Navigation Links
'Gene Therapy for Human Disease: Clinical Advances and Challenges'
Date:3/21/2013

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.


'/>"/>

Contact: Sarah Barth
s.barth@elsevier.com
215-239-6087
Elsevier
Source:Eurekalert

Related medicine news :

1. NIH awards $20 million over 5 years to train next generation of global health researchers
2. KRAS gene mutation and amplification status affects sensitivity to antifolate therapy
3. IntegraGen launches ARISk test, a genetic screening tool for autism in high-risk children
4. Researchers Find Gene Mutations That May Be a Key to Autism
5. Innovation Generation
6. 2 genetic deletions in human genome linked to the development of aggressive prostate cancer
7. Head, Body Lice Are Genetically Very Similar
8. Changes in gene expression may help explain high blood pressure in pregnancy
9. Certain Genetic Regions May Be Tied to Osteoporosis
10. Researchers ID Genes That May Determine Mental Illness
11. Target set on cancer gene MCL1
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/3/2016)... ... May 03, 2016 , ... Dr. Poneh Ghasri, cosmetic ... treatments to both new and existing patients. Cosmetic dentistry allows patients to improve the ... have healthy smiles with some minor or more serious cosmetic flaws. These specials allow ...
(Date:5/3/2016)... ... May 03, 2016 , ... Celestix ... announced the new SecureAccess feature for its CelestixEdge solution. CelestixEdge is the ... enable organizations to get the DirectAccess user experience on unsupported DirectAccess clients ...
(Date:5/3/2016)... , ... May 03, 2016 , ... ... helps healthcare organizations, especially medium and small physician practices, to better grasp and ... unique step-by-step approach that guides practices on a well-defined, expert-created path to compliance. ...
(Date:5/2/2016)... ... May 03, 2016 , ... RNK Products, Inc. (RNK) ... software that enables the stethoscope stream to go over the video conferencing audio ... flagship PCP-USB stethoscope. , Remote auscultation involves two software elements on the PC: ...
(Date:5/2/2016)... ... May 02, 2016 , ... The ... Patient Experience Journal (PXJ), an international, open access, peer-reviewed journal focused on research ... issue representing international (non-US) based authors, the third volume of PXJ continues to ...
Breaking Medicine News(10 mins):
(Date:5/2/2016)... NEW YORK , May 2, 2016 ... the EMR (Electronic Medical Records) market in a recent ... International sales, vendor switches, Increased physician usage, a growing market ... trends were noted in Kalorama,s report EMR ... The report marks Kalorama,s seventh complete study of the ...
(Date:5/2/2016)...  Celsion Corporation (NASDAQ: CLSN ), an ... the first cohort of patients in its Phase ... combining GEN-1, the Company,s DNA-based immunotherapy, with the ... patients with advanced ovarian cancer who will undergo ... the first three patients dosed, GEN-1 plus standard ...
(Date:4/29/2016)... April 29, 2016 Acquisition ... Sciences, Product Development Capabilities in North ... Base . Indegene ( http://www.indegene.com ... the acquisition of Skura Corporation,s life science business. ... adaptive sales enablement technology for life science organizations ...
Breaking Medicine Technology: