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:6/25/2016)... ... ... First Choice Emergency Room , the largest network of independent freestanding emergency ... its new Mesquite-Samuell Farm facility. , “We are pleased to announce Dr. Ogunleye ... M. Muzzarelli, Executive Medical Director of First Choice Emergency Room. , Dr. Ogunleye ...
(Date:6/25/2016)... Montreal, Canada (PRWEB) , ... June 25, 2016 , ... ... the pursuit of success. In terms of the latter, setting the bar too high ... low, risk more than just slow progress toward their goal. , Research from ...
(Date:6/24/2016)... ... ... a crisis. Her son James, eight, was out of control. Prone to extreme mood shifts ... upset him, he couldn’t control his emotions,” remembers Marcy. “If there was a knife ... and say he was going to kill them. If we were driving on the ...
(Date:6/24/2016)... ... 2016 , ... Comfort Keepers® of San Diego, CA is excited to announce ... program to drive cancer patients to and from their cancer treatments. Comfort Keepers ... of life and ongoing independence. Getting to and from medical treatments is one ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... ... Living, is proud to recognize Dr. Barry M. Weintraub as a prominent plastic ... most beautiful women in the world, and the most handsome men, look naturally ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... June 23, 2016 Bracket , a leading ... next generation clinical outcomes platform, Bracket eCOA (SM) 6.0, ... June 26 – 30, 2016 in Philadelphia ... Clinical Outcome Assessment product of its kind to fully integrate ... Bracket eCOA 6.0 is a flexible platform for electronic ...
(Date:6/23/2016)... announced the establishment of Astellas Farma Colombia (AFC), a new affiliate with operations headquartered in Bogota. Colombia ... ... ... ... Astellas is a pharmaceutical ...
(Date:6/23/2016)... MEDIA, Pa. , June 23, 2016 ... treatments in an outpatient dialysis facility.  Treatments are usually ... to 6 hours per visit, including travel time, equipment ... on a patient, but especially grueling for patients who ... residents of a skilled nursing and rehabilitation centers for ...
Breaking Medicine Technology: