You are a researcher at Advanced Bio Delivery Systems (ABDS), a company that supplies vectors for use in gene therapy. The marketing department has come to you for assistance. They would like to sell the vectors made by ABDS to biotech companies pursuing specific gene therapies. It is your job to research the disorders these biotech companies are targeting and decide on a vector that has potential to work. You will be presenting your findings to the marketing department for approval. There may be more than one vector possibility, so your job is to choose what you feel is the best one and defend your position. Your company stands to make a considerable profit if you are able to "sell" their vector for use in research.
Activity Details
Acting as employees of a vector supply company, research a disease that is a candidate for gene therapy and determine the best vector to use. You must then present your choice in a brochure and the reasoning behind it in order to help "sell" the vector to a biotech company developing a therapy for this disease.
Here are some choices for diseases and genes to research but feel free to select your own:
- neurofibromatosis type 1 and the NF1 gene
- hemophilia and blood clotting factors
- non-small cell lung cancer and p53, a tumor suppressor gene
- SCID and ADA gene
Some things to consider for your brochure:
What are important characteristics of the disease being investigated (genetic defect, body system affected...)?
Will the gene fit into the vector?
What types of cells would the therapy need to target (dividing/non-dividing)?
What will happen to the genetic material once it is inserted into the cells?
Does gene expression need to last a long time?
What kinds of safety issues need to be considered before recommending a vector type?
A tool to create your 3-Panel brochure is is linked: Brothers Creative Center. You can also use the brochure template on any word processing program--Word or Google docs. Just make sure that the file is in a readable format for everyone before you submit it.
Post your completed brochure by the end of Week 9.
Review the brochures posted by your peers. Share thoughts on the brochures. Leave comments to at least two brochures. You can use the following questions to direct your comments:
Were important characteristics of the disease addressed and described in detail?
Were important characteristics of the vector addressed and described in detail?
Will the vector chosen bind to the target cells? Was the description of targeting strategy detailed?
Did the brochure include a detailed description of what will happen to the DNA as it is integrated into the cell?