HOME >> BIOLOGY >> NEWS
New gene therapy tool successfully treats mice with hemophilia A

(Embargoed) CHAPEL HILL -- Scientists at the University of North Carolina have successfully treated mice with hemophilia A using a new approach to gene therapy - RNA trans-splicing. The experimental procedure repairs a mutated section of the gene responsible for hemophilia A, a hereditary bleeding disorder.

Dr. Hengjun Chao, a research assistant professor at the UNC School of Medicine, Gene Therapy Center will present the new research Saturday June 8 in Boston at the Presidential Symposium of the American Society of Gene Therapy Annual Meeting.

Hemophilia A is a sex-linked congenital disease, occurring in one out of 5,000 to 10,000 live males in all populations and is caused by a defect in coagulation factor VIII. The mutation renders the factor VIII gene non-functional resulting in recurrent, non-predictable, spontaneous bleeding into major joints and soft tissues. Currently, the disorder is treated with injections of factor VIII protein in response to bleeding incidents. Conventional approaches to gene therapy have not proven successful against hemophilia A, partially due to difficulties involved in packaging and delivering the large factor VIII gene.

This new study in mice with hemophilia A was conducted in collaboration with scientists from Intronn Inc., Rockville, Maryland, where RNA trans-splicing was pioneered. A "pre-trans-splicing molecule" (PTM) was injected into some of the mice. The molecule, or "cassette," is designed to produce RNA that binds and splices onto the existing faulty RNA, correcting it. The corrected RNA then encodes for the normal factor VIII protein.

"Preliminary data using the hemophilia A mice is very encouraging," said Dr. Hengjun Chao, "After injecting hemophilia A mice with the PTM cassette, factor VIII levels in the blood rose from lower than 1% to a maximum of 20 % of normal factor VIII activity. These levels of activity corrected the bleeding tendency of the hemophilia A mice, thus protect
'"/>

Contact: Leslie H. Lang
LLANG@MED.UNC.EDU
919-843-9687
University of North Carolina School of Medicine
8-Jun-2002


Page: 1 2

Related biology news :

1. Targeted therapy knocks out pediatric brain cancer in mice
2. New anti-inflammatory strategy for cancer therapy identified by UCSD researchers
3. Columbia research to examine gene influence on severity of peridontal disease, therapy response
4. Case for IBD combination therapy comes from research at Baylor, MIT and Hebrew University
5. The search for a kinder, gentler chemotherapy
6. Embryonic stem cell therapy shows steady benefits in rebuilding infarcted heart
7. Retroviral gene therapy? ASLV, HIV, and MLV show distinct target site preferences
8. Gene changes linked to increased eye pressure may have implications for glaucoma therapy
9. Jefferson Lab detector technology aids development of cystic fibrosis therapy
10. Stroking up cellular therapy
11. Smart gene therapy protects against damage from heart attack

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:1/20/2019)... ... January 17, 2019 , ... ... metabolism and distribution has been documented for many years. These observed differences may ... of adverse event profiles in patients residing in different geographic regions. This created ...
(Date:1/14/2019)... SUNNYVALE, Calif. (PRWEB) , ... January 14, 2019 ... ... a proprietary technology to detect precancer and cancer cells in blood, will present ... revealing significant advancement in the fight against colorectal cancer. The results from this ...
(Date:1/10/2019)... Va. (PRWEB) , ... January 09, 2019 , ... Dr. ... Ottawa Heart Institute in Ottawa, Ontario, Canada. He is also Director of its ... Department of Medicine, in the Department of Radiology, and in the Department of Cellular ...
Breaking Biology News(10 mins):
(Date:1/20/2019)... ... 17, 2019 , ... Consumers are directing healthcare in many ... That's changing as many services now exist for patients to order tests without ... $208 million in 2018, according to Kalorama Information’s new report, The Direct-To-Consumer ...
(Date:1/15/2019)... ... 2019 , ... Personalized treatment plans may extend life expectancy for early-stage kidney ... study published in the journal Radiology. , Kidney, or renal, tumors are often ... procedure in which the tumor and part of the kidney are removed. However, some ...
(Date:1/10/2019)... ... January 08, 2019 , ... Rascal was adopted from a local shelter when he was ... mixed breed.” Unfortunately, just six weeks after he was adopted, he tore his right ... to form in the joint after such a traumatic injury. , Sure enough, when Rascal ...
(Date:1/8/2019)... ... January 08, 2019 , ... The American Society of Gene ... named by a selection committee made up of industry leaders identified by the ASGCT ... grants are mentored awards created to support ASGCT members designing transformative pilot studies in ...
Breaking Biology Technology:
Cached News: