A consortium of esteemed scientists are working together as one team, bringing their respective expertise, to accelerate a cure for FOXG1 syndrome.
Dr. Soo-Kyung Lee and Dr. Jae Lee
Neurodevelopmental Biologists - The Lee Lab - University at Buffalo - FOXG1 Center of Excellence
Dr. Soo-Kyung Lee is an Empire Innovation Professor of SUNY and Om P. Bahl Endowed Professor at the Department of Biological Sciences of University at Buffalo. She is an expert in gene regulation in CNS development, has contributed greatly to our understanding of gene regulatory events in neurodevelopment.
Dr. Jae Lee is a professor at the Department of Biological Sciences of University at Buffalo and has made several seminal contributions to our understanding of transcriptional regulation, including the discovery of the first member of the SET1-like epigenetic transcriptional coregulator complex family.
The Lees have a daughter named Yuna with FOXG1 syndrome, and since her diagnosis have been focusing on finding a cure for FOXG1 syndrome. To understand the etiology of FOXG1 syndrome and test various therapeutic strategies, they have partnered with the University at Buffalo to establish a FOXG1 Center of Excellence, where they have created a team of scientists solely focused on FOXG1 research and are engaged in collaborations with numerous research teams throughout the world. This team is characterizing a series of mouse models with mutant versions of FOXG1 gene from FOXG1 syndrome patients, and also striving to decode the basic biology of FOXG1 via biochemical approaches and genome-wide studies. With these efforts, Dr. Lees aim to understand the etiology of FOXG1 syndrome, and design and test novel therapeutic strategies. Click here and here to read press on the Lees and here for a recent academic publication.
Lee Lab University at Buffalo FOXG1 Center of Excellence Team Members:
Dr Priya Banerjee focuses on biological physics
Dr Michael Yu, focuses on Protein Arginine Methylation
Dr Stewart Clark focuses on behavioral phenotyping .
Dr Yungki Park focuses on transcriptional regulatory network
Dr David Poulsen focuses on EEG analyses on FOXG1 mutant mice
Seven postdoctoral graduates and seven postdoctoral students are exclusively devoted to FOXG1 Research
Dr. Alysson Muotri
University of California San Diego, San Diego, USA
Dr. Alysson R. Muotri, is a professor in the Departments of Pediatrics and Cellular & Molecular Medicine, and the Director of the Stem Cell Program at the University of California, San Diego. His lab is studying the brain from a novel evolutionary and developmental perspective, differentiating stem cells to recreate "brain organoids" in the controlled setting of a lab. These brain organoids capture the genetic information from the donor, mimicking their function. They work as brain avatars for patients, allowing the scientists to test different personalized treatments before moving into clinics. Muotri is focused on disease modeling and characterization of FOXG1 iPS lines and brain organoids. Further, his lab will test a variety of gene therapies, antisense therapies and small molecule therapies on these lines and will be a key partner to understand which treatments rescue symptoms.
Dr. Kathrin Meyer
Nationwide Children‘s Hospital, Columbus Ohio
Dr Kathrin Meyer is a Principal Investigator at Nationwide Children’s Hospital and Assistant Professor in the Department of Pediatrics at The Ohio State University. Meyer trained in Brian Kaspars laboratory at the Center for Gene Therapy in Columbus Ohio. During that time, Meyer established a new and fast reprogramming method for in vitro modeling of neurodegenerative diseases using patient skin cells. Moreover, she developed intrathecal gene therapy programs for several neurodegenerative diseases including Spinal Muscular Atrophy and Batten Disease. Multiple clinical trials that are based on this work are currently ongoing at Nationwide Children's Hospital. Meyer’s lab has a strong translational focus in her projects with the goal to move additional programs towards clinical trials. These projects include the optimization of delivery strategies and evaluation of efficacy and targeting of various areas of the nervous system with gene therapy.
Dr. Corinne Houart
Kings College, London, United Kingdom
Dr Corinne Houart is the Deputy Director of the Centre for Developmental Neurobiology. Houart's group are global experts in the molecular and cellular mechanisms that drive zebrafish forebrain development and the mechanisms underlying vertebrate brain regionalisation. They have successfully embarked in identifying similarities and divergences between zebrafish and mouse early forebrain regionalization. In the last 5 years, Houart has pioneered the use of genome editing in zebrafish and understanding motor neurodegeneration. Hoart’s team is using the unique advantages of the zebrafish to 1) Screen FDA approved drug libraries for compounds able to restore neuronal functions affected during development in the heterozygous FOXG1 mutant, and 2) Measure how many pathological aspects of the syndrome an be corrected by the discovered drug candidates at stages equivalent to human infants. Here’s a recent academic publication by Houart on FOXG1 and here is a podcast by lab member Dr Hannah Bruce.
Dr. Antonello Mallamaci
SISSA, Trieste, Italy
Dr Antonello Mallamaci is Head of the Laboratory of Cerebral Cortex Development. Mallamaci has contributed to reconstruction of molecular mechanisms controlling early cortical specification of the dorsal telencephalon, regionalisation of the cortico-cerebral field and appropriate sizing of distinctive areas arising from it, paying special attention to a small set of evolutionarily conserved transcription factors genes and non-coding RNAs implicated in these processes. More recently, he moved to molecular control of cortico-cerebral astrogenesis and neuronal morphogenesis, and developed innovative methods for RNA-therapy of neurodevelopmental haploinsufficiencies. His goals are to 1) Set up and validate an integrated and comprehensive array of histogentic assays for systematic and fast, quantitative multidimensional interrogation of neuropathogenic FOXG1 mutations in the contest of key developmental processes mastered by this gene, modelled within rodent neural cultures, and 2) Verify if accurate correction and/or compensation of abnormal FOXG1 activity levels associated to specific FOXG1 mutations, by small therapeutic RNAs, can rescue the corresponding neuro-developmental errors. Click here and here to read recent press on Mallamaci’s work on FOXG1. Here’s a recent academic publication.
Dr. Goichi Miyoshi
Gunma University Graduate School of Medicine, Japan
Dr. Miyoshi received B.S. and Ph.D. from Kyoto University and carried on his postdoctoral training at New York University from 2004 to 2015. After taking Assistant Professor position at Tokyo Women’s Medical University, was appointed as full Professor at Gunma University Graduate School of Medicine. Throughout his career in research, he focused on the development of GABAergic neuronal systems by utilizing novel genetic strategies in mice. Currently, he addresses the inhibitory circuit development in neurodevelopmental disorders including the ASD FOXG1 syndrome.
Dr Christopher Hart
Creyon Bio, San Diego, USA
Creyon Bio is focused on making new gene-centric medicines affordable and rapidly available to patients globally. Creyon Bio is adopting advances in computational chemistry, biophysics and ML/AI to build a scalable platform that will transform the way oligonucleotide-based medicines (OBMs) are created. The Creyon team brings decades of experience in quantitative biology, genomics and OBM development and is forging a strong collaboration with the FRF Scientific Team, including Dr. Alysson Muotri, Dr. Soo-Kyung Lee, Hebbian Lab, and others to develop and test a series of OBMs on FOXG1 models.
FOXG1 In-House Cellular Lab - Hebbian Bio
New York, New York
Dr. Hourinaz Behesti is Founder and CEO of our FOXG1 in-house accelerator lab, Hebbian Bio, a patient-informed therapeutic target discovery hub for autism & related conditions. Behesti is a leading multidisciplinary expert in brain development, uniquely bridging three distinct scientific fields (relevant to the planned work), combined with a patient-engaged approach
Dr. Chris Ahern
University of Iowa, Iowa City, USA
The Ahern lab works at the interface of membrane biophysics and chemical biology. Dr Ahern’s particular expertise is in the synthesis and expression of tailor made non-natural amino acids and the application of chemical biological approaches to the study of membrane proteins. They have had success in expressing synthetic amino acids in many channel and receptor types for pharmacological studies of ligand and drug interactions as well as structure function relationships. They are currently developing synthetic amino acid spin labels and fluorophores for use in cellular and cell-free expression systems. Click here to read about Dr Ahern’s talk at our FOXG1 Science Symposium.
Dr. Jeanne Paz
Gladstone Institute, of Neurological Disease, San Francisco California
Dr. Jeanne Paz at Gladstone is conducting a detailed characterization of seizure types across multiple mouse models of FOXG1 syndrome, and identifying key brain regions involved in seizures. The innovation in this approach uses a sophisticated in vivo electrophysiology approach to interrogate which brain regions are involved in seizure expression in multiple established FOXG1 mouse models.
Dr. David Bedwell
University of Alabama at Birmingham, Birmingham, USA
Dr David Bedwell is Chair and Co-Director of UAB’s Structural Biology Program, and Associate Director of the Gregory Fleming James CF Research Center. His lab seeks to understand the mechanistic details of translation termination in eukaryotes, and to use that knowledge to develop therapeutic strategies for a range of genetic diseases caused by premature translation termination mutations (PTCs). In addition, other important cellular processes also intersect with the process of translation termination. For example, conserved cellular machineries also regulate the abundance of mRNAs based on the location of stop codons through the process of Nonsense-Mediated mRNA Decay (NMD). They are using a combination of genetics, biochemistry, and cell biology to better understand the molecular details of these processes. Bedwell’s goals for FOXG1 is to identify compounds capable of suppressing FOXG1 nonsense mutations and restoring full-length, functional FOXG1 protein. He hypothesizes that PTC suppression compounds can be identified that restore enough full-length FOXG1 protein to improve many syndrome phenotypes.