university at buffalo

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New publication shows groundbreaking results in FOXG1 AAV9 gene therapy studies; rescuing structural brain abnormalities

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Groundbreaking Study Shows Promise in AAV9 Gene Therapy for FOXG1 Syndrome; Rescue of Brain Structure Abnormalities and Deficits.

[Buffalo, New York June 10, 2024] – A landmark study led by Dr. Soo-Kyung Lee, Chief Scientific Officer at the FOXG1 Research Foundation and Empire Innovation Professor and Om P. Bahl Endowed Professor in the Department of Biological Sciences at University at Buffalo, and Dr. Jae Lee, Professor in the Department of Biological Sciences at University at Buffalo, in collaboration with Dr. Kathrin Meyer (responsible for the SMA gene therapy), has been published in Molecular Therapy Methods & Clinical Development.

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University at Buffalo launches center to find treatments for FOXG1 syndrome

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University at Buffalo launches center to find treatments for FOXG1 syndrome

University at Buffalo announces the launch of the FOXG1 Research Center to study FOXG1 syndrome’s impact on brain development and translate research to treatments for FOXG1 syndrome. The FOXG1 Research Center will be led by leading experts Soo-Kyung and Jae Lee, whose own daughter has FOXG1 syndrome.

“This center will make UB the home of the world’s premier research center devoted to the studies of FOXG1 syndrome, as well as provide our campus with a new neurodevelopmental biology training program and numerous research funding opportunities,” says Soo-Kyung Lee, PhD, Empire Innovation Professor and Om P. Bahl Endowed Professor in the UB Department of Biological Sciences, who will serve as the FRC’s inaugural director as well as the Chief Scientific Officer of the FOXG1 Research Foundation.

Dr. Soo-Kyung Lee Named FOXG1 Research Foundation Chief Scientific Officer

The FOXG1 Research Foundation (FRF), announced today the appointment of Dr. Soo-Kyung Lee as the new Chief Scientific Officer of the FOXG1 Research Foundation. Dr. Lee is an Empire Innovation Professor and Om P. Bahl Endowed Professor at University at Buffalo Department of Biological Sciences. Since joining the FRF in 2017, Dr. Lee has elevated the FOXG1 research center at the University at Buffalo to a global leader in the field. Her exceptional work has garnered around $3M annually from federal agencies, FRF, UB, and the Simon Foundation Autism Research Initiative (SFARI), notably receiving SFARI's 2022 Genomics of ASD: Pathways to Genetic Therapies award.

Dr. Lee’s dedication to curing FOXG1 syndrome is deeply personal as a mother to Yuna, diagnosed with FOXG1 syndrome in 2009. Her passion and commitment have inspired everyone fortunate enough to work with her. It's a rare and special circumstance to have FOXG1 parents leading the scientific journey towards effective therapeutics for all FOXG1 children globally.

For two UB scientists, love means studying their daughter's rare disease

For two UB scientists, love means studying their daughter's rare disease

University at Buffalo biologists Soo-Kyung Lee (left) and Jae Lee are researching the FOXG1 gene. Their daughter, Yuna, has a mutation in the gene, which has severely impacted her development. The Lees hope their scientific work will lead to a treatment. Credit: Douglas Levere / University at Buffalo.

For Yuna: OHSU Scientist Unveils Origins Of Daughter’s Rare Condition: Study Findings Could Lead To New Treatment Options For FOXG1 Syndrome

For more than 20 years, Soo and Jae W. Lee have studied the specialized functions of transcription factors including FOX proteins, a family of 40-plus genes integral to the lifetime development and function of such organs as the brain and heart.

Infinitesimal Odds: A Scientist Finds Her Child’s Rare Illness Stems From the Gene She Studies - NYT Video

Infinitesimal Odds: A Scientist Finds Her Child’s Rare Illness Stems From the Gene She Studies - NYT Video

By the time her mother received the doctor’s email, Yuna Lee was already 2 years old, a child with a frightening medical mystery. Plagued with body-rattling seizures and inconsolable crying, she could not speak, walk or stand.

“Why is she suffering so much?” her mother, Soo-Kyung Lee, anguished. Brain scans, genetic tests and neurological exams yielded no answers. But when an email popped up suggesting that Yuna might have a mutation on a gene called FOXG1, Soo-Kyung froze.