Fourth Idea for a Homework for those wishing Credit/No Credit
Stem Cell Project
Many inherited diseases can be treated with drugs, hormones or other biological products that are missing or abnormally regulated in the disease patients. Some diseases can also be treated by nutritional methods, especially those diseases which alter metabolism. However many diseases which are missing membrane proteins or protein complexes in the pateints cells cannot be treatedso easily. These diseases of missing membrane proteins, missing protein complexes or even missing or defective cell types or tissues are candicates for treatment with stem cell therapies.
For this homework, I would like you to choose a disease not mentioned in class, that is a candidate for stem cell therapy and describe 1) why the disease might be best treated by stem cells, 2) which stem cells might be used, 3) can the patients own stem cells (iPSCs) be used, 4) is genetic therapy on the stem cells required to replace a defective gene, 5) has a protocl for a clinical trial beeen submitted and 6) what is the preclinical evidence that the method will work.
The best way to discover a potential candidate disease is to go to the web site clinicaltrials.gov and search either for a disease of interest or for the term stem cell. This will give you an extensive list of clinical trials involving stem cells (over 3,000 at the moment). Please write a short report (2 pages max) that answers the 6 questions above for a disease of interest to you. Please send the paper to firstname.lastname@example.org.