Gene Therapy as a Cure for Fatal Bone Diseases in Children?

This article detailed current research about how, although low bone density and osteoporosis is quite prevalent in the public eye, that excessive bone density and subsequent diseases also present a significant medical challenge. The article reported that such diseases of excessive bone density, such as malignant infantile osteopetrosis, or MIOP, are only currently treated with a risky transplant procedure, but research has shown that gene therapy might provide a safe and effective alternative. This therapy would allow experts to extract stem cells from the patients themselves, eliminating any external donor, and have the non-functioning gene replaced with a working copy, and then reinserted into the patient. Researchers note that the method is not risk-free, and that much work is still required before the technique is ready for practical application.

The article and the research it entails were intriguing in that they display great promise in aiding children suffering from such diseases as malignant infantile osteopetrosis (MIOP). I enjoyed the fact that experts discuss the possibility that once such gene therapy, which does not require external stem cell donors (a fact that I thought would limit the critics of such therapy), is proven successful at treating MIOP, there are several other similarly related ostepetrosis diseases which could also be treated with such therapy. I thought this was interesting due to the fact that much controversy surrounds stem cell research as violating certain natural rights by extracting external stem cells, however in this case, no external stem cell donors would be required. Though this is quite notably early in the development and implementation of such research, I look forward to the results of studies and their practical implementation into alleviating and treating such diseases in the near future.