Science, medicine, and the future: Molecular genetic approaches to understanding diseaseBMJ 1997; 314 doi: https://doi.org/10.1136/bmj.314.7074.126 (Published 11 January 1997) Cite this as: BMJ 1997;314:126
- John Savill, professor in medicinea
Molecular genetics has greatly increased the understanding of diseases in which there is a single gene defect such as cystic fibrosis. Discovering the gene responsible and its function not only helps determine the pathogenesis of the disease but also offers a possible treatment-gene therapy. Polygenic disorders such as diabetes may soon yield their secrets to the same approach. Animal models of genetic diseases are proving useful research tools, and transgenesis has made xenografting possible. Furthermore, antisense technology allows specific inhibition of undesirably overexpressed genes such as those driving unwanted vascular cell proliferation and restenosis after angioplasty. The completion of the human genome project should make the search for “disease” genes much quicker and will increase still further the importance of these gene based approaches toward diseases.