BCM Department of Molecular and Human Genetics

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...

Join us Wed Feb. 18 @ 12 p.m. CST for the next Genetics Education Webinar: Updates in Angelman Syndrome: From Testing to Treatment w/ Dr. Carlos Bacino of @bcmgenetics.bsky.social & Laura Buckner, M.Ed., LPC, Program Manager, Texas Center for Disability Studies at UT Austin Register: bit.ly/4rk4bH2

Congratulations to our 2025 @bcmgenetics.bsky.social Clinical Faculty Award Winner for Excellent Research Mentor, Dr. Fernando Scaglia!

Genetic Duo: ATP13A2 and GBA1 Interactions Fuel Neurodegeneration Parkinson’s disease (PD) ranks as the second most prevalent neurodegenerative disorder following Alzheimer’s disease, impacting more than 10 million individuals globally. The condition manifests thr

Genetic Duo: #ATP13A2 and #GBA1 Interactions Fuel #Neurodegeneration. M. Gu and H Bellen explain their findings. @bcmhouston.bsky.social #TexasChildrens
@molneurodegen.bsky.social @bcmgenetics.bsky.social #BCMPostDoc bioengineer.org/genetic-duo-...

Dr. Bo Yuan, @bcmhouston.bsky.social @bcmhgsc.bsky.social @bcmgenetics.bsky.social, received the 2026 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award from the ACMG Foundation for the discovery and characterization of multiple novel genetic disorders. tinyurl.com/yh7fyefs

‘Sticky coat’ boosts triple negative #breastCancer’s ability to metastasize www.eurekalert.org/news-release... #CChengLab, G Bobkov, K Patel et al @natcomms.nature.com @bcmhouston.bsky.social @bcmgenetics.bsky.social #BCMCancerCenter #BCMpostdoc #Cellclusters

Researchers #LabShulman et al uncovered how #SPTSSB gene variant triggers a process that can lead to #ParkinsonsDisease. #lipids @bcmhouston.bsky.social #TexasChildrens #BrainJournal @oxunipress.bsky.social @bcmgenetics.bsky.social @bcmneurosci.bsky.social medicalxpress.com/news/2026-02...

O’Donnell Awardee in Medicine, Dr. Pengfei Liu with family, Dr. Arthur Beaudet (right) & Dr. Brendan Lee (left) at the 20th Anniversary Edith and Peter O’Donnell Awards Dinner & Ceremony, held on Feb. 3, 2026 in San Antonio, Texas during the @TAMESTX 2026 Annual Conference.

It takes two: Genes ATP13A2 and GBA1 interact to drive neurodegeneration Parkinson's disease (PD) is the second most common neurodegenerative disease after Alzheimer's disease, affecting more than 10 million people worldwide. People with this condition may experience tremo...

How do two altered genes, #ATP13A2 and #GBA1, in two different cell types lead to neurodegeneration? M. Gu and H Bellen explain their findings. @bcmhouston.bsky.social #TexasChildrens @molneurodegen.bsky.social @bcmgenetics.bsky.social #BCMPostDoc medicalxpress.com/news/2026-02...