Possible treatment of some genetic deficiency diseases--a hypothesis.
A conceptual hypothesis for the possibility of treatment of genetic deficiency diseases utilizing genetic engineering techniques is presented. It is proposed that the gene responsible for the synthesis of a protein which is missing in the patient may be inserted into the patient's stem cells using already established techniques of gene splicing and delivery. The so modified stem cells, if put back into the patient's system (e.g. bone marrow), by virtue of their ability of self multiplication are likely to start synthesizing and continue to produce missing protein. Since stem cells are also capable of differentiating into various blood cells, the nucleated blood cells are also likely to begin production of the protein whose gene was inserted into the stem cells. In this way a blood protein synthesized by any organ (e.g. liver) in normal persons may be synthesized by stem cells or their differentiated forms in the patient resulting in correction of the deficiency. Certain genetically deficient animals may be used to prove this hypothesis.
D005818:Genetic Engineering / Q000379:methods*; D006801:Humans; D011488:Protein Deficiency / Q000235:genetics / Q000628:therapy*