Question

Describe some specific failures of gene therapy trials, which have negatively affected public opinion and slowed the development of gene therapy techniques in medicine. How have researchers tried to solve the problems associated with these failures?

Answer 1

Gene therapy is the therapeutic delivery of nucleic acid into patients cells as a drug to treat disease. Gene therapy is very complex medical treatment that is associated not only with great potential for treating monogenic diseases but which is also associated with significant risks. The ten childrens who suffered from Wiskott- Aldrich syndrome ( WAS) a rare genetic disease, were treated as a part of clinical gene therapy trial carried out in Germany. After treatment, health of childrens improved significantly. Then after one to three years following gene therapy, seven of ten childrens developed blood cancer.

Public attitudes towards gene therapy and acceptability of altering genes varied a great deal from one scenario to another. There are many techniques that still need to be developed and diseases that need to be understood more fully before gene therapy involves making changes to body's genetic setup, it raises many unique ethical concerns. Researchers have been working for decades to bring gene therapy to clinic, yet very few patients have received any effective gene therapy treatments, but that doesn't mean gene therapy is an impossible dream. Even though gene therapy has been slow to reach patients, it's future is encouraging.