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 hem
'/>"/>

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

Page: 1 2

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:7/9/2014)... of flatulence and have a reputation for being highly toxic, ... is now being being found to offer potential health benefits ... attacks and dementia. A new compound (AP39), designed and made ... future therapies, by targeting delivery of very small amounts of ... , Scientists in Exeter have already found that the ...
(Date:7/9/2014)... the most important organs in the human body. Its ... properly this is its synthesis function and ... this is its detoxification function. Lack of exercise and ... the liver. The resulting diseased cells can lead to ... failure. According to the German Liver Foundation, over five ...
(Date:7/9/2014)... prescription labels handed out by pharmacists may be ... medication, according to new research by the University ... the Blind)., The study, published recently in the ... on prescription medications dispensed by pharmacies do not ... By simply following recommended guidelines for font size, ...
(Date:7/9/2014)... , , , ... , , , , ... downrange from the firer and approximately 30cm from the bullet... , , ... , , , , ... , , , , , , University of Southampton researchers, with ...
(Date:7/9/2014)... The occurrence of allergic diseases has risen dramatically in ... are less exposed to microorganisms and have fewer infections ... system. , A study by researchers at Sahlgrenska Academy, ... three to examine maturation of the immune system in ... in rural areas of the Vstra Gtaland Region, half ...
Breaking Medicine News(10 mins):Health News:Rotten egg gas holds key to healthcare therapies 2Health News:Telemedicine for patients with chronic liver diseases 2Health News:Telemedicine for patients with chronic liver diseases 3Health News:Most prescription labels fail to meet guidelines, risking dosage errors 2Health News:Fit for the frontline? New study identifies the hearing requirements of British soldiers 2Health News:Fit for the frontline? New study identifies the hearing requirements of British soldiers 3Health News:Fit for the frontline? New study identifies the hearing requirements of British soldiers 4Health News:Fit for the frontline? New study identifies the hearing requirements of British soldiers 5Health News:Fit for the frontline? New study identifies the hearing requirements of British soldiers 6Health News:Fit for the frontline? New study identifies the hearing requirements of British soldiers 7
... March 17 JumpStart Inc. , ... progress of high growth early-stage businesses, and Case ... and technology validation fund at Case Western Reserve ... in Neuros Medical, Inc. , a Cleveland, OH ...
... Research Group, Inc. has been covering the biologics market ... series which are syndicated primary research reports ... and Rheumatologists. The company is now expanding these ... reports which aim to compare self-reported treatment patterns ...
... in Mexico for Minimally Invasive Treatment for Prostate ... elite urologistsCHARLOTTE, N.C., March 16 Mexico HIFU ... international entities of USHIFU, a global leader in ... focused ultrasound (HIFU) technologies, will host the very ...
... 16 Star Scientific, Inc. (Nasdaq: STSI ... with the Securities and Exchange Commission. The company reported ... unchanged from net sales for 2007. However, volume sales ... and Stonewall(R), increased 7.8% in 2008 compared to 2007. ...
... March 16 /PRNewswire-Asia-FirstCall/ -- BMP,Sunstone Corporation (Nasdaq: ... that the Company is calling its $12.4 million of ... Notes"). In addition, the,Company announced the completion of ... Exchanged $1.0 million in principal amount 10.0% senior secured, ...
... in St. Louis and Johns Hopkins University, in partnership with Experience Corps, has showed that people over 55 ... Click here for more information. , , ... , , , , ... , , , ...
Cached Medicine News:Health News:JumpStart and Case Technology Ventures Invest in Neuros Medical 2Health News:JumpStart and Case Technology Ventures Invest in Neuros Medical 3Health News:JumpStart and Case Technology Ventures Invest in Neuros Medical 4Health News:JumpStart and Case Technology Ventures Invest in Neuros Medical 5Health News:BioTrends Prepares to Field ChartTrends(TM) Reports to Cover the Use of Biologic Agents in Psoriasis and Rheumatoid Arthritis 2Health News:High Intensity Focused Ultrasound Physician Training Seminar in Mexico City 2Health News:Star Scientific Files Annual Financial Report 2Health News:Star Scientific Files Annual Financial Report 3Health News:Star Scientific Files Annual Financial Report 4Health News:BMP Sunstone Exchanges and Calls Outstanding Notes Due May 2009 2Health News:Researchers find sustained improvement in health in Experience Corps tutors over 55 2Health News:Researchers find sustained improvement in health in Experience Corps tutors over 55 3Health News:Researchers find sustained improvement in health in Experience Corps tutors over 55 4
(Date:1/15/2014)... 2014 Reportlinker.com announces that a new ... catalogue: Global Markets ... -- Focus on End Users ... A drug delivery system is ...
(Date:1/15/2014)... , Jan. 15, 2014  A novel wearable injector slightly larger ... painless for patients to self-inject prescription drugs in the large ... cancer, blood diseases, autoimmune deficiencies, and genetic disorders.  ... to $220B by 2018, according to analysts.   Many of these ...
(Date:1/14/2014)... HAMILTON, N.J. , Jan. 14, 2014 InformEx, ... buyers and sellers of high-value chemistry, will hold the 30 ... Miami Beach Convention Center from January 21-24. ... than 3,500 attendees from top pharmaceutical, fine chemical and specialty ...
Breaking Medicine Technology:Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 2Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 3Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 4Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 5Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 6Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 7Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 8Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 9Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 10Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 11Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 12Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 13Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 14Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 15Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 16Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 17Global Markets and Technologies for Advanced Drug Delivery Systems -- Focus on End Users 18Enable Injections Presents A New Class of Injectors for Drug Delivery at PEPTalk, Drug Delivery Partnership Meetings 2Media Advisory: InformEx 2014 Events & Speaker Lineup for 30th Edition Conference in Miami Beach 2
... REDWOOD CITY, Calif., July 14, 2011 Codexis, Inc. (Nasdaq: ... in the 13th Annual Pacific Crest Global Technology Leadership Forum, ... Robert Lawson, Senior Vice President and Chief Financial Officer, will ... Biofuels and Renewable Chemicals," scheduled for August 8 at 12:30 ...
... ACCESS PHARMACEUTICALS, INC. (OTCBB: ... to develop treatments in areas of oncology, cancer supportive ... oral mucositis, MuGard , has been added to ... is now available with standard pharmacy benefit copayment.  Placement ...
Cached Medicine Technology:Access Pharmaceuticals MuGard Added to CVS Caremark's Pharmacy Benefit Network 2Access Pharmaceuticals MuGard Added to CVS Caremark's Pharmacy Benefit Network 3
... PS+ lead series are endocardial ... the best characteristics of polyurethane ... polyurethane outer insulation provides a ... lubricity while the silicone inner ...
The Vitatron Slimtine S lead series are endocardial leads with an excellent history of reliability and optimal handling en pacing performance....
Unipolar Atrial "J" Lead...
VascoTip is a unipolar tined permanent pacing lead. Due to advanced technology, VascoTip 9/60 M is compatible to all pacemaker-cavities with the size of IS-1 (3.2 mm), 5 mm, 6 mm....
Medicine Products: