Vol. 1, Issue 3 (2013)
GENE THERAPY: AN UPDATED REVIEW
Author(s): Amit Kumar, Peeyush Sharma, & Anil Bhandari
Abstract: Novel treatment modalities like gene therapy and cell therapy are attractive for such diseases where current therapies have failed. Although the clinical potential of these therapies has been demonstrated in the West, their use still remains in experimental stages. In India a number of groups are carrying out basic research on gene delivery vector development and strategies for cancer gene therapy, and development of adult and embryonic stem cells. With the establishment of human embryonic stem cell (hESC) lines in a few centres, India is now geared to become one of the major contributors to the emerging field of DSC. This review will cover some of the published and unpublished work with potential to be translated into clinical trials, and discuss the possible hurdles in carrying out clinical trials. Much attention has been focused on the so-called genetic metabolic diseases in which a defective gene causes an enzyme to be either absent or ineffective in catalyzing a particular metabolic reaction effectively.