About the NGEC

The Northwest Genome Engineering Consortium (NGEC) brings together researchers at Seattle Children’s Research Institute, Fred Hutchinson Cancer Research Center and the University of Washington to develop new methods for gene repair, an innovative approach to gene therapy.

The NGEC is funded by the National Institutes of Health (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.

Applying Gene Repair to Immune System and Blood Cell Disorders

Single-gene disorders of the immune system and blood cells have significant health impacts both within the United States and on a global scale. Thus, these diseases are an important area for the development of new treatment approaches. Genome engineering technology has progressed to the point that it is presently feasible to modify individual defective genes in primary human cells, a process termed gene repair.

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.

The main goal of the NGEC is to develop and apply gene repair to the treatment of single gene disorders of the immune system and blood cells. This goal will be accomplished through pursuing two major scientific aims:

Aim #1: Development of methods for LAGLIDADG homing endonuclease engineering
The first aim will support a team approach that combines computational protein design, directed protein evolution, structural analysis and in vitro biochemical and molecular analyses with the goal of pioneering methods for producing artificial LAGLIDADG homing endonucleases (LHEs), for use in a variety of types of genome engineering applications.
Learn more about the NGEC research labs focusing on this area.

Aim #2: Application of LAGLIDADG homing endonucleases to gene repair of hematopoietic stem cells
The second aim will support a team approach to the application of LHE’s for site specific gene repair in hematopoietic stem cells. This approach will combine novel LHE’s created in Aim #1 with the use of non-integrating lentiviral vectors (NIL) for the introduction of an LHE and a repair/modification template to hematopoietic stem cells for the purpose of gene repair. It will also explore new methods for manipulating endogenous DNA repair mechanisms to enhance the in situ efficacy of LHE-induced gene repair in hematopoietic stem cells, and develop methods for autologous hematopoietic stem cell engraftment optimized for gene repair applications.
Learn more about the NGEC research labs focusing on this area.

NGEC Laboratory Connections




In addition to its focus on applying gene repair to hematopoietic stem cells, the NGEC is working to contribute to the development of genome engineering as a broader scientific discipline through sponsoring a postdoctoral training program, an annual genome engineering workshop and smaller-scale, pilot projects relating to genome engineering at other institutions. Developing the Field of Genome Engineering

NGEC Workshop on Genome Engineering
The NGEC hosts an annual genome engineering workshop open to students and faculty from any institution. Workshop talks by NGEC investigators help to disseminate the concepts, methods and tools developed by the NGEC to outside investigators, while talks from investigators outside the NGEC bring in new ideas from across the country and around the world. Learn more.

NGEC Pilot Projects in Genome Engineering
The NGEC supports innovative exploratory work outside of the core goals of the NGEC that would contribute to the development of genome engineering and/or potentially contribute to or expand applications of NGEC core work. By funding projects in which an outside investigator proposes to apply genome engineering methods to a new area or field of work, the impact of the NGEC will be substantially broadened. Learn more.

NGEC Postdoctoral Training Program
The NGEC postdoctoral training program provides an opportunity for qualified fellows to obtain experience and training in the process of LHE-based genome engineering. The program supports postdoctoral fellows who undertake interdisciplinary projects requiring mentorship by two principal investigators with complementary expertise in genome engineering-related fields. Learn more.