Special Issue "RNAi-Based Therapeutics"
A special issue of Pharmaceuticals (ISSN 1424-8247).
Deadline for manuscript submissions: closed (30 December 2012)
Prof. Dr. John J. Rossi
Department of Molecular and Cellular Biology, Irell and Manella Graduate School of Biological Sciences, Beckman Research Int. of City of Hope, 1500 East Duarte Road, Duarte, CA 91010, USA
Interests: siRNA; shRNA; RNAi; aptamers; small RNA gene activators; silencers
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.
Pharmaceuticals 2011, 4(12), 1591-1606; doi:10.3390/ph4121591
Received: 16 September 2011; in revised form: 8 December 2011 / Accepted: 9 December 2011 / Published: 16 December 2011| Download PDF Full-text (337 KB) | Download XML Full-text
Review: Potential Use of Polyamidoamine Dendrimer Conjugates with Cyclodextrins as Novel Carriers for siRNA
Pharmaceuticals 2012, 5(1), 61-78; doi:10.3390/ph5010061
Received: 31 October 2011; in revised form: 20 December 2011 / Accepted: 21 December 2011 / Published: 30 December 2011| Download PDF Full-text (572 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(1), 85-107; doi:10.3390/ph6010085
Received: 21 December 2012; in revised form: 8 January 2013 / Accepted: 14 January 2013 / Published: 16 January 2013| Download PDF Full-text (222 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(2), 124-160; doi:10.3390/ph6020124
Received: 20 December 2012; in revised form: 10 January 2013 / Accepted: 22 January 2013 / Published: 28 January 2013| Download PDF Full-text (594 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(2), 223-250; doi:10.3390/ph6020223
Received: 19 December 2012; in revised form: 19 January 2013 / Accepted: 1 February 2013 / Published: 6 February 2013| Download PDF Full-text (1276 KB)
Pharmaceuticals 2013, 6(3), 287-294; doi:10.3390/ph6030287
Received: 31 December 2012; in revised form: 18 February 2013 / Accepted: 27 February 2013 / Published: 5 March 2013| Download PDF Full-text (127 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(3), 295-319; doi:10.3390/ph6030295
Received: 11 January 2013; in revised form: 9 February 2013 / Accepted: 1 March 2013 / Published: 14 March 2013| Download PDF Full-text (1085 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(4), 440-468; doi:10.3390/ph6040440
Received: 24 January 2013; in revised form: 27 February 2013 / Accepted: 13 March 2013 / Published: 25 March 2013| Download PDF Full-text (679 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(4), 510-521; doi:10.3390/ph6040510
Received: 31 December 2012; in revised form: 19 March 2013 / Accepted: 27 March 2013 / Published: 10 April 2013| Download PDF Full-text (622 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(4), 522-535; doi:10.3390/ph6040522
Received: 27 December 2012; in revised form: 28 March 2013 / Accepted: 2 April 2013 / Published: 11 April 2013| Download PDF Full-text (270 KB) | Download XML Full-text
Pharmaceuticals 2013, 6(5), 659-680; doi:10.3390/ph6050659
Received: 28 March 2013; in revised form: 10 April 2013 / Accepted: 24 April 2013 / Published: 29 April 2013| Download PDF Full-text (693 KB) | Download XML Full-text
The below list represents only planned manuscripts. Some of these manuscripts have not been received by the Editorial Office yet. Papers submitted to MDPI journals are subject to peer-review.
Title: Nanoparticles-based Delivery of RNAi Therapeutics: Progress and Challenges
Authors: Jiehua Zhou 1,†, Kato Shum 1,†, John J. Rossi 1,2*
1 Division of Molecular and Cellular Biology, Beckman Research Institute of City of Hope; 2 Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, City of Hope, 1500 East Duarte Rd, Duarte, CA 91010, USA; Email address: Jzhou@coh.org, Kshum@coh.org and Jrossi@coh.org.
Abstract: RNA interference (RNAi) is an evolutionarily conserved, endogenous process for post-transcriptional regulation of gene expression. Although RNAi has recently made it through the pipeline to clinical trials, its application as an ideal, clinical therapeutics require the development of safe and effective delivery systems. Inspired by the immense progress with nanotechnology in drug delivery, efforts have been dedicated to the development of nanoparticles-based RNAi delivery system. For example, a precisely engineered, multifunctional nanocarrier with combined passive and active targeting capabilities may address the delivery challenge to the widespread use of RNAi as a therapy. Therefore, in this review we introduce the major hurdles in achieving efficient RNAi delivery and discuss the current advances in applying nanotechnology-based delivery systems to overcome the delivery drawbacks. In particular, some representative examples of design and apply nanoparticle-based delivery formulations for targeted RNAi therapeutics are highlighted.
Keywords: small interfering (si)RNA; non-viral vector; multifunctional nanoparticle; targeting delivery; cell-specific aptamer.
Type of the Paper: Review
Tentative title: RNAi-based therapy for Diabetic Nephropathy
Authors: Mitsuo Kato & Rama Natarajan
Affiliations: Department of Diabetes, Beckman Research Institute of City of Hope, 1500 East Duarte Road, Duarte, CA 91010, USA; E-Mail: RNatarajan@coh.org
Abstract: Diabetes is associated with significantly increased rates of kidney disease or diabetic nephropathy (DN), a severe microvascular complication that can lead to end-stage renal disease. End-stage renal disease needs to be treated by dialysis or kidney transplantation and also is associated with cardiovascular disease and macrovascular complications. Therefore, effective approaches are needed to reduce the progression of renal dysfunction associated with diabetes. Many contributing factors such as hyperglycemia, hyperlipidemia, advanced glycation end products, growth factors, inflammatory cytokines/chemokines and non-coding RNAs such as microRNAs have been implicated in the pathogenesis of DN. Therefore, targeting genes related to these key factors by small interfering RNAs or chemically-modified oligo-nucleotides can provide new therapeutic modalities for DN. In this review, we will summarize RNA interference-based approaches (including microRNA inhibitors) for the prevention and treatment of DN.
Type of the Paper: Review
Title: New Aspects of Gene-Silencing for Treatment of Cardiovascular Diseases
Authors: Koenig-O, Nolte-A, Walker-T, Schlensak-C, Wendel-HP
Affiliations: Clinical Research Laboratory, Dept. of Thoracic, Cardiac and Vascular Surgery, University Hospital Tuebingen, Calwerstr. 7/1, 72076 Tuebingen, Germany email@example.com (H.P.W.
Abstract: RNA interference (RNAi) represents a novel therapeutic strategy due to sequence-specific gene-silencing through the use of small interfering RNA (siRNA). The modulation of gene expression by short RNAs provides a powerful tool to silence every disease-related or disease-promoting gene of interest. In this review we will outline RNAi mechanisms, currently used delivery systems and its possible applications to the cardiovascular system.
Type of the Paper: Review
Title: RNAi-based therapies and drug delivery for lung diseases.
Authors: Yu Fujita 1,2 , Kazuyoshi Kuwano 2 and Takahiro Ochiya 1
1 Division of Molecular and Cellular Medicine, National Cancer Center Research Institute,Tokyo, Japan.Address; E-Mails: firstname.lastname@example.org; email@example.com 2 Division of Respiratory Diseases, Department of Internal Medicine, Jikei University School of Medicine,Tokyo, Japan.Address; E-Mail: firstname.lastname@example.org
Abstract: RNA interference (RNAi) is rapidly becoming an important method for analyzing gene functions in many eukaryotes and holds promise for the development of therapeutic gene silencing. The induction of RNAi relies on small silencing RNAs, which effect the specific messenger RNA degradation. Two types of small RNA molecules – small interfering RNAs (siRNAs) and microRNAs (miRNAs) – are central to RNAi. Drug discovery studies and novel treatments of siRNAs are currently targeting a wide range of diseases, including viral infections and cancer. Lung diseases in general are attractive targets for siRNAs therapeutics because of their lethality and prevalence. In addition, lung is anatomically accessible to therapeutic agents via intrapulmonary route. Recently, increasing evidence indicates that miRNAs play an important role in lung abnormalities such as inflammation and oncogenesis. It is therefore hoped that the targeting of microRNAs also can be exploited for therapeutic purposes. In this review, we present RNAi delivery method strategies and discuss the current state-of-the-art RNAi-based therapeutics for various lung diseases.
Keywords: RNAi; siRNA; miRNA; drug delivery system; lung diseases; lung cancer
Last update: 18 October 2012