Grant is administered by the National Institute of Neurological Disorders and Stroke see more
The Greenwood Genetic Center’s (GGC) Rich Steet, PhD, Director of Research, and Heather Flanagan-Steet, PhD, Director of Functional Studies, have secured a renewal of their long-standing grant titled 'Pathogenic Mechanisms of Lysosomal Disease.'
The R01 grant, which is administered by the National Institute of Neurological Disorders and Stroke (NINDS), a division of the National Institutes of Health (NIH), provides $1.2 million over four years to continue the team's ongoing work to better understand the mechanisms behind lysosomal storage disorders (LSDs).
The duo has spent years studying the function of lysosomes and how their abnormal function results in the clinical features associated with LSDs. While the clinical features vary between different types of LSDs, common findings include coarse facial features, cardiac and skeletal anomalies, learning difficulties, and a shortened life span.
Prior research under this grant focused on a single disorder, mucolipidosis type II (MLII). The research team has made significant progress in understanding how MLII symptoms develop, and they have been able to successfully treat cardiac and skeletal disease manifestations in their zebrafish models. This breakthrough has fostered collaborations to advance treatment studies into a mammalian model with the ultimate goal of developing an effective therapy for patients with this rare disease.
"We are pleased that our prior work on LSDs has been so fruitful and that we are able to continue to move toward better understanding and novel treatment options for families impacted by these rare disorders," said Steet.
The primary hypothesis for this new round of funding is that the mechanisms identified in MLII are similar across other LSDs, which could lead to novel therapies for several disorders.
The grant renewal will also allow the research team to expand their work on the NUS1 gene which came to the team's attention through functional studies on a GGC patient.
Through both cellular and zebrafish experiments, researchers were able to confirm that this patient’s NUS1 variant was pathogenic, and they were also able to identify the likely mechanism and a possible therapy.
"Zebrafish that mimic this NUS1 variant not only exhibited a movement disorder as seen through abnormal swimming patterns, but they also displayed significant accumulation of cholesterol in their lysosomes," said Flanagan-Steet. "By using an FDA-approved small molecule, we were able to reduce cholesterol storage in the zebrafish and restore normal swimming behaviors."
The additional funding through the grant renewal will also help the research team to refine how the storage of cholesterol in the lysosomes occurs in patients with NUS1 mutations and why it leads to neurological symptoms and movement disorders.
"After completing 13 years of research through this grant funding, we are excited by the potential to further unravel the mysteries of lysosomal storage disorders and identify novel treatments that can help GGC patients and the thousands of others who are impacted by LSDs," said Steet. "While this renewal continues to support our ongoing basic research, it also allows us to expand our work into patient-specific projects here at GGC."
About Greenwood Genetic Center
The Greenwood Genetic Center (GGC), founded in 1974, is a nonprofit organization advancing the field of medical genetics and caring for families impacted by genetic disease and birth defects. At its home campus in Greenwood, South Carolina, a talented team of physicians and scientists provides clinical genetic services, diagnostic laboratory testing, educational programs and resources, and research in the field of medical genetics. GGC’s faculty and staff are committed to the goal of developing preventive and curative therapies for the individuals and families they serve. GGC extends its reach as a resource to all residents of South Carolina with satellite offices in Charleston, Columbia, Florence and Greenville. The GGC Foundation provides philanthropic financial support for the mission of the Center. For more information about GGC or the GGC Foundation please visit www.ggc.org.
GlycoPath joins other life science organizations in SCRA portfolio of companies see more
All SCRA member companies receive coaching, access to experts in SCRA’s Resource Partner Network, eligibility to apply for grant funding, and the potential to be considered for an investment from SCRA’s investment affiliate, SC Launch Inc.
Glycopath Inc. received a $50,000 federal matching grant, the release said. The Charleston-based startup is working to improve biomarker research by streamlining methods with proven clinical impact and simplifying assays to improve patient care and treatment.
Grant funding is made possible in part by the Industry Partnership Fund contributions. Contributors to the IPF receive a dollar-for-dollar state tax credit.
Chartered in 1983 by the state as a public, nonprofit corporation, S.C. Research Authority is intended to fuel South Carolina’s innovation economy through the impact of its four programs. SC Academic Innovations provides funding and support to advance translational research and accelerate the growth of university-based startups. SC Facilities offers high-quality laboratory and administrative workspaces for technology-based startups and academic institutions. SC Industry Solutions facilitates and funds partnerships between and among startups, industry and academia. SC Launch mentors and funds technology-based startups that may also receive investments from SCRA’s investment affiliate, SC Launch Inc.
Health Sciences Center at Prisma Health awards Clemson grants for research on cancer treatment, genetics, patient care16 projects funded with generosity of Prisma Health team see more
The Health Sciences Center at Prisma Health has awarded Clemson University researchers 16 grants that range from projects on cancer treatments to the use of exoskeletons for health care providers.
The seed funding supports the mission of the center, a collaborative effort between Clemson University, the University of South Carolina, Furman University and Prisma Health to foster cooperative research.
Windsor Westbrook Sherrill, associate vice president of health research at Clemson University and chief science officer at Prisma Health, hopes that these projects will inform best practices within health care research and influence positive change within the health care system.
“This year’s submissions were phenomenal, and we look forward to seeing the results from these 16 funded projects. Having clinicians and academic researchers involved in these projects ensures that the research has the best chance of creating transformation in health care and health outcomes,” Sherrill said. “Since this program began seven years ago, several projects have received large federal funding and results have been implemented at Prisma Health, helping improve the care of their patients.”
Click here to read complete details about the one-year grant projects, including the names of Clemson and Prisma Health researchers.
Long-standing partnership between Clemson University and MUSC paying off see more
South Carolina is strengthening its position as a hub for high-impact biomedical research with a new multi-million-dollar project that undergirds the long-standing partnership between Clemson University and the Medical University of South Carolina (MUSC) and loops in crucial support from the National Institute of Dental and Craniofacial Research (NIDCR) at the National Institutes of Health (NIH).
Researchers will study temporomandibular joint (TMJ) function, how the TMJ functions in different craniofacial developmental disorders that seem to put the joint at risk for degeneration and how the joint responds to surgical correction of these disorders, researchers said.
The TMJ makes it possible to move the lower jaw to eat and talk. Understanding the stresses on the TMJ before temporomandibular joint disease (TMD) occurs will unlock the mechanisms that put certain individuals at risk for TMD.
The focus of the research aligns with the recommendations made by an ad hoc committee on temporomandibular disorders that was formed under the auspices of the National Academies of Sciences, Engineering, and Medicine’s Health and Medicine Division.
Four of the researchers involved in the new project are connected to the Clemson-MUSC Bioengineering Program. As part of the program, Clemson bioengineering faculty and students are based at MUSC’s Charleston campus where they collaborate closely with MUSC researchers and clinicians.
The new project, funded by a $3.18-million U01 grant from NIDCR, has two principal investigators. Hai Yao, serves as the Ernest R. Norville Endowed Chair and professor of bioengineering at Clemson, professor of oral health sciences at MUSC, the associate department chair for the Clemson-MUSC Bioengineering Program and a member of the national temporomandibular disorder ad hoc committee. Janice Lee is the clinical director of the NIDCR and chief of the Craniofacial Anomalies and Regeneration Section within the NIH intramural research program.
Yao said the project is possible only because of the synergy and complementary strengths of Clemson, MUSC and NIDCR.
“Clemson and MUSC work together so seamlessly it’s as if we are one university, and we both collaborate closely with NIDCR,” he said. “This project is the latest example of how these strategic partnerships are making South Carolina a hub of biomedical research that is recognized globally. Through these partnerships, we are well positioned to address urgent healthcare needs identified by the NIDCR and the National Academy of Medicine.”
Lee said the researchers are uniquely positioned for success.
“The U01 is an extremely competitive grant that requires intra- and extra- mural collaboration utilizing the world-renown resources at the NIH Clinical Center,” Lee said. “It is extra special as this is a first for NIDCR intramural as well. Temporomandibular joint disorders are debilitating conditions, and I am thrilled to be working with Hai Yao and his team to truly move the research forward. His team brings outstanding bioengineering technology to examine craniofacial musculoskeletal function to the Clinical Center; our discoveries will be translated and, ideally, will initiate first-in-human therapies for TMD at the NIH.”
Lee continued: “NIDCR is committed to working with world-class partners such as Clemson and MUSC to advance translational research into temporomandibular disorders. This project will help improve understanding of these disorders, thereby improving outcomes for patients.”
This is particularly important to Lee as she is the oral and maxillofacial surgeon who will be providing the surgical treatments and is acutely aware of the impact that surgery can have on TMD, she said.
Özlem Yilmaz, chair of the Department of Oral Health Sciences at MUSC, said the new project presents an important venue to help patients debilitated with TMJ disorders and underpins South Carolina’s leading position in temporomandibular disorders research.
“New measurement tools and computational models will be tested on patients at the NIH Dental Clinic,” Yilmaz said. “These novel technologies, stemming from more than a decade of teamwork bringing together bioengineers, oral surgeons, and oral biologists at MUSC and Clemson, will push the boundary of the current temporomandibular disorders research.”
Sarandeep Huja, dean of the College of Dental Medicine at MUSC, said the new project further solidifies MUSC’s partnership with Clemson and NIDCR.
“This partnership will help us innovate the future of oral health and wellness,” Huja said. “We will not only be advancing knowledge of temporomandibular disorders but also expanding knowledge for the next generation of oral health providers and researchers. As a practicing clinician and orthodontist, I frequently encounter patients with temporomandibular disorders, in the very type of patients that will be recruited in this study. It is critical we find evidence based treatments for these patients.”
The vice presidents of research at Clemson and MUSC are crucial to the institutions’ partnership, Yao said. Tanju Karanfil is vice president of research at Clemson, and Lori L. McMahon is vice president for research at MUSC.
“We look forward to solidifying the strong foundation that Clemson and MUSC have built,” Karanfil and McMahon said in a joint statement. “These large, high-impact projects are advancing knowledge and creating a new generation of talent, while strengthening the state’s national and international reputation for biomedical research and education.”
Researchers are calling their project “Assessment of Temporomandibular Joint Morphology, Mechanics, and Mechanobiology in Class II and III Target and Surgical Phenotypes.”
Part of what makes the project unique is the collaboration that maximizes the expertise of the investigators.
“Dr. Lee and her craniofacial team at NIDCR will recruit the large number of patients that will be required for the research, characterize the patients, and support their travel and treatment costs,” Yao said.
Clemson and MUSC will perform analysis of temporomandibular joint biomechanics and mechanobiology and put that information into context to better understand patients’ health status and the potential for future problems
Martine LaBerge, chair of Department of Bioengineering at Clemson, said the U01 grant that funds the new project is the first of its kind at Clemson.
“This grant is a testament to the strength of the biomedical research enterprise that Clemson and MUSC are building in partnership with federal collaborators, especially the National Institutes of Health,” she said. “Dr. Yao’s leadership has been crucial to the partnership’s success, and it remains in good hands with him at the helm.”
The project is the latest major NIH grant led by Yao. He is also principal investigator on South Carolina Translational Research Improving Musculoskeletal Health (SC TRIMH), a Center for Biomedical Research Excellence that was founded with an $11-million NIH grant in 2018. Researchers associated with the center have accounted for $8 million in NIDCR awards over the past year.
Anand Gramopadhye, dean of the College of Engineering, Computing and Applied Sciences, said the success underscores the high quality of research that has come out of interdisciplinary partnerships such as the Clemson-MUSC Bioengineering Program.
“Working together in collaboration with federal partners is elevating South Carolina’s position as a place for top-tier biomedical research and predoctoral and postdoctoral education,” he said. “Dr. Yao and his team have built a high-impact program and are continuing to climb. I offer them my whole-hearted congratulations.”
It is the largest amount the Endowment has awarded MUSC in a grant cycle see more
The Medical University of South Carolina (MUSC) Foundation has received four grants totaling $4.59 million from The Duke Endowment. It is the largest amount the Endowment has awarded MUSC in a grant cycle. Since 1994, the Endowment has invested nearly $40 million in MUSC’s lifesaving mission.
“We are grateful to The Duke Endowment for its major investment in our mission and their ongoing partnership to help us lead health innovation for the lives we touch,” said David J. Cole, M.D., FACS, MUSC president. “These grants will make a significant difference as we seek to improve the well-being of children and their families, expand access to care and bolster mental health support.”
The grants will launch four initiatives, each with long-term sustainability plans that advance MUSC’s vision to lead health innovation for the lives we touch.
“The Duke Endowment is proud to partner with MUSC in developing and providing these innovative models of care,” said Lin Hollowell, director of the Endowment’s Health Care program area. “Our founder wanted his philanthropy to increase access to health care and improve well-being for all Carolinians, and that still drives our work today.”
- Virtual home visits for newborns and their families
Grant amount: $1,850,000
South Carolina’s infant mortality rate consistently ranks among the highest in the nation. Low birth weight, sudden infant death syndrome (SIDS) and accidents are among the leading causes of death for babies in South Carolina.
With an Endowment grant of $1,850,000, MUSC will start a virtual home visit program that gives families the healthiest start possible. Before they leave the hospital, families will be asked if they’d like a registered nurse to follow up with them at home.
During a virtual home visit, the nurse will check on the health and safety of the whole family, also screening for signs of depression and domestic violence. If needed, the nurse will connect families with additional resources and support available through the nonprofit SC Thrive.
- Transform health care in rural Pee Dee region
Grant amount: $1,325,000
A $1,325,000 grant from The Duke Endowment will help MUSC to transform health care in South Carolina’s rural Pee Dee region.
With this grant, MUSC will develop an innovative care model for its new hospital under construction in northern Williamsburg County.
This innovative care model will address one of the biggest issues facing Williamsburg County: a lack of diverse health care providers. MUSC will create a pipeline program to recruit diverse Doctor of Nursing Practice and Master of Science in Physician Assistant Studies students from rural and low-income communities. Students who commit to working at the new hospital for at least two years after graduation will receive a scholarship to MUSC.
The hospital will also be fully integrated with the MUSC Health system, with shared medical records and robust telemedicine capabilities that will provide patients with access to services and specialists throughout the entire statewide system.
- Support mental health of pregnant women and new mothers
Grant amount: $895,229
An estimated one in seven pregnant women and new mothers become clinically depressed during pregnancy or in the year after birth. Most obstetricians and gynecologists do not have the training or resources to help these women. As a result, few are diagnosed or treated.
A $895,229 grant from The Duke Endowment will support a new MUSC program that provides pregnant women and new moms with immediate access to mental health care. The program will connect women to a care coordinator who can assess their risk and, if needed, get them access to a psychiatrist within 30 minutes of the call.
The program also includes real-time psychiatric consultations and training for providers who serve pregnant and postpartum women.
- Mental health support for sickle cell disease patients
Grant amount: $525,229
Sickle cell disease is a hereditary blood disorder that predominately affects the Black community. In South Carolina, as many as 4,500 people are living with the disease.
Sickle cell disease can cause extreme pain and other serious health issues that lead to frequent hospital stays. Symptoms of depression and anxiety are also common in these patients. Some patients also develop substance abuse issues, trying to manage the pain.
Currently, MUSC’s adult and pediatric sickle cell disease clinics are focused on pain management. With an Endowment grant of $525,229, MUSC will be able to dedicate a clinical psychologist and licensed professional counselor to embed mental health services in these clinics.
Heartbeat Technologies, Integrated Micro-Chromatography Systems, and Victory Exofibres were accepted see more
Heartbeat Technologies, Integrated Micro-Chromatography Systems, and Victory Exofibres were accepted as South Carolina Research Authority (SCRA) Member Companies. All SCRA Member Companies receive coaching, access to experts in SCRA’s Resource Partner Network, eligibility to apply for grant funding, and the potential to be considered for an investment from SCRA’s investment affiliate, SC Launch Inc.
Heartbeat Technologies LLC has been accepted as an SCRA Member Company. The Charleston-based startup is dedicated to improving cardiac arrest outcomes and developed a device called ‘The SAVER,’ to better perfuse the heart and brain during emergency events.
Integrated Micro-Chromatography Systems Inc has been accepted as an SCRA Member Company. The Irmo-based startup creates, manufactures, and distributes next-generation biotechnology products to clinical and forensic toxicology organizations, academic research facilities, federal government agencies, and health science companies.
Victory Exofibres LLC has been accepted as an SCRA Member Company. The Greenville-based startup company produces super-efficient viral particle isolation kits that enable more efficient medical diagnostic testing.
SCRA welcomes these new Member Companies.
Grant funding is made possible, in part, by the Industry Partnership Fund (IPF) contributions that fuel the state’s innovation economy. Contributors to the IPF receive a dollar-for-dollar state tax credit, making it an easy and effective way to help one of the fastest growing segments of the South Carolina economy. Grant funding for Member Companies creates a direct, positive economic effect and job creation.
Chartered in 1983 by the State of South Carolina as a public, nonprofit corporation, South Carolina Research Authority (SCRA) fuels South Carolina’s innovation economy through the impact of its four programs. SC Academic Innovations provides funding and support to advance translational research and accelerate the growth of university-based startups. SC Facilities offers high-quality laboratory and administrative workspaces for technology-based startups and academic institutions. SC Industry Solutions facilitates and funds partnerships between and among startups, industry, and academia. SC Launch mentors and funds technology-based startups that may also receive investments from SCRA’s investment affiliate, SC Launch, Inc.