Biochemistry 118

Sophomore Seminar


Genomics & Medicine

Doug Brutlag

Stem Cell Therapy Research Project

Many inherited diseases can be treated with drugs, hormones or other biological products that are missing or abnormally regulated in the disease. Some diseases can also be treated by nutritional methods, especially those diseases that alter metabolism. However, many diseases that are missing membrane proteins or protein complexes in the patient's cells cannot be treated so easily. These diseases of missing membrane proteins, missing protein complexes or even missing or defective cell types or tissues are candidates 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 cell

2) which stem cells might be used

3) can the patients own stem cells (iPSCs or others) be used

4) is genetic therapy on the stem cells required to replace a defective gene

5) has a protocol for a clinical trial been 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 and search either for a disease of interest or for the term stem cell. If you use the Advanced Search button on you can specify the condition (disease) and the intervention (stem cells) at the same time. This will give you an extensive list of clinical trials involving stem cells (over 3,600 at the moment). Please write a short report (4 pages max) that answers the 6 questions above. Please send the paper to

© Doug Brutlag September 7, 2015