Special Issue "Gene Therapy"
A special issue of Pharmaceuticals (ISSN 1424-8247).
Deadline for manuscript submissions: closed (20 September 2012)
Prof. Dr. Leonidas A. Phylactou
Acting Chief Executive Medical Director, The Cyprus Institute of Neurology & Genetics, PO Box 23462, 1683 Nicosia, Cyprus
Interests: RNA biology; regulatory RNA molecules; muscle regeneration, identification of genetic defects in inherited diseases
Gene therapy is considered very promising for the therapy of several diseases, whether these are inherited or acquired during life. Although it is a field which has peaked relatively recently, the rationale behind all the gene therapy approaches are based on fundamental genetics and molecular biology, aiming at interfering at all stages of the flow of the genetic information. Currently, gene therapy protocols are developed for the delivery of genetic material with viral and non-viral methods, the down-regulation of gene expression at the DNA, RNA and protein levels (anti-gene, RNA interference, antisense, ribozymes, aptamers etc) and the expression of transgenes and genetic sequences. Gene therapy is now advanced against several common and rare diseases. For example, several approaches are being developed for the therapy of cancer, diabetes, cardiovascular diseases, infectious and inherited disorders. Some of these approaches are at the pre-clinical stage but some have progressed to clinical trials. Gene therapy is the way to treat several diseases in the future and research progress in this field has shown that this is achievable.
Dr. Leonidas A. Phylactou
- gene therapy
- viral vectors
- non-viral vectors
- RNA interference
- inducible expression
- tissue-specific expression
- genome modification