Congratulations to the 2026 Arthur L. Beaudet Prize for Scholarship in Genetics winners: Rhys Duarte and David Curtis! The prize recognizes excellence in scholarly achievement of individuals in the Genetics & Genomics Medical Student Pathway.

News News about the National Urea Cycle Disorders Foundation

Read the story of a family's fight for a #rareDisease treatment and the role Baylor College of Medicine researchers played setting the foundation for developing the novel therapy. @bcmhouston.bsky.social @bcmgenetics.bsky.social nucdf.org/news.html/ar...

Baylor College of Medicine researchers set the foundation for novel rare disease treatment The story behind the discovery that led to the novel treatment revealed that scientific breakthroughs can sometimes happen in unexpected ways.

B. Lee, L. Burrage et al set the scientific foundation for LOARGYS, now FDA approved to treat #arginaseDeficiency, a condition in which muscles of the legs, hips and pelvis are stiff, making it difficult to walk without help. Read the story: tinyurl.com/yfsc4xfe @bcmgenetics.bsky.social

Congratulations to Dr. Lindsay Burrage and medical genetics and genomics resident, Dr. Omer Shlomovitz on their @bcmhouston.bsky.social Power of Professionalism awards! Lindsay and Omer received their awards from Dr. Stacey Rose last Friday March 20.

Join us Tues March 31 @ 7 p.m. at the Children's Museum Houston for Evenings with Genetics Seminar: "Sparking the powerhouse: understanding & treating mitochondrial disorders" w/ Drs. F. Scaglia, K. Machol & L. Emrick. Co-sponsored by @UMDF Register: bit.ly/40MKLiI

Research @bcmgenetics.bsky.social helped lay the groundwork for LOARGYS, now FDA approved for the treatment of hyperargininemia in patients ages 2+ with Arginase 1 Deficiency (ARG1‑D), based on reduction of plasma arginine. Read the original From the Labs story: blogs.bcm.edu/2024/05/30/f...

Lurie Autism Institute Announces Dr. Huda Zoghbi as the Inaugural Recipient of the Nancy Lurie Marks Prize for Autism Research | Texas Children's The Lurie Autism Institute, a joint initiative of Children’s Hospital of Philadelphia (CHOP) and Penn Medicine created to drive discovery, develop new treatments, and improve the lives of individuals ...

Congratulations to Dr. Huda Zoghbi for being named on inaugural recipient of the Nancy Lurie Marks Prize for #Autism Research! @bcmhouston.bsky.social
www.texaschildrens.org/content/news...

Join us next Thursday March 19, for a virtual town hall exploring career pathways and real-world experiences in medical genetics w/ Drs. Lee, Murray, Sutton, Lang and medical student Elyssa Smith. Time: 5:30–6:30 p.m. contact: geneticscareers@bcm.edu for more details!

A potential new approach for treating Rett syndrome This work lays the foundation and provides preclinical evidence for a therapeutic approach for Rett syndrome that increases MeCP2 and confers functional improvement.

#RettSyndrome is a neurodevelopmental condition more common in girls than boys, for which there is no treatment. But a new study by the Zoghbi lab et al shows a new path that may lead to therapies. @bcmhouston.bsky.social @bcmgenetics.bsky.social #SciTranslMed blogs.bcm.edu/2026/03/05/f...

Boosting a key brain protein could help treat Rett syndrome Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and patient-...

A new gene-targeting strategy that boosts a critical brain protein may offer a future path toward treating #RettSyndrome. From H Zoghbi lab, @harinipt6 et al @bcmhouston.bsky.social @bcmgenetics.bsky.social #ScienceTM @rettsyndrome.bsky.social www.sciencedaily.com/releases/202...

Congratulations to Dr. Gary Huang of the Dierick lab on your successful dissertation! Gary’s dissertation was “A suite of inducible tools to conditionally manipulate transgene expression in Drosophila melanogaster.” 🥳 #genetics #genomics #PhD

Join us for the next session in the Genetics Education Webinar Series "Urea Cycle Disorders: Recognition, Diagnosis, and Management" on Wednesday, March 18, 2026, at 12 p.m. CST. Lindsay Burrage, M.D., Ph.D. and Laura Buckner, M.Ed., L.P.C. Register: bit.ly/4tZf42W

A promising potential therapeutic strategy for Rett syndrome A team of researchers at Baylor College of Medicine and the Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital reports...

The H Zoghbi lab et al provide evidence for new approach to treat #RettSyndrome. @bcmhouston.bsky.social #TexasChildrens @bcmgenetics.bsky.social s.bsky.social @rsrt.bsky.social www.bcm.edu/news/a-promi...

Tomorrow is #RareDiseaseDay, we @bcmgenetics.bsky.social recognize the strength of the rare disease community. We stand with patients, families, and advocates pushing for awareness, research, and hope. www.rarediseaseday.org

Congratulations to Dr. Bing Xie of the Martin lab on your successful dissertation! Bing’s dissertation was “TEAD-independent mechanisms of YAP function in cardiomyocyte cell cycle re-entry.” 🥳 #genetics #genomics #PhD

Happy #ThanksAResidentDay! Huge thanks to our incredible genetics residents: Raiza, Yoel, Roni, Yishay, Dan, Ryan, Steven, Aaron, Alison, Eliyahu, Gregory, Simon, Vladislav, Bridget, Mona, & Christian - for your dedication, compassion, & commitment to patient care and discovery.

Researchers connect the dots between cause-effect events in Alzheimer’s disease A study published in Molecular Psychiatry reveals a path of cause-effect molecular...

Researchers look to better understand how typical #Alzheimer’s brain changes, such as the formation of amyloid plaques and tau tangles, can lead to #neurodegeneration and #cognitiveDecline. #LabShulman @bcmhouston.bsky.social @bcmgenetics.bsky.social #TexasChildrens www.bcm.edu/news/researc...

Connecting the dots between cause-effect events in Alzheimer’s disease This cross-species approach reveals that some paths seem to aggravate the disease while others may protect against it.

This study reveals that some molecular paths linked to #Alzheimers seem to aggravate the disease while others may protect against it. J Shulman et al @bcmhouston.bsky.social #TexasChildrens @bcmneurosci.bsky.social @bcmgenetics.bsky.social blogs.bcm.edu/2026/02/24/f...

Interested in metabolic ‘mimickers’ of #rheumatologicalDisease? L. Burrage, T. Vogel et al have a new review on the topic. @natrevrheum.nature.com @bcmhouston.bsky.social @bcmgenetics.bsky.social #TexasChildrens www.nature.com/articles/s41...

Breast cancer cell clusters The Image this month shows a mechanism that mediates the formation of breast cancer cell clusters and promotes cancer metastasis.

Check out #FromtheLabs' new Image of the Month! Image courtesy of the C. Cheng lab. @bcmhouston.bsky.social @bcmgenetics.bsky.social #TripleNegativeBreastCancer #Metastasis #BCMCancerCenter #BCMpostdoc blogs.bcm.edu/2026/02/26/f...

Congratulations to Dr. Megan Mair of the Botas lab on your successful dissertation! Megan’s dissertation was “Mining Centenarian Genes for Neuroprotection Against Alzheimer's Disease.” 🥳 #genetics #genomics #PhD

Excellent Prenatal Genetic Counselor: Sandra Darilek; Excellent Metabolic Dietician: Brandy Rawls-Castillo; Baylor Genetics Service Award: Xiaonan Zhao. Clinical Resident Awards: Amber Nakar Weinstein & Aaron Williams; Laboratory Fellow Award: Haowei Du; Congrats to all!

Excellent Metabolic Genetics Attending: Sanmati Rao Cuddapah; Excellent Reproductive Genetics Attending: April Adams; Excellent Genetics Educator: Lindsay Burrage; Excellent Adult Genetic Counselor: Darwin F. Arguenta; Excellent Pediatric Genetic Counselor: Emily Magness Bland;

Integrative spatial omics reveals distinct tumor-promoting multicellular niches and immunosuppressive mechanisms in Black American and White American patients with TNBC - PubMed Racial disparities in the clinical outcomes of triple-negative breast cancer (TNBC) have been well-documented, but the underlying biological mechanisms remain poorly understood. To investigate these disparities, we employed a multi-omic approach integrating imaging mass cytometry and spatial transcr …

Best Papers for Calendar Year 2025
Qian Zhu (Open) “Integrative spatial omics reveals distinct tumor-promoting multicellular niches and immunosuppressive mechanisms in Black American and White American patients with TNBC” pubmed.ncbi.nlm.nih.gov/40675986/

MeCP2 interacts with the super elongation complex to regulate transcription - PubMed Loss-of-function mutations in methyl-CpG binding protein 2 (<i>MECP2</i>) cause Rett syndrome. While we know that MeCP2 binds to methylated cytosines on DNA, the full breadth of the molecular mechanisms by which MeCP2 regulates gene expression remains incompletely understood. Here, using a genetic m …

Best Papers for Calendar Year 2025
Jun Young Sonn (Postdoc fellow) “MeCP2 interacts with the super elongation complex to regulate transcription”
pubmed.ncbi.nlm.nih.gov/41296873/

Adeno-associated Virus-mediated Silencing of Sox4 Leads to Long-Term Amelioration of Liver Phenotypes in Mouse Models of Alagille Syndrome - PubMed Preclinical studies provide proof of principle for postnatal AAV-mediated Sox4 knockdown in thyroxine-binding globulin⁺ cells as a therapeutic approach for ALGS liver disease.

Best Papers for Calendar Year 2025
Duncan Fox (Graduate Student) “Adeno-associated Virus-mediated Silencing of Sox4 Leads to Long-Term Amelioration of Liver Phenotypes in Mouse Models of Alagille Syndrome”
pubmed.ncbi.nlm.nih.gov/40446945/

Best Talks
3rd: Emily Busse “A novel mouse model of osteogenesis imperfecta type V”

More 2026 Department Retreat Awards
Best Talks
1st: Gwynna Fuller “How did mechanosensory hair cells evolve?” 2nd: Andrea Rauschmayer “The decision to fission: regulation of asexual reproduction in Schmidtea mediterranea”...

Dr. S. Yamamoto recognized with International Honor for Groundbreaking #RareDisease Discovery. @bcmhouston.bsky.social @bcmgenetics.bsky.social #TexasChildrens #NISTEP texaschildrens.org/content/news...

Partners in crime – genes ATP13A2 and GBA1 interact to drive neurodegeneration The findings reveal promising biological pathways that could be explored in future therapies.

Why some people with #geneticRisk factors develop #ParkinsonsDisease while others never do? M Gu, H Bellen et al have found an explanation. @bcmhouston.bsky.social @bcmgenetics.bsky.social #TexasChildrens @molneurodegen.bsky.social #BCMPostDoc blogs.bcm.edu/2026/02/12/f...