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.
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So proud of voitlab.org post-doc @GauravJoshi_PhD who presented a fantastic talk AND poster at the NAPAAC symposium at @StJude about two new DBA genes that he and @nandakkumarn have uncovered. @UTSWMedCenter @utswpediatrics
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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…
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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.
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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
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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
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❓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…
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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…
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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…
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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.
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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
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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
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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