The Coriell Institute for Medical Research has added a new collection of stem cells to its biobank offerings. The new collection is a result of a collaboration between Coriell and the Rett Syndrome Research Trust (RSRT) and consists of 10 lines of human induced pluripotent stem cells created from blood donated by individuals with Rett syndrome.
Rett syndrome is a rare, neurological disorder that presents itself in children—mostly girls—from 6 to 18 months old. Early signs of the disease include loss of speech and hand use, and other neurological functions. Rett syndrome results from mutations in the MECP2 gene, a gene critical to the production of a protein needed for brain function.
The cell lines in this collection are induced pluripotent stem cells (iPSCs), a type of stem cell that is created in the laboratory from skin or blood cells. Using state-of-the-art techniques, scientists can reprogram these cells back into a stem cell state, and from there these cells can be coaxed into becoming nearly any type of cell found in the human body, including neurons and other types of brain cells. The ultimate goal of RSRT is to cure Rett syndrome and the ability to grow neurons from patient-derived iPSCs provides Rett researchers with a more relevant cell model in which to develop and test treatments.
“Induced pluripotent stem cells have opened a new frontier in rare disease research,” said Nahid Turan, PhD, Chief Biobanking Officer at Coriell. “They allow scientists access to previously unobtainable biomaterials, such as living neuronal material representative of a person with a certain disease. At Coriell, we pride ourselves on being able to offer these vital resources to enable biomedical and disease research and drug discovery and therapeutics, and we are thrilled to include these lines to our biobank collection.”
Unlike the majority of cell lines in the Coriell biobank, the cell lines in this collection are not found in the main catalog yet. For more information on ordering, please click here.
“These resources will facilitate research in the field of Rett syndrome and represent a unique and necessary model system for optimizing curative therapeutics directed at restoring MECP2 mutations in DNA or RNA in humans. We are happy to share these resources with academia and industry alike to expedite the development of curative treatments for Rett,” said Jana von Hehn, PhD, Sr. Director of Research and Clinical Strategy at RSRT. Since the collection became available, biotech companies developing a diverse array of therapeutic approaches as well as academic labs at home and abroad have requested these resources, demonstrating an active need for these tools across the field.
The IRB-approved study collected blood and tissue from 30 families providing a wide range of mutations from which to create iPSCs and support different types of therapeutic development. “We are extremely grateful to the many families who donated their child’s blood and tissue and contributed to the creation of this important resource,” said Monica Coenraads, Executive Director of RSRT.
About the Coriell Institute for Medical Research
Founded in 1953, the Coriell Institute for Medical Research is a nonprofit research institute dedicated to improving human health through biomedical research. Coriell scientists lead research in personalized medicine, cancer biology, epigenetics, and the genomics of opioid use disorder. Coriell also hosts one of the world's leading biobanks—comprised of collections for the National Institutes of Health, disease foundations and private clients—and distributes biological samples and offers research and biobanking services to scientists around the globe. To facilitate drug discovery and disease study, the Institute also develops and distributes collections of induced pluripotent stem cells. For more information, visit Coriell.org.
About RSRT
RSRT was launched in 2008 with a singular goal: to drive the development of treatments and cures for Rett Syndrome and related MECP2 disorders. To date, RSRT has funded over $66 million dollars in academic and industry research. Recent industry partnerships focus on the development of therapeutics targeting the underlying cause of Rett Syndrome, the MECP2 gene.
Rett Syndrome is a neurological disorder resulting from a spontaneous genetic mutation at an incidence of approximately 1 in 10,000 female births. Although rare it is also possible for Rett Syndrome to affect males. After a period of normal development through ages 6 to 18 months, Rett patients develop a spectrum of symptoms, including loss of speech, stereotypic hand movements, cardio-respiratory dysfunction, difficulty coordinating movement and increased risk of seizures. Currently, no cure for Rett Syndrome exists.
