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

Explanation:

Gene therapy is the therapeutic delivery of the nucleic acid into the patients cells in the form of drug to treat disease. It is a complex treatment involves more potential for the treatment of the monogenic diseases and is also associated with a lot of significant risks . The children who were suffering from Wiskott- Aldrich syndrome which is a rare disease and were treated as the part of the clinical gene therapy trial which was carried out in the Germany. After treatment, health of children improved significantly. Then after one to three years following the gene therapy, seven out of ten children developed the cancer in the blood .

Public response towards gene therapy and acceptability of changing the genes varied a good deal from one scenario to the another. Many more techniques needed to be developed and more information of the diseases that need to be understood before the gene therapy which involve changes on the genetic setup of the body , it raises many unique ethical concerns. For decades researches are done to bring the gene therapy to clinic , still very less patients are given any effective gene therapy treatments . Even though gene therapy has been slow to reach patients, it's future is encouraging.