GGC’s Flanagan-Steet Awarded $100,000 Grant from National MPS Society

07.29.21
Flanagan-Steet headshot

GREENWOOD, SC  – The National MPS Society has awarded a $100,000 grant to Heather Flanagan-Steet, PhD, Director of Functional Studies at the Greenwood Genetic Center (GGC). Flanagan-Steet will lead the two-year project designed to better understand the biology of skeletal disorders in patients with these rare disorders and identify new therapies.

Mucopolysaccharidoses (MPS), as a group, affect approximately 1 in 25,000 individuals. These progressive diseases cause widespread damage throughout the body, including the heart, bones, joints, respiratory system and central nervous system, leading to a shortened lifespan. Skeletal abnormalities are a common feature of MPS disorders and can lead to pain, impaired growth, and limited mobility.

Flanagan-Steet’s work will involve the use of zebrafish models of three MPS disorders, MPS II (Hunter syndrome), MPS IVA (Morquio syndrome), and ML II (I-cell disease), to pinpoint common pathways involved in the bone and cartilage disease seen in patients with MPS.

“Because zebrafish and humans share 70% of the same DNA, we can mimic MPS in the zebrafish, allowing us to study how the patient mutations actually cause the clinical features of these disorders,” said Flanagan-Steet. “The fish provide a really powerful tool both to help us understand exactly how these skeletal issues develop and then to assess which treatments may improve or prevent those issues. By directly comparing the mechanisms in these three different disease models, we hope to identify therapeutic approaches that may benefit multiple diseases.”

The MPS Society provides support resources for families as well as funding for research into this group of disorders which can affect the health, development, quality of life, and lifespan of affected individuals. GGC has a long-standing interest in MPS disorders including providing clinical care, diagnostic testing, and research for many of these rare conditions.

“We are so grateful for the support of the MPS Society, an organization led by families who are dedicated to raising awareness, supporting research, and ultimately finding cures for these disorders,” said Flanagan-Steet. “We are excited to advance this work and contribute to improving the quality of life for all individuals and families impacted by MPS disorders.”

About the MPS Society

The National MPS Society exists to cure, support, and advocate for MPS and ML. Their mission serves individuals, families, and friends affected by Mucopolysaccharidoses and Mucolipidosis through supporting research, supporting families, and increasing public and professional awareness. For more information on MPS and ML, please visit www.mpssociety.org.

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Makayla was diagnosed with Rett Syndrome in April of 2015 at the age of two. At about 18 months, we noticed she had started some repetitive hand motions, and her pediatrician was a little concerned that she did not have many words. She had learned some sign language, and was saying ‘mama’ and ‘dada’, but that was it. Then she suddenly stopped all of that. At her 2 year old check-up, the pediatrician referred us to BabyNet, SC’s early interve...

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