Gene Therapy for Human Disease: Clinical Advances and Challenges

Special issue of Translational Research is devoted to the topic of the gene therapy

Philadelphia, PA, March 21, 2013 - The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today. Research articles included in the special issue highlight current studies that, after decades of trial and error, may provide evidence for a clear path of treatment and cure for many diseases. There are more than 1,800 genetic disorders known in humans, and only a small fraction of these can be treated and even fewer cured. Some of these disorders are exceedingly rare, others more common. The approach of gene therapy however may be applicable to all.

“The thirteen articles included in this special issue of Translational Research provide critical examples of the tools and practice of gene therapy today. They all focus on clinically meaningful studies that combine patient observations with smart experiments. The authors hope these articles will facilitate conversion of individual and disease-specific insight into a collective understanding of emerging gene transfer platforms and their subsequent translation to the bedside,” explained contributing author Dr. Jakub Tolar of the Stem Cell Institute and Pediatric Blood and Marrow Transplant Program at the University of Minnesota, in his introduction to the issue. “The concept of gene therapy for genetic disorders is one of the most appealing in biomedicine because it is aimed at the cause rather than the symptoms of the disease.”

Each article of this issue focuses on either a specific condition or a delivery method. Article topics included are: arthritis gene therapy, immunotherapies for type 1 diabetes mellitus, immune responses in liver-directed, lentiviral gene therapy, gene therapy for retinal disease, gene therapy in cystic fibrosis, evaluating risks of insertional mutagenesis by DNA transposons in gene therapy, pluripotent stem cells and gene therapy, gene therapy for hemoglobinopathies: progress and challenge, hemophilia clinical gene therapy-brief review, gene transfer for congestive heart failure, gene therapy for the prevention of vein graft disease, gene therapy for brain tumors, oncolytic virus therapy for cancer, and T cell-based gene therapy of cancer.

With the publication of this special issue, Translational Research identifies a need for clinical trial coordination among researchers worldwide, a focused goal of a world-scale change in medical practice, and real-time data exchange and evaluation, With these elements in place the true potential of gene therapy to treat and cure disease becomes apparent.

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Notes for Editors
The articles appear in Translational Research, Volume 160, Issue 5 (April 2013), titled “Gene Therapy for Human Disease: Clinical Advances and Challenges,” published by Elsevier, now available on ScienceDirect.

Full text of the articles included in the special issue is available to credentialed journalists upon request. Contact Sarah Barth at +1 215 239 6087, s.barth@elsevier.com to obtain copies or to schedule an interview with Dr. Jeffrey Laurence, MD, Editor-in-Chief.

About Translational Research
The official journal of the Central Society for Clinical Research (www.cscr.org), Translational Research delivers original investigations in the broad fields of laboratory, clinical, and public health research. Interdisciplinary and cross-disciplinary in scope, it keeps readers up-to-date on significant biomedical research from all subspecialties of medicine. Aiming to expedite the translation of scientific discovery into new or improved standards of care, it promotes a wide-ranging exchange between basic, preclinical, clinical, epidemiologic, and health outcomes research. www.translationalres.com

Translational Research encourages submission of studies describing preclinical research with potential for application to human disease, and studies describing research obtained from preliminary human experimentation with potential to refine the understanding of biological principles underpinning human disease. Also encouraged are studies describing public health research with potential for application to the clinic, disease prevention, or healthcare policy.

About Elsevier

Elsevier is a world-leading provider of information solutions that enhance the performance of science, health, and technology professionals, empowering them to make better decisions, deliver better care, and sometimes make groundbreaking discoveries that advance the boundaries of knowledge and human progress. Elsevier provides web-based, digital solutions — among them ScienceDirect, Scopus, Elsevier Research Intelligence, and ClinicalKey — and publishes nearly 2,200 journals, including The Lancet and Cell, and over 25,000 book titles, including a number of iconic reference works.

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Media contact
Sarah Barth
Elsevier
+1 215 239 6087
s.barth@elsevier.com