Volume 1, Issue 1, July 2009
Instructions for Authors
If you are an active scientific researcher in the gene therapy arena, we would like to invite you to contribute to this new open resource. It is our aim to build a content-rich site that can be browsed by anyone wanting to expand their understanding of the development and application of novel genetic therapeutics. We ask you to submit a short description of the work that you and your laboratory currently undertake in gene therapy.
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Copyright Transfer Agreement
If you are interested in publishing an article in the Gene Therapy Review please read the CTA to ensure that you accept our copyright transfer policy.
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Editorial: About the Gene Therapy Review
The Gene Therapy Review was founded in 2008 by Dr Michael L Roberts with a view to establishing the most comprehensive gene therapy resource in the web with content provided by the gene therapy community. Dr Roberts has over 15 years experience in the field, having obtained his PhD from University of Cambridge, UK and and has since spent time in both the academic and industrial sector.
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An Introduction to Gene Therapy, page 1
In this category we introduce gene therapy with basic cell biology articles intended for those wishing to learn more about DNA, genes, cells etc. Teachers or University Professors/Lecturers are welcome to submit their teaching notes in this category.
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Adenoviral Vectors, page 4
Michael L. Roberts
Adenoviruses have the potential to be good gene transfer agents because they can be prepared to high concentrations, infect a broad range of cells and are non-tumourigenic, as their DNA does not integrate into the host genome. This article introduces their application as gene transfer agents.
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Adeno-Associated Viral Vectors, page 7
Magali Cucchiarini
Vectors based on a new class of virus, the adeno-associated virus (AAV), have emerged as favored gene vehicles. AAV is non-pathogenic, replication-defective small human parvovirus (25 nm in diameter) with a 4.7-kb single-stranded DNA (ssDNA) genome. Its application in gene therapy is the subject of this article.
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Baculoviral Vectors, page 15
Jani K. Raty
In 1985 it was discovered that baculovirus with suitable promoter was able to transduce mammalian cells, an observation confirmed not until 1995. Even though baculoviruses are hindered by complement-system of blood, successful ex vivo experiments soon led to successful in vivo experiments in 2000. Since then, there have been several publications using baculoviruses with various targets in vitro and in vivo.
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Retroviral Vectors, page 21
Michael L. Roberts
Retroviruses are one of the most widely applied gene transfer vectors used to date and have been used to cure at least one inherited genetic disorder (ADA-SCID). In this article we briefly introduce the retrovirus as a gene transfer agent.
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Gene Augmentation with p53, page 24
Michael L. Roberts
Replication-defective adenoviruses are widely used as gene transfer vectors to deliver cytostatic or tumour suppressor genes into a variety of cancers, yielding some of the most promising results in the clinic. Indeed, the most successful adenoviral vectors used to date are those that are designed to deliver p53 into tumours. Tumor cells have lost the function of p53 because of mutations in the DNA-binding region of the molecule, a feature that is present in over 50% of all human malignancies. In this article we introduce the concept of reducing tumour mass by over expressing p53.
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Targeted Prostate Cancer Gene Therapy, page 26
Yi Lu
Prostate cancer is the most frequently diagnosed cancer and the second leading cause of cancer deaths in American males today. Novel and effective treatment such as gene therapy is greatly desired. Gene therapy is the direct transfer of DNA into patients’ diseased cells for the purpose of therapy. Viral based gene therapy is to use a genetically-modified, replication defective or so-called cold virus as the gene transfer vehicle. In contrast, nonviral gene therapy is to deliver DNA by nonviral methods. At the current stage, viral gene therapy in general has a much higher gene transfer efficiency in vivo compared to nonviral gene therapy. The early viral-based gene therapy uses tissue-nonspecific promoters, which causes unintended toxicity to other normal tissues. In this mini-review, we will focus on discussion of strategy using transcriptionally-regulated gene therapy strategy for prostate cancer treatment.
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Gene Therapy in Duchenne’s Muscular Dystrophy, page 32
Christophe Pichavant and Jacques P. Tremblay
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease characterized by the absence of dystrophin in the muscle. This large protein of 427 kDa is encoded by a 14 kb mRNA (79 exons). This dystrophin protein is located under the membrane of the muscle fiber and interacts with other trans-membrane proteins. It is needed to insure mechanical stress resistance during muscle contraction. The lack of dystrophin weakens the sarcolemma and thus makes fibers less resistant to stress. In this article we discuss the approaches we have employed in an effort to develop effective gene therapy treatments for DMD.
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Imaging of Gene Therapy Treatments, page 38
Jani K. Raty
We introduce the concept of imaging for gene therapy, specifically in developing protocols designed to assess the efficiacy of gene transfer in vivo.
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Controlling Gene Expression in Ischemic Tissues, page 40
Ethan Yang and Hua Su
Prolonged ischemic insult can cause irreversible tissue injuries. Despite advances in medical and surgical therapies, myocardial infarction and stroke, both consequences of ischemic insults, remain to be the top two causes of morbidity and mortality in the Western world, with survivals usually carrying permanent disabilities. Treatments that help restoring blood flow to ischemic area remain to be one of the most important therapeutic goals. Enhancing the innate angiogenesis by exogenous delivery of angiogenic factors lessens the ischemic injury. However, uncontrolled angiogenic gene expression can cause some unwanted side effects. In this mini-review, we describe two systems that can be used to mediate hypoxia-inducible and tissue-specific gene expression.
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Gene Therapy Clinical Trials Resources, page 44
In this article we describe the various international gene therapy clinical trial resources.
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Research Profile: Magali Cucchianni, page 46
An introduction to the research interests of Magali Cucchianni at the Saarland University Medical Center.
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Research Profile: Jacques P. Tremblay, page 50
An introduction to the research interests of Jacques Tremblay at the CHUL Medical Research Centre.
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Academic Services: RIKEN bank, page 53
The RIKEN DNA Bank [Gene Engineering Division of RIKEN BioResource Center (RIKEN BRC)] maintains a Recombinant Virus Bank (RVB; 1,2) as a stock center of recombinant viruses. The RVB supplies a large number of scientists working in molecular biology and human gene therapy. The RIKEN BRC is a nonprofit organization whose activities are supported by the Ministry of Education, Culture, Sports, Science and Technology (MEXT), Japan. Researchers can access the web site of the RIKEN DNA Bank to search for recombinant adenoviruses and obtain the RDB (RIKEN DNA Bank) number (catalog number) of the recombinant adenoviruses in which they are interested. To recombinant request viruses, they need to prepare request-documents and the indicated material transfer agreement (MTA). Moreover, researchers can access the site ‘How to obtain resources’. The RVB is a unique bank of readymade recombinant viruses that are now available on demand for the cost of shipping and handling only.
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Research Profile: Hua Su, page 56
Hua Su is an Assistant Professor at the Center for Cerebral Vascular Research, Department of Anesthesia and Perioperative, University of California, San Francisco and focusses on developing strategies for angiogenic gene and and cell Therapies for myocardial infarction and ischemic brain injuries.
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Research Profile: Yi Lu, page 57
Dr. Lu has contributed pioneering work in adenoviral vector-based gene therapy for prostate cancer including the comparison study of vector delivery routes to the prostate, development of prostate-specific targeting vectors, and oncolytic virotherapy. His group also cloned and performed functional analysis of a novel apoptosis-inducing gene pHyde.
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Company Profile: Florida Biologix, page 58
Florida Biologix’s primary focus is to produce, test and/or fill cGMP compliant biopharmaceutical clinical trial material on time and at a reasonable price. Their unique combination of assets allows them to meet your outsourcing needs quickly and cost-effectively; accomplishing your goal of moving biologics from bench to clinic.
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Company Profile: Diamond BioPharm, page 60
Diamond BioPharm Limited is one of very few European regulatory companies which has experience in clinical and regulatory development programmes within the gene therapy sector, to an advanced stage of development. Its first European Marketing Authorisation Application submission for a gene therapy product will be in 2009.
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