Navigation Links
Seattle Children's Hospital leads $23.7 million NIH grant to study gene repair
Date:9/6/2007

SEATTLE: Sept. 6, 2007 Seattle Childrens Hospital will receive the largest research grant in its 100-year history for a new five-year, eleven-part grant awarded by the National Institutes of Health (NIH) to study gene repair. The $23.7 million grant will support the Northwest Genome Engineering Consortium, led by Andrew M. Scharenberg, MD, of Seattle Childrens Hospital Research Institute in partnership with the University of Washington School of Medicine (UWSOM) and Fred Hutchinson Cancer Research Center. Approximately $13.2 million will be directly awarded to Childrens, with $5.3 million going to UWSOM and $5.2 million to the Hutchinson Center.

The project is funded by the NIH Roadmap for Medical Research, a new type of NIH grant program designed to address especially complex problems in research that require expertise across multiple scientific disciplines. This large award comes fresh on the heels of Childrens Hospital being ranked ninth in the nation among the countrys top pediatric hospitals, at a time when Childrens is significantly expanding its research agenda, facilities and staff. During the past year Childrens has acquired nearly four acres and 1.5 million developable-square feet of research property in downtown Seattles 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 patients 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 patients 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, said Scharenberg.

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 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 Seattles previous bone marrow transplant work and related biotech research in our region, firmly placing Childrens Hospital, UWSOM and the Hutchinson Center at the epicenter of groundbreaking genetic developments that will impact the medicine of tomorrow, said 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 programs 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, said NIH Director Elias A. Zerhouni, MD.


'/>"/>

Contact: Teri Thomas
206-987-5213
Children's Hospital and Regional Medical Center of Seattle
Source:Eurekalert

Related biology news :

1. New strategies to reduce hospital-aquired infections
2. Hospitalizations because of chicken pox down dramatically since implementation of vaccine
3. Bacteria can survive for weeks on hospital surfaces
4. One in 14 men having a heart attack drive themselves to hospital
5. Minorities, uninsured less likely to receive care at high-volume hospitals
6. Hospital-acquired infections -- Inevitable?
7. Funding to tackle hospital superbugs
8. Controlling antibiotics and antibiotic resistance in hospitals
9. Temple University Hospital investigates treatment for cervical dysplasia
10. Study identifies risk factors for spread of respiratory infections in hospitals
11. Restricting hospital-based services during SARS outbreak had modest impact
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/26/2016)... , April 27, 2016 ... "Global Multi-modal Biometrics Market 2016-2020"  report to their ... , The analysts forecast the global ... of 15.49% during the period 2016-2020.  ... of sectors such as the healthcare, BFSI, transportation, ...
(Date:4/13/2016)... CHICAGO , April 13, 2016  IMPOWER physicians ... are setting a new clinical standard in telehealth ... By leveraging the higi platform, IMPOWER patients can ... weight, pulse and body mass index, and, when they ... quick and convenient visit to a local retail location ...
(Date:3/22/2016)... India , March 22, 2016 /PRNewswire/ ... market research report "Electronic Sensors Market for Consumer ... Proximity, & Others), Application (Communication & IT, ... Geography - Global Forecast to 2022", published ... industry is expected to reach USD 26.76 ...
Breaking Biology News(10 mins):
(Date:5/25/2016)... ... ... Lady had been battling arthritis since the age of two and at ... owner Hannah sought the help of Dr Jeff Christiansen of Superior Veterinary Surgical ... and help with the pain of Lady’s arthritis. Dr Christiansen suggested that in conjunction ...
(Date:5/24/2016)... Mass. (PRWEB) , ... May 24, 2016 , ... ... heart attacks, diabetes, and traumatic injuries, will be accelerated by research at Worcester ... cells into engines of wound healing and tissue regeneration. , The novel method, ...
(Date:5/24/2016)... ... May 24, 2016 , ... Last week, Callan Capital, an integrated wealth management ... The Future of San Diego Life Science event at the Estancia La Jolla Resort ... the event with speakers Dr. Rich Heyman, former CEO of Aragon and Seragon, and ...
(Date:5/23/2016)... ... , ... The need for blood donations in South Texas and across the nation is growing. ... Tissue Center, blood donations are on the decline. In fact, donations across the country are ... in South Texas in the last four years alone. , There is no substitute for ...
Breaking Biology Technology: