Molecular and Neurobiological Studies in Rett Syndrome and MECP2 Disorders

Event Details

Type

Friday Lecture Series

Speaker(s)

Huda Y. Zoghbi, M.D., distinguished service professor, departments of pediatrics, molecular and human genetics, neurology and neuroscience, Baylor College of Medicine; director, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital; investigator, Howard Hughes Medical Institute

Speaker bio(s)

FLS lectures will take place in Caspary Auditorium and virtually via Zoom. We recommend virtual participants log out of VPN prior to logging in to Zoom. Please do not share the link or post on social media. This talk will be recorded for the RU community. 

Huda Zoghbi, M.D., is Distinguished Service Professor at Baylor College of Medicine, an Investigator with the Howard Hughes Medical Institute, founding Director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, and Research-in Chief, Texas Children’s Hospital.

Dr. Zoghbi graduated from the American University of Beirut (AUB) and received her medical degree from Meharry Medical College. She joined Baylor College of Medicine for her residency and subsequently obtained postdoctoral research training in molecular genetics.

Dr. Zoghbi’s expertise ranges from neurodevelopment to neurodegeneration. She and Dr. Harry Orr discovered that Spinocerebellar Ataxia type 1 is caused by expansion of a polyglutamine tract. Her subsequent studies demonstrating that CAG expansion leads to accumulation of the mutant protein in neurons has had profound ramifications since many late-onset neurological disorders involve similar accumulations of disease-driving proteins and inspired her studies in Alzheimer disease. Her work in neurodevelopment led to the discovery of the gene Math1/Atoh1 and showed that it governs the development of several components of the proprioceptive, balance, hearing, vestibular, and breathing pathways. Dr. Zoghbi’s pursuit of the genetic basis of Rett syndrome led to her discovery that mutations in MECP2 cause this postnatal neurological disorder.  Her mechanistic studies highlighted the importance of MeCP2 levels for normal brain function. She and her team provided the proof-of-concept data showing that antisense sense oligonucleotide therapy can normalize MeCP2 levels and reverse phnotypes of a MECP2 duplication mouse model.

Dr. Zoghbi has trained over 110 scientists and physician-scientists and is committeed to educating the next generation. She has been elected to the National Academy of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. Dr. Zoghbi’s honors include the Pearl Meister Greengard Prize from Rockefeller University; Breakthrough Prize in Life Sciences; the Canada Gairdner International Prize; the Lundbeck Foundation’s 2020 Brain Prize; the Kavli Prize from the Kavli Foundation, and most recently the August M. Watanabe Prize in Translational Research by Indiana University.

Open to

Tri-Institutional