Huda Zoghbi facts for kids

Quick facts for kids Huda Yahya Zoghbi
Born	Huda El-Hibri (1954-06-20) 20 June 1954 (age 71) Beirut, Lebanon
Alma mater	Baylor College of Medicine Meharry Medical College American University of Beirut
Known for	Research in Rett syndrome and spinocerebellar ataxia type 1
Spouse(s)	William Zoghbi
Awards	Texas Women Hall of Fame (2008) Gruber Prize in Neuroscience (2011) Pearl Meister Greengard Prize (2013) Dickson Prize (2013) Shaw Prize in Life Science and Medicine (2016) Canada Gairdner International Award (2017) Breakthrough Prize in Life Sciences (2017) Brain Prize (2020)
Scientific career
Fields	Genetics Neuroscience
Institutions	Regeneron Pharmaceuticals Baylor College of Medicine Howard Hughes Medical Institute Texas Children's Hospital

Huda Yahya Zoghbi is a famous scientist born in Beirut, Lebanon. She is also an American citizen. She is a professor at Baylor College of Medicine, where she studies genetics and the brain. She leads the Jan and Dan Duncan Neurological Research Institute.

From 2018 to 2024, she was the main editor for a science journal called Annual Review of Neuroscience.

Dr. Zoghbi's research has helped us understand the genes that cause serious brain disorders like Rett syndrome and spinocerebellar ataxia type 1. Her discoveries have also changed how scientists think about other brain conditions. These include Parkinson's disease, Alzheimer's, autism, and intellectual disability. Her work could lead to new and better treatments in the future.

In 2017, she received two major awards: the Canada Gairdner International Award and the Breakthrough Prize in Life Sciences.

Early Life and Education

Huda Zoghbi was born in Beirut, Lebanon, on June 20, 1954. She grew up there and loved reading books by authors like William Shakespeare and Jane Austen. She first wanted to study literature at university.

However, her mother encouraged her to study biology instead. Her mother believed that a woman in the Middle East should choose a career that offered independence and security. She told Huda that she could always write in her free time.

Starting Medical School

In 1973, Zoghbi started studying biological sciences at the American University of Beirut (AUB). Two years later, she entered the university's medical school.

The Lebanese Civil War began in 1976, during her first year of medical school. Even though she and her classmates wanted to stay, her parents sent her and her brother to Austin, Texas. This happened after her brother was hurt by shrapnel. They planned to return the next summer.

Moving to the United States

The war continued, making it impossible for Zoghbi to go back to Lebanon. She thought American medical schools started in October, like Lebanese ones. But by October, she learned that US schools had already begun their fall term two months earlier.

Family friends in America suggested she apply to Vanderbilt University. Vanderbilt did not accept transfer students, but they recommended Meharry Medical College. Meharry accepted her right away. Even though she still hoped to return to Lebanon, her professors at AUB advised her to stay. She earned her MD degree from Meharry in 1979.

After graduating, she joined Texas Children's Hospital at the Baylor College of Medicine. She began her training there as a pediatric resident, which means a doctor who specializes in children's health.

Discovering a Passion for the Brain

Zoghbi first planned to specialize in pediatric cardiology, which is heart health for children. But during her training in pediatric neurology, the head of that department, Marvin Fishman, convinced her. He told her that the brain was much more interesting than the heart.

After finishing her residency in 1982, she began a three-year period as a postdoctoral researcher in pediatric neurology. This meant she continued her training and research in brain disorders affecting children.

Career Highlights

Huda Zoghbi, Kavli Prize Laureate, interviews at Munch Museum, Oslo, Norway. Photo: Thomas Eckhoff

From 1982 to 1985, Dr. Zoghbi worked as a postdoctoral researcher in pediatric neurology at the Baylor College of Medicine. She became an assistant professor in the Department of Pediatrics at Baylor in 1988. She was then promoted to associate professor in 1991 and full professor in 1994.

Today, Dr. Zoghbi is a professor in several departments at Baylor. These include Molecular and Human Genetics, Neuroscience, and Pediatrics Section of Neurology and Developmental Neuroscience. She holds the Ralph Feigin, M.D. Endowed Chair. She is also the director of the Texas Children's Hospital Jan and Dan Duncan Neurological Research Institute.

She is also a member of the Dan L. Duncan Comprehensive Cancer Center at Baylor. Additionally, she is an investigator at the Howard Hughes Medical Institute. She serves on the board of directors for Regeneron Pharmaceuticals, a biotechnology company.

Important Research

In 1983, Dr. Zoghbi learned about Rett syndrome from a medical article. This article helped her diagnose a five-year-old girl she was treating at Texas Children's Hospital. A week later, she saw another patient with the same symptoms. When she looked at older medical records, she found more cases of Rett syndrome that had been misdiagnosed.

This sparked her interest in Rett syndrome. At that time, no one in the US had reported much about this disease. An article she published in 1985 brought many Rett syndrome patients to Texas Children's Hospital. This gave her a chance to study many cases.

Since most Rett syndrome patients were girls, and their symptoms were very similar, Zoghbi believed genetics played a role. In 1985, she joined Dr. Arthur Beaudet's group to learn about genetics and molecular biology. Beaudet suggested she study spinocerebellar ataxia type 1 (SCA1) instead of Rett syndrome. SCA1 was a more straightforward genetic disorder to study at the time.

In 1988, Zoghbi left Beaudet's group and started her own lab at Baylor. Besides her work on SCA1 and Rett syndrome, she is also part of a research project on CDKL5 Deficiency Disorder. This project is funded by the Loulou Foundation, Baylor College of Medicine, and the Jan and Dan Duncan Neurological Research Institute (NRI).

Spinocerebellar Ataxia Type 1 (SCA1)

After starting her own lab, Dr. Zoghbi continued to study spinocerebellar ataxia type 1 (SCA1). She worked with Harry Orr from the University of Minnesota. On the same day, April 8, 1993, both Zoghbi and Orr identified the ATXN1 gene as the cause of SCA1.

They found that the disease is caused by a mistake in this gene. A small part of the gene, called a CAG repeat, is too long. They also learned that the younger a person was when symptoms started, the longer this CAG repeat was. Further research by their teams showed that the protein made by this gene, called Ataxin 1, folds incorrectly. This misfolding plays a role in the disorder.

Math1 Gene Research

After figuring out the cause of spinocerebellar ataxia type 1, Dr. Zoghbi began studying animal genes related to balance. Dr. Hugo J. Bellen at Baylor described how the atonal gene helps with balance in fruit flies. Zoghbi decided to study a similar gene in mammals.

In 1996, a member of her lab successfully copied the mouse version of this gene, called Math1. Her team then discovered that Math1 is important for more than just balance. It is also crucial for hearing, forming special cells in the gut, and controlling breathing in newborns. It also helps regulate certain brain cells in adults.

Her lab also found that if Math1 is too active, it can lead to medulloblastoma. This is a common brain tumor in children. They showed that mice without the Math1 gene did not develop this tumor.

Understanding Rett Syndrome

From early in her career, Dr. Zoghbi has worked on Rett syndrome. She continued this research even when other scientists and funding groups were not very interested. This was mainly because there were very few patients and families available for study.

In the 1990s, she worked with Uta Francke from Stanford University to find the gene responsible for Rett syndrome. In 1992, she narrowed down the search to a specific part of the X chromosome. In 1999, a postdoctoral researcher in Zoghbi's lab identified MECP2 as the gene causing the disorder.

The MECP2 protein attaches to certain parts of DNA. It is essential for almost all brain cells. In their research paper, Dr. Zoghbi and her team showed that Rett syndrome is an X-linked dominant disorder. This means that if one of the two copies of the MECP2 gene is abnormal, Rett syndrome will occur.

Dr. Zoghbi's team continues to study MECP2. In 2004, they found that having too much of this protein in mice led to a brain disorder similar to autism. In 2009, she discovered that mice lacking the Mecp2 gene had lower levels of certain brain chemicals like norepinephrine, dopamine, and serotonin. This matched what she had seen in Rett syndrome patients back in 1985.

More recently, Zoghbi confirmed that the MECP2 protein also attaches to other parts of DNA, not just the ones previously known. She also found that putting the MECP2 protein back into some neurons (brain cells) was enough to help with some of the symptoms of Rett syndrome.

Ataxin-1 and Alzheimer's Disease

After connecting the gene Ataxin-1 to SCA1, Dr. Zoghbi's lab was asked to investigate a possible link between Ataxin-1 and Alzheimer's disease. This request came from Dr. Jaehong Suh at the Massachusetts General Hospital's MassGeneral Institute for Neurodegenerative Disease.

Their study found that if there isn't enough Ataxin-1, it can increase the levels of a protein called BACE1. This can also lead to problems with another protein called Aβ in mouse models. This suggests that Ataxin-1 might play a role in the risk and development of Alzheimer's disease.

Awards and Honors

Personal Life

Huda Zoghbi met her husband, William Zoghbi, when they were both medical students. They studied at the American University of Beirut. In 1977, Huda continued her medical studies at Meharry Medical College. William transferred to Meharry the next year.

After they both graduated, they completed their medical residencies at the Baylor College of Medicine. They got married in 1980 and have two children. William Zoghbi is now the chief of the Department of Cardiology (heart health) at Houston Methodist Hospital.