Dr. Huda Y. Zoghbi, professor and Howard Hughes Medical Institute Investigator at Baylor, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, has been elected as one of the newest members of the American Academy of Arts and Sciences, one of the nation’s most prestigious honorary titles.
The Academy is one of the country’s oldest societies and independent policy research centers. It recognizes exceptional scholars, leaders, artists and innovators and engages them in sharing knowledge and addressing challenges facing the world. This year, Zoghbi joins more than 200 other individuals from a wide range of disciplines and professions as elected members of the Class of 2018.
“It is very fitting that the Academy recognizes Huda Zoghbi, without a doubt one of the most influential faculty members at Baylor College of Medicine,” said Dr. Paul Klotman, president, CEO and executive dean of the College. “Through science and advocacy, she has brought attention to important issues facing our world.”
Zoghbi, who is the director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and professor of pediatrics, molecular and human genetics, neurology and neuroscience at Baylor, is the world’s leading expert on Rett syndrome. The disease strikes after about a year of normal development and presents with developmental regression, social withdrawal, loss of hand use and compulsive hand wringing, seizures and a variety of neurobehavioral symptoms.
After encountering girls with Rett syndrome, Zoghbi set out to find the genetic cause of the disease. She and her research team identified mutations in MECP2 as the cause and revealed the importance of MeCP2 for the function of various neuronal subtypes. Her work in mouse models showed just how sensitive the brain is to the levels of MeCP2. Too little MeCP2 causes Rett syndrome; doubling MeCP2 levels causes progressive neurological deficits. The latter disorder is now recognized as MECP2 duplication syndrome.
The discovery of the Rett syndrome gene provided a straightforward diagnostic genetic test, allowing early and accurate diagnosis. It also revealed that mutations in MECP2 can cause a host of other neuropsychiatric features ranging from autism to juvenile onset schizophrenia. Further, it provided evidence that an autism spectrum disorder or an intellectual disability disorder can be genetic even if it is not inherited.
Her discovery opened up a new area of research on the role of epigenetics in neuropsychiatric disorders. Her more recent work has shown that symptoms of adult mice modeling the duplication disorder can be reversed using antisense oligonucleotides that normalize MeCP2 levels. This discovery provides a potential therapeutic strategy for the MECP2 duplication syndrome and inspires similar studies for other duplication disorders.
Zoghbi and collaborators also have made many discoveries toward understanding mechanisms driving adult-onset neurodegenerative disorders and are now focused on identifying potential therapeutics for these disorders.
Zoghbi earned a B.S. from the American University of Beirut, where she also started medical school, later moving to Meharry Medical College in Nashville, Tenn., to complete her M.D. She then joined Baylor College of Medicine where she trained in pediatrics, neurology and molecular genetics.
“Membership in the Academy is not only an honor, but also an opportunity and a responsibility,” said Jonathan Fanton, president of the American Academy. “Members can be inspired and engaged by connecting with one another and through Academy projects dedicated to the common good. The intellect, creativity and commitment of the 2018 Class will enrich the work of the Academy and the world in which we live.”
The Class of 2018 members were elected in 25 categories and are affiliated with 125 institutions from across the globe. They include scientists, scholars, an academy award winner, philanthropists, CEOs, historians, a past U.S. president and a current Supreme Court judge.
Baylor College of Medicine faculty who currently are American Academy of Arts and Sciences members include Drs. James Lupski, professor of pediatrics and the Cullen Foundation Endowed Chair in Molecular Genetics; Bert W. O’Malley, chair and professor of molecular and cellular biology and the Thomas C. Thompson Chair in Cell Biology; JoAnne Richards, professor of molecular and cellular biology and member of the Dan L Duncan Comprehensive Cancer Center; and Dora Angelaki, professor of neuroscience and the Wilhelmina Robertson Chair in neuroscience.
This class of 2018 is a testament to the Academy’s ability to both uphold our 238-year commitment to honor exceptional individuals and to recognize new expertise,” said Nancy C. Andrews, the chair of the board of the American Academy. “John Adams, James Bowdoin and other founders did not imagine climatology, econometrics, gene regulation, nanostructures or Netflix. They did, however, have a vision that the Academy would be dedicated to new knowledge – and these new members help us achieve that goal.”
The new class will be inducted at a ceremony in October 2018 in Cambridge, Mass., at which the newly elected members will sign the Book of Members, and their signatures will be added to the Academy members who came before them, including Benjamin Franklin (1781) and Alexander Hamilton (elected 1791) in the 18th century; Ralph Waldo Emerson, (1864), Maria Mitchell (1848) and Charles Darwin (1874) in the 19th; and Albert Einstein (1924), Robert Frost (1931), Margaret Mead (1948), Milton Friedman (1959) and Martin Luther King, Jr. (1966) in the 20th.