Research
A collaborative project between Dr. Tyler Pierson of Cedars-Sinai Medical Center and Dr. Juan Young of the University of Miami seeks to use human cellular and mouse models of GAND to further characterize and understand this rare disorder. They also aim to eventually develop methods of screening potential therapeutic compounds that may ameliorate symptoms of GAND. Helping Hands for GAND has awarded $150,000 in research grants to support this important work. With your help, we hope to continue supporting this essential project! Drs. Young and Pierson provide more details about their studies in the videos below.
Dr. Young discusses his work with mouse models of GAND
Dr. Pierson explains his current GAND research project
A Note from Dr. Pierson
March 2020
Hello, everyone! Here is a video summary of some of the things we are studying in the lab with regards to GAND and the GATAD2B gene and protein. Our group is focusing on using Induced Pluripotent Stem Cells that have been derived from children with GAND (GAND-IPSCs) to generate brain tissues in tissue culture. We are studying five GAND-IPSC lines, and our work treats them with compounds to differentiate them into GAND-neural progenitor cells (GAND-NPCs) and then cortical neurons (GAND-Neurons). This allows us to follow these GAND-cells through the same developmental processes that occur in the brain during pregnancy to see where things may be different in children with GAND.
The first part of any big study is to determine an “assay,” which is an experiment where one type of cells or proteins act differently. Finding an assay where GAND cells are different from non-GAND cells will allow us to see if we can “fix the assay” by replacing the missing GATAD2B protein or by increasing expression of something else that can replace the function of the missing GATAD2B. Using patient-derived cells in tissue culture has allowed us to look at how fast GAND-NPCs grow and to see what types of neurons they become. Our “assays” have been getting better and better, which will allow us to start looking at compounds that might help GAND-cells behave in a more regular manner. This may help us find compounds that are already approved by the FDA to use to assist GAND children in the future.
Thank you for all of your help with our science and research!
Tyler Pierson, MD, PhD
Cedars-Sinai Medical Center
Los Angeles, CA
The first part of any big study is to determine an “assay,” which is an experiment where one type of cells or proteins act differently. Finding an assay where GAND cells are different from non-GAND cells will allow us to see if we can “fix the assay” by replacing the missing GATAD2B protein or by increasing expression of something else that can replace the function of the missing GATAD2B. Using patient-derived cells in tissue culture has allowed us to look at how fast GAND-NPCs grow and to see what types of neurons they become. Our “assays” have been getting better and better, which will allow us to start looking at compounds that might help GAND-cells behave in a more regular manner. This may help us find compounds that are already approved by the FDA to use to assist GAND children in the future.
Thank you for all of your help with our science and research!
Tyler Pierson, MD, PhD
Cedars-Sinai Medical Center
Los Angeles, CA