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Pediatrics - Neurology/

Houston, Texas

Pediatrics - Neurology and Developmental Neuroscience
Pediatrics - Neurology/
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Huda Y. Zoghbi, M.D.

Huda Y. Zoghbi, M.D.Professor
Director - Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital
Marvin Fishman Chair in Pediatric Neurology Research
Investigator - Howard Hughes Medical Institute
Member, Institute of Medicine and National Academy of Sciences

Contact Information:
Huda Y. Zoghbi, M.D.
Jan and Dan Duncan Neurology Research Institute
at Texas Children's Hospital
1250 Moursund St., Suite 1350
Houston, TX 77030
Tel: 713-798-6558
Fax: 713-798-8728


American University of Beirut/Meharry Medical College - M.D.
Beirut Medical School, Lebanon - B.S. & Med. I
Baylor College of Medicine - Postdoctoral Training

Current Positions in Professional Organizations

  • Member - Alpha Omega Alpha
  • Member - American Academy of Neurology
  • Member - American Neurological Association
  • Member - American Society of Human Genetics
  • Member - Child Neurology Society
  • Member - The Society for Pediatric Research
  • Member - Society of Neuroscience

Research Interests

  • Genetic and cell biological approaches to explore the pathogenesis of Rett syndrome and autistic spectrum disorders and polyglutamine neurodegenerative diseases, and to study genes essential for normal neurodevelopment.

Current Research & Funding

  • Molecular Studies in Spinocerebellar Ataxia Type 1. H. Zoghbi, Principal investigator. Sponsored by NIH/NINDS. The goal of this study is to advance our understanding of the mechanism(s) mediating neuronal degeneration in spinocerebellar ataxia type 1 (SCA1) and to translate basic research findings into preclinical trials in SCA1 mouse models.
  • Molecular Pathogenesis of Rett syndrome. Sponsored by NIH/NICHHD. The overarching goal is to gain insight into the molecular pathogenesis of Rett syndrome. Specifically, we are interested in understanding how dysfunction of MeCP2 causes a broad spectrum of neuropsychiatric phenotypes seen in Rett syndrome and related disorders. Towards this goal we are studying the consequences of loss of MeCP2 in specific neurons, and are pursuing the identification of genes that are misregulated when Mecp2 levels/activities are either decreased or increases.
  • Genetic Approaches to Study Neuronal Function and Dysfunction. H. Zoghbi, Principal investigator. Sponsored by the Howard Hughes Medical Institute. Goals are to study (1) pathogenesis of SCA7 and SCA6 polyglutamine disorders through the generation and characterization of mouse models for SCA6 and SCA7 and the study of the mechanism of neurodegeneration; (2) functional analysis of genes essential for nervous system development using a cross species approach. We are studying the function of mouse atonal homologue (Math1) in neurodevelopment and gut development. So far when have shown that it is essential for the genesis of several groups of neurons in the proprioceptive and auditory pathways, inner hair cells, and secretory cells in the gut. We are now studying the role of Math1 in brainstem development and in CNS control of breathing and pursuing its transcriptional targets.
  • Mental Retardation Research Center. H. Zoghbi, Principal investigator. Sponsored by NIH/NICHHD. Goals are to advance understanding of molecular basis and pathogenesis of several human diseases that cause developmental disabilities and/or mental retardation; identify the basis of several MRDD disorders; improve diagnosis; develop an animal model for these human disorders; identify the genes that are essential for normal development; and make new advances in gene therapy research.

Recent Publications

  • Rose MF, Ahmad KA, Thaller C, Zoghbi HY. Excitatory neurons of the proprioceptive, interoceptive, and arousal hindbrain networks share a developmental requirement for Math1. Proc Natl Acad Sci U S A. 2009; 106(52):22462-7.
  • Ramocki MB, Peters SU, Tavyev YJ, Zhang F, Carvalho CM, Schaaf CP, Richman R, Fang P, Glaze DG, Lupski JR, Zoghbi HY. Autism and other neuropsychiatric symptoms are prevalent in individuals with MeCP2 duplication syndrome. Ann Neurol. 2009; 66(6):771-82.
  • Samaco RC, Mandel-Brehm C, Chao HT, Ward CS, Fyffe-Maricich SL, Ren J, Hyland K, Thaller C, Maricich SM, Humphreys P, Greer JJ, Percy A, Glaze DG, Zoghbi HY, Neul JL. Loss of MeCP2 in aminergic neurons causes cell-autonomous defects in neurotransmitter synthesis and specific behavioral abnormalities. Proc Natl Acad Sci U S A. 2009; 106(51):21966-71.
  • Flora A, Klisch TJ, Schuster G, Zoghbi HY. Deletion of Atoh1 disrupts Sonic Hedgehog signaling in the developing cerebellum and prevents medulloblastoma. Science. 2009; 326(5958):1424-7.
  • Rose MF, Ren J, Ahmad KA, Chao HT, Klisch TJ, Flora A, Greer JJ, Zoghbi HY. Math1 is essential for the development of hindbrain neurons critical for perinatal breathing. Neuron. 2009; 64(3):341-54.
  • Maricich SM, Xia A, Mathes EL, Wang VY, Oghalai JS, Fritzsch B, Zoghbi HY. Atoh1-lineal neurons are required for hearing and for the survival of neurons in the spiral ganglion and brainstem accessory auditory nuclei. J Neurosci. 2009; 29(36):11123-33.
  • Maricich SM, Wellnitz SA, Nelson AM, Lesniak DR, Gerling GJ, Lumpkin EA, Zoghbi HY. Merkel cells are essential for light-touch responses. Science. 2009; 324(5934):1580-2.
  • Jorgensen ND, Andresen JM, Lagalwar S, Armstrong B, Stevens S, Byam CE, Duvick LA, Lai S, Jafar-Nejad P, Zoghbi HY, Clark HB, Orr HT. Phosphorylation of ATXN1 at Ser776 in the cerebellum. J Neurochem. 2009; 110(2):675-86.
  • Ben-Shachar S, Chahrour M, Thaller C, Shaw CA, Zoghbi HY. Mouse models of MeCP2 disorders share gene expression changes in the cerebellum and hypothalamus. Hum Mol Genet. 2009; 18(13):2431-42.
  • Carvalho CM, Zhang F, Liu P, Patel A, Sahoo T, Bacino CA, Shaw C, Peacock S, Pursley A, Tavyev YJ, Ramocki MB, Nawara M, Obersztyn E, Vianna-Morgante AM, Stankiewicz P, Zoghbi HY, Cheung SW, Lupski JR. Complex rearrangements in patients with duplications of MECP2 can occur by fork stalling and template switching. Hum Mol Genet. 2009; 18(12):2188-203.
  • Chao HT, Zoghbi HY. The yin and yang of MeCP2 phosphorylation. Proc Natl Acad Sci U S A. 2009; 106(12):4577-8.
  • Zoghbi HY. Rett syndrome: what do we know for sure? Nat Neurosci. 2009; 12(3):239-40.
  • Miesegaes GR, Klisch TJ, Thaller C, Ahmad KA, Atkinson RC, Zoghbi HY. Identification and subclassification of new Atoh1 derived cell populations during mouse spinal cord development. Dev Biol. 2009; 327(2):339-51.
  • Carlson KM, Melcher L, Lai S, Zoghbi HY, Clark HB, Orr HT. Characterization of the zebrafish atxn1/axh gene family. J Neurogenet. 2009; 23(3):313-23.
  • Zoghbi HY, Orr HT. Pathogenic mechanisms of a polyglutamine-mediated neurodegenerative disease, spinocerebellar ataxia type 1. J Biol Chem. 2009; 284(12):7425-9.

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