'Gene Therapy for Human Disease: Clinical Advances and Challenges'

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

2013-03-21

(Press-News.org) 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.

### 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, 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. http://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 publisher of scientific, technical and medical information products and services. The company works in partnership with the global science and health communities to publish more than 2,000 journals, including The Lancet and Cell, and close to 20,000 book titles, including major reference works from Mosby and Saunders. Elsevier's online solutions include SciVerse ScienceDirect, SciVerse Scopus, Reaxys, MD Consult and Nursing Consult, which enhance the productivity of science and health professionals, and the SciVal suite and MEDai's Pinpoint Review, which help research and health care institutions deliver better outcomes more cost-effectively.

A global business headquartered in Amsterdam, Elsevier employs 7,000 people worldwide. The company is part of Reed Elsevier Group PLC, a world-leading publisher and information provider, which is jointly owned by Reed Elsevier PLC and Reed Elsevier NV. The ticker symbols are REN (Euronext Amsterdam), REL (London Stock Exchange), RUK and ENL (New York Stock Exchange).

ELSE PRESS RELEASES FROM THIS DATE:

'Sideline quasars' helped to stifle early galaxy formation, says CU-Boulder study

2013-03-21

University of Colorado Boulder astronomers targeting one of the brightest quasars glowing in the universe some 11 billion years ago say "sideline quasars" likely teamed up with it to heat abundant helium gas billions of years ago, preventing small galaxy formation. CU-Boulder Professor Michael Shull and Research Associate David Syphers used the Hubble Space Telescope to look at the quasar -- the brilliant core of an active galaxy that acted as a "lighthouse" for the observations -- to better understand the conditions of the early universe. The scientists studied gaseous ...

Reward linked to image is enough to activate brain's visual cortex

2013-03-21

Once rhesus monkeys learn to associate a picture with a reward, the reward by itself becomes enough to alter the activity in the monkeys' visual cortex. This finding was made by neurophysiologists Wim Vanduffel and John Arsenault (KU Leuven and Harvard Medical School) and American colleagues using functional brain scans and was published recently in the leading journal Neuron. Our visual perception is not determined solely by retinal activity. Other factors also influence the processing of visual signals in the brain. "Selective attention is one such factor," says Professor ...

Do disruptions in brain communication have a role in autism?

2013-03-21

New Rochelle, NY, March 21, 2013—A new study of patterns of brain communication in toddlers with autism shows evidence of aberrant neural communication even at this relatively early stage of brain development. The results are presented in an article in Brain Connectivity, a bimonthly peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Brain Connectivity website at http://www.liebertpub.com/brain. A team of researchers from The Netherlands (University Medical Center Utrecht and Utrecht University, Radboud University Nijmegen ...

Cancer drug shortages mean higher costs and greater risk for patients

2013-03-21

A national survey of health professionals showed that drug shortages are taking a heavy toll on cancer patients, forcing treatment changes and delays that for some patients meant worse outcomes, more therapy-related complications and higher costs. St. Jude Children's Research Hospital investigators played an important role in the study. The survey queried oncology pharmacists and others involved in managing cancer drug shortages for academic medical centers, community hospitals and other cancer treatment facilities nationwide. Of the 243 individuals who completed the ...

Scientists identify gene that is consistently altered in obese individuals

2013-03-21

AUGUSTA, Ga. – Food and environment can chemically alter your gene function and scientists have identified a gene that is consistently altered in obesity. The gene LY86 was among a group of 100 genes identified as likely contributors to obesity through genome-wide association studies comparing the DNA of thousands of obese and lean individuals, said Dr. Shaoyong Su, genetic epidemiologist at the Medical College of Georgia at Georgia Regents University. Su looked at progressively larger groups of obese versus lean individuals and found LY86 consistently and highly chemically ...

Researchers' new method may sharpen microscopic images

2013-03-21

UT Dallas researchers are developing a new low-light imaging method that could improve a number of scientific applications, including the microscopic imaging of single molecules in cancer research. Electrical engineering professor Dr. Raimund Ober and his team recently published their findings in the journal Nature Methods. In the journal, they describe a method which minimizes the deterioration of images that can occur with conventional imaging approaches. "Any image you take of an object is translated by the camera into pixels with added electronic noise," Ober said. ...

Do I know you? Memory patterns help us recall the social webs we weave, finds new Cornell study

2013-03-21

ITHACA, N.Y. — With a dizzying number of ties in our social networks – that your Aunt Alice is a neighbor of Muhammad who is married to Natasha who is your wife's boss – it's a wonder we remember any of it. How do we keep track of the complexity? We cheat, says a Cornell University sociologist in Scientific Reports (March 21), a publication of Nature. Study: http://www.nature.com/srep/2013/130321/srep01513/full/srep01513.html Humans keep track of social information not by rote memorization but with simplifying rules, as you might remember a number sequence that always ...

Genetic risk strategies needed for young, black, female breast cancer patients, Moffitt study shows

2013-03-21

Researchers at Moffitt Cancer Center and colleagues in Canada have published study results focused on black women younger than 50, a population disproportionately afflicted with and dying from early-onset breast cancer compared to their white counterparts. The research published in the Jan. 16 issue of The Breast Journal. Early-onset breast cancer has been associated with mutations in the BRCA1 and BRCA2 genes. These breast cancer predisposing genes were discovered almost 20 years ago and confer a lifetime risk of breast cancer of 60 to 70 percent, as well as a much higher ...

Berkeley Lab scientists read the cosmic writing on the wall

2013-03-21

Thanks to a supersensitive space telescope and some sophisticated supercomputing, scientists from the international Planck collaboration have made the closest reading yet of the most ancient story in our universe: the cosmic microwave background (CMB). Today, the team released preliminary results based on the Planck observatory's first 15 months of data. Using supercomputers at the U.S. Department of Energy's (DOE) National Energy Research Scientific Computing Center (NERSC) Planck scientists have created the most detailed and accurate maps yet of the relic radiation ...

For the first time, recommendations offer guidance about incidental genetic findings

2013-03-21

Boston – In a highly anticipated report, landmark recommendations on the handling of incidental findings in clinical genome and exome sequencing are being issued from the American College of Medical Genetics and Genomics (ACMG). A report of the recommendations, led by Robert C. Green, MD, MPH, a medical geneticist at Brigham and Women's Hospital (BWH), outlines for the first time a minimum list of genetic conditions, genes and variants that laboratories performing clinical sequencing should seek and report to the physicians that ordered the testing -- regardless of the ...

'Gene Therapy for Human Disease: Clinical Advances and Challenges'

ELSE PRESS RELEASES FROM THIS DATE:

LAST 30 PRESS RELEASES: