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
Head, Department of Molecular Genetics, Function & Therapy, 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
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. Papers will be published continuously (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are refereed through a peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed Open Access monthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 500 CHF (Swiss Francs). English correction and/or formatting fees of 250 CHF (Swiss Francs) will be charged in certain cases for those articles accepted for publication that require extensive additional formatting and/or English corrections.
- gene therapy
- viral vectors
- non-viral vectors
- RNA interference
- inducible expression
- tissue-specific expression
- genome modification
Pharmaceuticals 2011, 4(10), 1381-1399; doi:10.3390/ph4101381
Received: 1 August 2011; in revised form: 3 October 2011 / Accepted: 11 October 2011 / Published: 24 October 2011| Download PDF Full-text (518 KB) | Download XML Full-text
Article: The Signalling Role of the avβ5-Integrin Can Impact the Efficacy of AAV in Retinal Gene Therapy
Pharmaceuticals 2012, 5(5), 447-459; doi:10.3390/ph5050447
Received: 20 February 2012; in revised form: 3 April 2012 / Accepted: 19 April 2012 / Published: 2 May 2012| Download PDF Full-text (572 KB) | Download XML Full-text
Review: Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications
Pharmaceuticals 2012, 5(6), 553-590; doi:10.3390/ph5060553
Received: 17 April 2012; in revised form: 18 May 2012 / Accepted: 23 May 2012 / Published: 4 June 2012| Download PDF Full-text (442 KB) | Download XML Full-text
Article: Physical Factors Affecting Plasmid DNA Compaction in Stearylamine-Containing Nanoemulsions Intended for Gene Delivery
Pharmaceuticals 2012, 5(6), 643-654; doi:10.3390/ph5060643
Received: 17 May 2012; in revised form: 9 June 2012 / Accepted: 13 June 2012 / Published: 18 June 2012| Download PDF Full-text (442 KB) | Download XML Full-text
Review: The Liver as a Target Organ for Gene Therapy: State of the Art, Challenges, and Future Perspectives
Pharmaceuticals 2012, 5(12), 1372-1392; doi:10.3390/ph5121372
Received: 16 November 2012; in revised form: 5 December 2012 / Accepted: 6 December 2012 / Published: 10 December 2012| Download PDF Full-text (419 KB) | Download XML Full-text
Review: Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy
Pharmaceuticals 2013, 6(7), 813-836; doi:10.3390/ph6070813
Received: 13 May 2013; in revised form: 14 June 2013 / Accepted: 14 June 2013 / Published: 27 June 2013| Download PDF Full-text (682 KB) | Download XML Full-text
Last update: 11 December 2012