Strategic Plan

Our strategic plan continues to identify selective delivery of gene therapeutics as the crucial bottleneck problem of the envisioned gene therapy. This view remains strongly consistent with consensus in the field. We are focusing on two complementary classes of ultimate engineered gene delivery systems as driving our integrated research, education, and industry interaction efforts:

1) an ex vivo approach employing genetically engineered stem cells (Project Area A)
2) an in vivo approach employing targeted viral or synthetic vectors (Project Area B)

We can effectively motivate this strategic plan by communicating an explicit statement of our Vision for what BPEC aims to accomplish in this broad and vigorous field of research, using some important illustrations.

Figure 5.1 clarifies and motivates the BPEC vision in the context of the gene therapeutics field. Our vision is to generate, as novel engineered systems, prototype selective gene delivery vehicles in each of the gene therapeutics categories still requiring major research advances in order to achieve appropriately effective gene delivery vehicles. Viewing gene delivery as the bottleneck problem, as stated earlier, three gene therapy categories can be identified and illustrated.


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Strategic Plan Our strategic plan continues to identify selective delivery of gene therapeutics as the crucial bottleneck problem of the envisioned gene therapy. This view remains strongly consistent with consensus in the field. We are focusing on two complementary classes of ultimate engineered gene delivery systems as driving our integrated research, education, and industry interaction efforts: 1) an ex vivo approach employing genetically engineered stem cells (Project Area A) 2) an in vivo approach employing targeted viral or synthetic vectors (Project Area B) We can effectively motivate this strategic plan by communicating an explicit statement of our Vision for what BPEC aims to accomplish in this broad and vigorous field of research, using some important illustrations. Figure 5.1 clarifies and motivates the BPEC vision in the context of the gene therapeutics field. Our vision is to generate, as novel engineered systems, prototype selective gene delivery vehicles in each of the gene therapeutics categories still requiring major research advances in order to achieve appropriately effective gene delivery vehicles. Viewing gene delivery as the bottleneck problem, as stated earlier, three gene therapy categories can be identified and illustrated.	Category I comprises therapeutics involving delivery where the cell type in which the transgene is expressed is essentially unimportant, the expression level needs to only be above a minimal threshold, and there is little need for longevity or repeatability of expression. Example clinical applications for Category I include DNA vaccines and plasmid enhanced wound healing. In this category, effective delivery vehicles are already approved or close to approval for therapeutic indications. Thus, we see no significant role for BPEC in this category because the knowledge base and technology base for the endpoint engineered systems are adequate. Category II comprises therapeutics involving delivery where the cell type in which the transgene is expressed is very important, the expression level again needs to be merely above a threshold, and longevity or repeatability of expression may or may not necessarily be important. continue
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