Navigation Links
Commentary and podcast on landmark gene therapy clinical trial for hemophilia B

New Rochelle, NY, December 12, 2011A recent article in the New England Journal of Medicine showed that Factor IX gene therapy in patients with hemophilia B was able to convert severe hemophilia to moderate or even mild disease. In a timely commentary, Thierry VandenDriessche, PhD, Methods Editor for Human Gene Therapy and Human Gene Therapy Methods says that this clinical trial represents an important milestone and "demonstrates unequivocally that gene therapy can result in a sustained therapeutic effect in hemophilia B patients." Read the commentary and listen to the accompanying podcast from Human Gene Therapy. Human Gene Therapy and HGT Methods are peer-reviewed journals published by Mary Ann Liebert, Inc..

The clinical trial reported in NEJM "builds on prior work and on encouraging pre-clinical data in non-human primates," states VandenDriessche and coauthor of the commentary with Marinee Chuah, PhD, Department of Gene Therapy & Regenerative Medicine, Free University of Brussels and the Center for Molecular and Vascular Biology, University of Leuven, Belgium. It demonstrated that injection of an adeno-associated virus (AAV) vector carrying the gene for the clotting factor FIX into patients with severe hemophilia B led to efficient delivery of the gene into liver cells and sufficient production of the FIX protein to transform the disease from severe to a moderate or mild form of hemophilia, although it does not represent a cure. This effect has been stable and has eliminated or substantially reduced the need for the patients to receive standard protein replacement, which is cumbersome and expensive. Substantial improvements in vector technology set the stage for this success; these included a self-complementary genome to increase efficiency pioneered by R. Jude Samulski, PhD, Director, Gene Therapy Center, University of North Carolina at Chapel Hill, and the use of a novel AAV capsid from a natural rhesus monkey isolate called AAV8, which was discovered and developed by James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy and Human Gene Therapy Methods, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia.

Recently published articles in Human Gene Therapy document the development and optimization of the AAV vectors used to deliver the FIX gene in this clinical trial. Two key papers are:

"The successful treatment of patients with hemophilia B with gene therapy is a landmark event representing the culmination of over 20 years of work by many laboratories," says James M. Wilson, MD, PhD. "We are delighted that Human Gene Therapy was a venue for publishing critical aspects of the development of this product."


Contact: Cathia Falvey
Mary Ann Liebert, Inc./Genetic Engineering News

Related biology news :

1. BUSM faculty author commentary on the global challenges of emerging viral infections
2. Expert commentary on Earth Days 40th anniversary
3. Issues Commentary for Investors of Homeland Defense Companies FOUR, MAGS, NSSC, PNTR, BCO, and CKP
4. New American Chemical Society podcast: Genetically-engineered spider silk for gene therapy
5. American Migraine Foundation launches new podcast series
6. New American Chemical Society podcast: Tiny sea creature and a new medical adhesive
7. LSUHSC conducts landmark study of mid, long-term health effects of oil exposure
8. Landmark report reveals immense burden of osteoporotic fractures in Europe
9. OHSU Doernbecher Childrens Hospital conducts second phase of landmark Batten study
10. Terumo Medical Corp. reports first US implant in landmark study enrolling in US and Japan
11. Fruit flies use horizontal landmarks for altitude control, says Caltech research team
Post Your Comments:
Related Image:
Commentary and podcast on landmark gene therapy clinical trial for hemophilia B
(Date:8/23/2017)... , Aug. 23, 2017  The general public,s help is being ... microbiome—the bacteria that live in and on the human body –and are ... The Microbiome ... the human microbiome, starting with the gut. The project's goal is to ... Photo credit: IBM ...
(Date:6/14/2017)... June 15, 2017  IBM (NYSE: IBM ) is introducing ... event dedicated to developing collaboration between startups and global businesses, ... 15-17. During the event, nine startups will showcase the solutions ... various industries. France ... market, with a 30 percent increase in the number of ...
(Date:4/24/2017)... 2017 Janice Kephart , former ... Strategy Partners, LLP (IdSP) , today issues the ... Trump,s March 6, 2017 Executive Order: Protecting ... can be instilled with greater confidence, enabling the ... refugee applications are suspended by until at least ...
Breaking Biology News(10 mins):
(Date:10/11/2017)... ... October 11, 2017 , ... At ... Purple announced Dr. Suneel I. Sheikh, the co-founder, CEO and chief research scientist ... has been selected for membership in ARCS Alumni Hall of Fame . ...
(Date:10/11/2017)... Oct. 11, 2017  VMS BioMarketing, a leading provider of ... oncology Clinical Nurse Educator (CNE) network, which will launch this ... communication among health care professionals to enhance the patient care ... staff, and other health care professionals to help women who ... ...
(Date:10/10/2017)... ... October 10, 2017 , ... San Diego-based team building and cooking ... initiative announced today. The bold new look is part of a transformation to ... into a significant growth period. , It will also expand its service offering from ...
(Date:10/10/2017)... 2017 SomaGenics announced the receipt of a ... RealSeq®-SC (Single Cell), expected to be the first commercially ... microRNAs) from single cells using NGS methods. The NIH,s ... accelerate development of approaches to analyze the heterogeneity of ... techniques for measuring levels of mRNAs in individual cells ...
Breaking Biology Technology: