The project is funded by the NIH Roadmap for Medical Research, a new type of NIH grant programme designed to address especially complex problems in research that require expertise across multiple scientific disciplines. This large award comes fresh on the heels of Children's Hospital being ranked ninth in the United States among the country's top paediatric hospitals, at a time when Children's is significantly expanding its research agenda, facilities and staff. During the past year Children's has acquired nearly four acres and 1,5 million developable-square feet of research property in downtown Seattle's prime biotech corridor.
The Northwest Genome Engineering Consortium (NGEC) is collaborative research composed of 11 different projects that will build upon each other to develop methods for gene repair, an innovative approach to gene therapy. Gene repair involves manipulating defective sequences of DNA in a targeted gene to change them to the correct sequence, restoring the gene to normal function and eliminating the cause of the patient's inherited disease. Gene repair requires multiple scientific disciplines to generate new kinds of proteins that can perform the required manipulations and then deliver them to a patient's diseased tissues.
"With this research we hope to develop new and efficient approaches for gene repair in certain types of stem cells and other tissues, and use these methods to improve treatments for genetic diseases affecting these tissues", stated Dr. Scharenberg.
While gene repair ultimately may be useful against a wide range of diseases, Dr. 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 Seattle's strong regional expertise and reputation in this type of stem-cell transplantation.
"We hope it will be possible to remove a patient's existing blood stem cells, repair defective genes in these cells, and then return them back to the same patient once corrected", stated NGEC co-director David J. Rawlings, MD, of Seattle Children's 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 we've previously had few answers for", stated Bruder Stapleton, MD, chief academic officer at Children's and chair, Department of Paediatrics at the UWSOM. "We have five years of very exciting, collaborative science ahead. We're also pleased to join ranks with other NIH Roadmap grant recipients including major research institutions like Harvard, MIT, Yale, Northwestern and the University of California at Los Angeles (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 Children's, UWSOM and the Hutchinson Center. Joining Drs. Scharenberg and Rawlings will be David Baker, PhD; Nancy Maizels, PhD; and Raymond J. Monnat, MD, from the UWSOM, and Hans-Peter Kiem, MD; and Barry Stoddard, PhD, from the Hutchinson Center.
"The Northwest Genome Engineering Consortium builds upon the tradition of Seattle's previous bone marrow transplant work and related biotech research in our region, firmly placing Children's Hospital, UWSOM and the Hutchinson Center at the epicenter of groundbreaking genetic developments that will impact the medicine of tomorrow", stated Dr. Stapleton.
The NIH Roadmap for Medical Research has awarded these significant grants to only nine interdisciplinary research groups in the United States. Each project integrates aspects of different disciplines to address complex health challenges that have so far resisted traditional research approaches. The funding of these nine consortia represents a fundamental change in how biomedical research is conducted.
"These programmes are designed to encourage and enable change in academic research culture to make interdisciplinary research easier for scientists who wish to collaborate in unconventional ways", stated NIH Director Elias A. Zerhouni, MD.
As the sole medical school for Washington, Wyoming, Alaska, Montana, and Idaho, the UWSOM ranks first among public medical schools and second among all medical schools in federal research funding.
At the forefront of paediatric research, the Seattle Children's Hospital Research Institute at Children's Hospital and Regional Medical Center in Seattle conducts research under nine major centres and is internationally recognized for its discoveries in cancer, genetics, health services, immunology, pathology, infectious disease and vaccines. Consistently ranked as one of the best children's hospitals in the country by U.S. News & World Report, Children's serves as the paediatric referral centre for Washington, Alaska, Montana and Idaho. Children's has been delivering superior patient care for 100 years, including advancing new discoveries and treatments in paediatric research, and serving as a primary teaching, clinical and research site for the Department of Paediatrics at the University of Washington School of Medicine.
Together the nine national NIH Roadmap consortia will be funded at a total level of $210 million over five years. Each consortium has an overall principal investigator that is responsible for co-ordinating the efforts of the grant's multiple components. For complete descriptions of all nine national NIH Roadmap for Medical Research projects you can visit the National Institutes of Health website.