Seattle Children's Hospital leads $23.7 million NIH grant to study g...(ir in certain types of stem cells and ot...)

While gene repair ultimately may be useful against a wide range of diseases, Scharenberg believes single-gene inherited disorders of the lymph and bone-marrow systems such as immune deficiencies, Sickle Cell Disease and thalassemias are the best place to start.

Collectively, these disorders are a major global disease burden in children. The target cells in these diseases that will be manipulated by the gene-repair process are blood stem cells, and they are readily accessible. By working with these disorders, the NGEC will build upon Seattles strong regional expertise and reputation in this type of stem-cell transplantation.

We hope it will be possible to remove a patients existing blood stem cells, repair defective genes in these cells, and then return them back to the same patient once corrected, said NGEC co-director David J. Rawlings, MD, of Seattle Childrens Hospital Research Institute.

This repair approach potentially bypasses the complications, treatments and costs associated with rejection experienced by patients receiving replacement stem cells from a different individual.

This complex project provides major new hope for many inherited diseases weve previously had few answers for, said Bruder Stapleton, MD, chief academic officer at Childrens and chair, Department of Pediatrics at the UWSOM. We have five years of very exciting, collaborative science ahead. Were also pleased to join ranks with other NIH Roadmap grant recipients including major research institutions like Harvard, MIT, Yale, Northwestern and UCLA to address some of the most complex problems in modern medicine.

The NGEC research project features 11 different inter-related components, bringing together staff and resources from Childrens, UWSOM and the Hutchinson Center. Joining Scharenberg and Rawlings will be D
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