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NGEC Pilot Projects in Genome Engineering


All spots in the NGEC Pilot Project program have been filled and no more pilot project grants are available.


The NGEC supported innovative exploratory work outside of the core goals of the NGEC that contributes to the development of genome engineering, and/or potentially contributes to or expands applications of NGEC core work. Two specific examples of project types that would be excellent candidates for funding are:

1. Evaluation of new technologies for potential future use in genome engineering
One can imagine a number of examples in which new technologies might be usefully applied to NGEC goals, if there were a mechanism for evaluating/demonstrating their utility in a small scale project. However, such evaluation might not fit within the constraints of NGEC component budgeting, or might be more fruitfully carried out initially by an outside investigator with expertise complementary to those of NGEC PIs, and later imported into NGEC applications if the pilot work proved promising.

2. Support of outside investigator use of NGEC-developed reagents and technologies in new applications
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. An example of this would be use of an alternative delivery methodology to deliver an engineered nuclease and template to a non-hematopoietic tissue for the purpose of gene repair.

The only requirement for a project to be considered for funding was that the project must carry out exploratory work in a genome engineering–related field. The use of homing endonucleases in the proposed research is welcomed but not obligatory. Funding was awarded based on discussions of the review committee, with the major criteria for evaluation of each project being the relationship of the project to genome engineering and overall goals of the NGEC, the scientific strength of the proposal and numerical scores provided by reviewers. This program provided a $50,000 grant the first year, with a possible second year at $75,000 (direct costs).

This program was administered with a rolling application cycle and proposals were considered as they were received. 


NGEC Supported Pilot Projects

Matthew Porteus, MD, PhD, Stanford University, Stanford CA
Refining Zinc Finger Nucleases to Target the B-Globin Gene
Completed June 2012

Dave Edgell, PhD, University of Western Ontario, London, ON
Using MUSE to inform design of LAGLIDADG endonucleases for gene targeting
Completed June 2012

Lynn Zechiedrich, PhD, Baylor College of Medicine, Houston, TX
New DNA vector to repair genetic mutation
Completed June 2012

Dave Segal, PhD, UC Davis Genome Center, Davis, CA
In vivo directed evolution of tools for genome engineering
Completed June 2012

Akiko Shimamura, MD, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA
Site-directed gene correction of iPS cells from patients with IBMFS
Completed June 2012

Keith Jerome, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA
Homing endonucleases for the treatment of latent viral infections in humans
Completed June 2011

Beverly Torok-Storb, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA
Development of canine transgenic models by spermatogonial stem cell transplantation
Completed June 2011

Matthew Hirsch, PhD, University of North Carolina, Chapel Hill, NC
Optimization and application of AAV-mediated gene correction in vivo
Completed June 2011

Dusty Miller, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA
Enhancement of homologous recombination with an AAV template by nicking and DSB-inducing homing endonucleases
Completed December 2010

Markus Grompe, MD, Oregon Health and Science University, Portland, OR
Double strand break–induced gene repair of hereditary tyrosinemia using AAV vectors and LAGLIDADG homing endonucleases
Completed June 2009


Publications by Pilot Project recipients


Catanese DJ, Fogg JM, Schrock DE, Gilbert BE, Zechiedrich L.  2012.  Supercoiled Minivector DNA resists shear forces associated with gene therapy delivery.. Gene therapy. 19(1):94-100.


Metzger MJ, McConnell-Smith A, Stoddard BL, Miller DA.  2011.  Single-strand nicks induce homologous recombination with less toxicity than double-strand breaks using an AAV vector template.. Nucleic acids research. 39(3):926-35.
Halbert CL, Metzger MJ, Lam S-L, Miller AD.  2011.  Capsid-expressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production.. Gene therapy. 18(4):411-7.
Aubert M, Ryu BY, Banks L, Rawlings DJ, Scharenberg AM, Jerome KR.  2011.  Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI.. PloS one. 6(2):e16825.
Takeuchi R, Lambert AR, Mak AN-S, Jacoby K, Dickson RJ, Gloor GB, Scharenberg AM, Edgell DR, Stoddard BL.  2011.  Tapping natural reservoirs of homing endonucleases for targeted gene modification.. Proceedings of the National Academy of Sciences of the United States of America. 108(32):13077-82.


Hirsch ML, Green L, Porteus MH, Samulski RJ.  2010.  Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair.. Gene therapy. 17(9):1175-80.
Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M.  2010.  Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.. Hepatology (Baltimore, Md.). 51(4):1200-8.


Knouf EC, Metzger MJ, Mitchell PS, Arroyo JD, Chevillet JR, Tewari M, Miller DA.  2009.  Multiple integrated copies and high-level production of the human retrovirus XMRV (xenotropic murine leukemia virus-related virus) from 22Rv1 prostate carcinoma cells.. Journal of virology. 83(14):7353-6.




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