now published, for in cellulo targeted integration of gene-sized DNA fragments using prime editing. nature.com/articles/s41586-0…
Richard Voit retweeted
High-risk, high-reward research is our whole mode, including pediatric cancer, where the @DickieV Pediatric Cancer Research Fund helps us fill critical funding gaps.
Richard Voit retweeted
Read this research article by Satish Nandakumar, et al. "Germline noncoding risk variants influence clonal hematopoiesis through altered hematopoietic enhancer activity."aacrjournals.org/bloodcancer…
This article was recently presented by co-author Keisuke Ito at #ISEH2026. @neuron81
Richard Voit retweeted
We cut the ribbon & the Wahlster Lab is officially open! 🎉🔬
#NewPI #NewLab #PediatricCancer #CancerResearch #Hematology #HumanGenetics #Genomics #PhysicianScientist
🙏 to @bloodgenes @BostonChildrens @DanaFarber @BWFUND @ASH_hematology @DamonRunyon – at Boston, MA
Congrats Satish!
Excited to share our work, now online in Blood Cancer Discovery! We systematically dissected germline noncoding variants influencing clonal hematopoiesis using MPRA.
Congrats to Trieu Nguyen, @JeejanJessica and the team. Thanks to @r_tewhey and his team for the collaboration!
Gene therapy for sickle cell and thalassemia are here to stay. But what about other red cell disorders?
Led by @GauravJoshi_PhD, we explored common themes and challenges for gene therapies beyond hemoglobinopathies, now out @BrJHaem
doi.org/10.1111/bjh.70675.
Next in line: DBA!
Congrats Walter! Trainees and folks looking for a new and exciting challenge: don’t pass up this opportunity to work with Walter!
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
Richard Voit retweeted
❓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: biorxiv.org/content/10.64898…
Richard Voit retweeted
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. fda.gov/news-events/press-an…
Richard Voit retweeted
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 nejm.org/doi/full/10.1056/NE…
Richard Voit retweeted
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.
Richard Voit retweeted
MECOM promotes malignant stem cell–like states in aggressive AMLs by directly repressing prodifferentiation gene regulatory programs. Read the plenary paper in Blood: ow.ly/Nrko50XNqJk #BloodJournal #PlenaryPaper #AML
Proud of you @tjflemin for your incredibly hard work on this. Now the next frontier: inhibiting this pathway in a clinically tractable way.
Terrific to have our paper, led by @tjflemin, featured as a plenary article in @BloodPortfolio today: ashpublications.org/blood/ar…
Congratulations @Mendell_lab. What an interesting mechanism!
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. biorxiv.org/content/10.1101/…
Richard Voit retweeted
Congrats! 👏👏👏 Two CRI scientists named 2025 Pew-Stewart & Pew Scholars — @JaviGBermudez & @HijaiShin — to advance cancer, biomedical research. Read more about their #relentlessdiscovery ⬇️ cri.utsw.edu/two-childrens-r… @pewtrusts
Looking forward to the #NFRF2025 @UTSWScience in October. utsouthwestern.edu/education…. 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: voitlab.org/ #NFRF2025 #NFRF
Richard Voit retweeted
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 nature.com/articles/s43018-0…
Richard Voit retweeted
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. doi.org/10.1158/2159-8290.CD… @PeterMacRes @DanaFarber
Richard Voit retweeted
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 doi.org/10.1158/2159-8290.CD…
@StJudeResearch @DanaFarber
Richard Voit retweeted
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!
