As a genetic condition, it is reasonable to consider treating Alpha-1 with gene therapy. Currently, a number of approaches are being evaluated. These include inserting the normal AAT gene into accessible cells such as muscle or bronchial cells, inserting the normal AAT gene into liver cells (the usual site of synthesis of AAT), and inserting genetic agents that “turn off” production of abnormal AAT in the liver. The first approach is already moving forward into early phase human trials with one particular vector. Recent reports have suggested the second approach may have merit as well. If one is looking to prevent the liver disease of Alpha-1, the third approach may be the most direct.
Stem Cell Therapy
Stem cell therapy, or therapy that employs undifferentiated cells to repair or replace mature tissues or organs, is currently in its infancy, especially with respect to Alpha-1. Stem cells are very primitive, undifferentiated cells and usually do not express proteins that cause a normal individual to recognizing them as “foreign.” Therefore, tissues derived from these cells may not be rejected when administered to a patient. In Alpha-1, research is looking toward repairing the liver or lung using stem cells derived from a variety of sources. Potentially, administering these cells to a damaged liver or lung could promote repair to the defects that are caused by having Alpha-1.
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