The intense social discourse conserning the propriety and etical implications of gene therapy has been largely resolved with the realization that somatic gene therapy is in principle no different than replacing deficient functions with enzyme replacement therapy or organ transplantation. Because of intense social concern generated by gene therapy, clinical trials in the United States are reviewed in public by the Recombinant Advisory Committee (RAC) of the National Institute of Health as well as by the Food and Drug Administration (FDA) through the traditional IND (Investigational Drug) process. A similar review process has been established by many European countries and the European Union,
It should be noted that genetic manipulation of the inherited germ line (sperm or eggs) continues to raise profound social and ethical questions, and clinical trials of such technologies are explicity excluded under current regulations in the United States and many other countries.
As public concern about gene therapy in general has abated, has been increased focus on the clinical and regulatory issues involved in developing gene therapies for clinical application. The initial clinical trials have been largely academic efforts to assess the feasibility of gene transfer into human subjects. These trials have used various vector delivery technologies and have focused on end-stage diseases such as cancer.Such trials provide little information about the pharmacology and toxicology of gene therapies, issues which have to be studied in a more normal physiological context. Most of these trials have also not had control groups to allow any significant assessment of effectiveness.
Future clinical trials will assume a different focus. These trials will increasingly resemble trials of conventional pharmaceuticals, designed to provide a critical assessment of therapeutic products leading to regulatory approval for marketing and clinical use. Such studies must be rigorous, ethical, controlled, statistically significant, and cost-effective and must be coupled with the development, of methods for manufacture of gene therapy products. These trials will follow the conventional guidelines of investigation and regulatory review.
 Somatic Gene Therapy | Customer Review: Click Here for More Info | $199.95  |
 Somatic Gene Therapy Technologies, Applications and Regulations | Customer Review: Click Here for More Info | $310.00 |
 Somatic Viruses in Human Gene Therapy | Customer Review: Click Here for More Info | $219.00 |
 Somatic Gene Therapy Methods, Processing, Regulation and Validation | Customer Review: Click Here for More Info | $159.00 |
 The Use of DNA as a Drug Somatic Gene Therapy | Customer Review: Click Here for More Info | $55.00 |
0 comments