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

Dr. Zoghbi's amazing work has helped us understand the genes that cause serious brain disorders. These include Rett syndrome and spinocerebellar ataxia type 1. Her discoveries have also given new ideas about diseases like Parkinson's disease, Alzheimer's, and autism. This research could lead to better treatments in the future.

In 2017, she won two big 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 famous writers like William Shakespeare. She first wanted to study literature in college.

However, her mother encouraged her to study biology instead. Her mother believed that a woman in the Middle East should have a career that offered independence. Huda started studying biology at the American University of Beirut in 1973. Two years later, she entered the university's medical school.

In 1976, during her first year of medical school, the Lebanese Civil War began. Because of the war, Huda and her brother moved to Austin, Texas, to live with their sister. She couldn't return to Lebanon, so she looked for a medical school in the U.S. She was accepted at Meharry Medical College.

Huda earned her medical degree (MD) in 1979. After that, she joined Texas Children's Hospital at the Baylor College of Medicine. She started training to become a doctor who specializes in children's health.

She first thought about becoming a heart doctor for children. But during her training, a doctor named Marvin Fishman convinced her that the brain was even more interesting than the heart. So, after finishing her first training in 1982, she decided to focus on children's brain problems.

A Career in Science

Huda Zoghbi, a Kavli Prize winner, being interviewed in Oslo, Norway.

From 1982 to 1985, Dr. Zoghbi worked as a researcher focusing on children's brain health at the Baylor College of Medicine. She became a professor there in 1988. Over the years, she was promoted several times because of her important work.

Today, Dr. Zoghbi is a leading professor in genetics and neuroscience at Baylor. She is also the director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital. She is also an investigator at the Howard Hughes Medical Institute, which supports top scientists.

Discovering Genes and Brain Disorders

In 1983, Dr. Zoghbi learned about a rare disorder called Rett syndrome. She read about it in a medical journal. Soon after, she saw two young girls at Texas Children's Hospital who had the same symptoms. She realized many other cases had been misdiagnosed. This made her very interested in Rett syndrome, which was not well-known in the U.S. at the time.

Because most patients with Rett syndrome were girls and had similar symptoms, Dr. Zoghbi thought that genetics played a role. In 1985, she joined a genetics lab to learn more about genes and how they work.

Her research has focused on several important brain disorders.

Understanding Spinocerebellar Ataxia Type 1

Dr. Zoghbi began studying spinocerebellar ataxia type 1 (SCA1) in her own lab. This is a brain disorder that affects balance and coordination. She worked with another scientist, Harry Orr.

On the same day in 1993, Dr. Zoghbi and Dr. Orr both found the gene responsible for SCA1. It's called ATXN1. They discovered 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 found that the longer this repeat is, the earlier the disease symptoms appear.

Their later work showed that a protein made by this gene, called Ataxin 1, doesn't fold correctly. This misfolding causes problems in brain cells.

The Role of Math1 Gene

After her work on SCA1, Dr. Zoghbi started looking at animal genes related to balance. Her team found a gene in mice called Math1 in 1996.

They discovered that Math1 is important for many things. It helps with balance and coordination, and it's key for hearing. It also helps form special cells in the gut and controls breathing in newborns. Her lab also found that if Math1 is too active, it can lead to a type of brain tumor in children called medulloblastoma. Mice that didn't have Math1 did not develop this tumor.

Researching Rett Syndrome

Dr. Zoghbi has worked on Rett syndrome throughout her career. This was challenging because few patients were available for study. In the 1990s, she worked with Uta Francke to find the gene causing Rett syndrome.

In 1992, she narrowed down the search to a specific part of the X chromosome. Finally, in 1999, a researcher in Dr. Zoghbi's lab identified the gene: MECP2. This gene makes a protein that is vital for almost all brain cells.

They showed that Rett syndrome is an X-linked dominant disorder. This means that if just one of the two copies of the MECP2 gene is faulty, a person will develop Rett syndrome.

Dr. Zoghbi's team continues to study MECP2. In 2004, they found that having too much of the MECP2 protein in mice led to a brain disorder similar to autism. In 2009, she discovered that mice without the Mecp2 gene had lower levels of important brain chemicals like dopamine. More recently, her team showed that putting the MECP2 protein back into some brain cells could help improve some Rett syndrome symptoms.

Ataxin-1 and Alzheimer's Disease

After connecting the Ataxin-1 gene to SCA1, Dr. Zoghbi's lab explored its link to Alzheimer's disease. They found that if there isn't enough Ataxin-1, it can increase certain proteins linked to Alzheimer's. 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 in Lebanon. They both moved to the U.S. to continue their medical studies and later married in 1980. They have two children. William Zoghbi is also a successful doctor and is the chief of cardiology at Houston Methodist Hospital.