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Name: Kishori
Status: student
Grade: n/a
Location: Outside U.S.
Country: India
Date: Summer 2013


Question:
When gene transplantation has come into service why can't we do gene transplantation for every disease instead for finding new drugs? wont it be a better idea.?Are there any genes that regulate the pace maker of heart ?



Replies:
Koshori,

Gene therapy certainly has many promising potential applications, though significant technical challenges remain before it can be used clinically. Scientists have suggested that gene therapy might be best suited for treating diseases that are the result of a mutation in a single gene - for example, cystic fibrosis or severe combined immunodeficiency (SCID). In these cases, there is a straightforward target - the mutated gene - and often a clear type of cell we need to reach. In such well-defined cases, it makes sense to use gene therapy to treat the disease.

Many diseases, however, are the result of other processes in the body and cannot be traced to a single mutation. Many aging-related diseases, for instance - such as heart disease or arthritis - are not the result of a single gene 'gone bad'. In fact, there might not even be any mutations! Many diseases occur because of natural processes in our body rather than a mutation. Even if genes are involved, they are often so complicated that targeting a single gene would not have a significant effect on the disease. As such, gene therapy might not be the best approach to treat the disease, especially given the technical challenges associated with safely implanting new genes.

These more complex diseases are often treated with drugs or surgery, which are also fairly blunt instruments to deal with a complex cascade of problems - just as a single gene might not solve the problem, a single drug or single surgery might just buy some time while the underlying problem continues. An emerging area of research, however, involves the transplantation of engineered cells rather than single genes. These cells, if transplanted appropriately, might be able to coax the body's own tissues to repair a problem through a complex, ever-changing set of 'drugs' synthesized by the cell itself. Whole cells are infinitely more complex than a single drug or gene, and provide an exciting opportunity to treat the underlying disease. There already exist some simple cell therapies in the clinic, and as research progresses, we hope that more and more potential therapies will start to help treat patients and save lives.

S. Unterman Ph.D.


#1 Gene transplantation would not be an effective treatment for every disease because not all diseases are caused by gene malfunction, such as insect vector diseases, and infections.

#2 There probably are genes which regulate the heart pacemakers (SA and AV nodes). See attached link regarding gene insertion experiment in which gene insertion into a ordinary guinea pig cardiac muscle cells caused them to transform into pacemaker cells. http://www.the-scientist.com/?articles.view/articleNo/33718/title/New-Biological-Pacemaker/

John Cowsar


Hi Kishori,

Thanks for the question. Gene transplantation will not cure every disease since genes are not directly responsible for all diseases. For instance, fungal infections in the toenail are not related to genetics--at least not in a direct manner. I am unaware of which genes are responsible for the pacemaker of the heart. In order to answer that question, one would need to search the PubMed database and search the original scientific literature.

I hope this helps. Please let me know if you have more questions. Thanks Jeff



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