Special Issue "Gene Therapy"
QuicklinksA special issue of Pharmaceuticals (ISSN 1424-8247).
Deadline for manuscript submissions: 20 September 2012
Special Issue Editor
Guest Editor
Prof. Dr. Leonidas A. Phylactou
Head, Department of Molecular Genetics, Function & Therapy, The Cyprus Institute of Neurology & Genetics, PO Box 23462, 1683 Nicosia, Cyprus
Website: http://www.cing.ac.cy/mgft/
E-Mail: laphylac@cing.ac.cy
Interests: RNA biology; regulatory RNA molecules; muscle regeneration, identification of genetic defects in inherited diseases
Special Issue Information
Dear Colleagues,
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
Guest Editor
Submission
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.
Keywords
- gene therapy
- viral vectors
- non-viral vectors
- antisense
- RNA interference
- ribozymes
- aptamers
- inducible expression
- tissue-specific expression
- genome modification
Published Papers (2 papers)
  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
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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
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Planned Papers
Title: The Nature of the Biologics Expands the Future Indications of Gene Therapy for the Treatment of Parkinson’s Disease
Author: Massimo S. Fiandaca
Affiliation: Bankiewicz Laboratory, Department of Neurological Surgery, University of California, San Francisco, USA; E-Mail: Massimo.Fiandaca@ucsf.edu
Abstract: The pharmaceutical industry’s development of therapeutic agents for the treatment of Parkinson’s disease (PD) endures, due to the continuing need for better agents, and from the increased demand due to the aging population. Each new drug offers advantages and disadvantages to patients when compared to other medical offerings. Deep brain stimulation (DBS) has become a standard surgical remedy for the effective treatment of select patients with PD, many in which various drug regimens have failed. Similar to DBS as a surgical option, gene therapy for the treatment of PD is evolving as a treatment option of the future. In the four different schemes of PD gene therapy that have reached clinical trials to date, investigators have documented an excellent safety profile associated with the stereotactic delivery, viral vectors utilized, and transgenes expressed. In this article, we review the clinically relevant gene therapy strategies for the treatment of PD, concentrating discussion on the published preclinical and clinical results, and the likely mechanisms involved. Based on these presentations and arguments, we advance an analysis of how the nature of the gene therapy biologic used may eventually expand the scope and utility for the management of PD.
Last update: 18 May 2012