HOME >> BIOLOGY >> NEWS
New gene therapy shows promise for hemophiliacs

MINNEAPOLIS / ST. PAUL -- University of Minnesota researchers have demonstrated that it may be possible to treat hemophilia A through the use of human blood outgrowth endothelial cells, or BOECs, as a vehicle for gene therapy. Lead researcher Robert Hebbel, M.D., the George Clark Professor and vice chair for research, department of medicine, reports that the use of BOECs has "resulted in sustained and therapeutic levels of factor 8 (FVIII) in mice and may comprise an effective therapeutic strategy for use in gene therapy for humans with hemophilia A." The results of this gene therapy, published in the January 15 issue of Blood, may pave the way for testing the therapy in humans.

Hemophilia A is an inherited blood disorder that affects one in 5,000 males and results in spontaneous bleeding due to the lack of coagulation factor 8 (FVIII) in the blood. Because hemophilia is such a severe disease, and because it is caused by absence of a replaceable blood protein, there has been a high level of interest in developing a gene therapy approach for its treatment. While there are currently some ongoing Phase I clinical studies of gene therapy for humans for hemophilia B (a related disorder that affects a different coagulation factor), development of gene therapy for hemophilia A has lagged behind. Current clinical therapy generally calls for the replacement of factor VIII after the patient has had some trouble.

The ex vivo process devised by Hebbel utilizes BOECs as carriers for gene therapy. The process begins with a blood sample taken from the patient as a source for autologous BOECs. (This use of autologous cells eliminates the concern for immunological compatibility or disease-tainted blood sources). The BOECs are separated from the blood and cultured in the lab and then transfected with a nonviral plasmid vector carrying the gene for human coagulation factor VIII. After allowing them to further expand, those stably transfected BOECs are then given
'"/>

Contact: Deane Morrison
morri029@umn.edu
612-624-2346
University of Minnesota
18-Jan-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:
TAG: New gene therapy shows promise for hemophiliacs

(Date:12/11/2014)... 2014  Data Sciences International (DSI), the global ... new series of digital telemetry implants to meet ... series, part of the PhysioTel™ Digital platform, was ... physiologic data when incorporating functional endpoints into repeat-dose ... toxicology studies has evolved from short ECG strips ...
(Date:12/10/2014)... DIEGO , Dec. 9, 2014 CIE ... collaboration catalyst that provides the connective tissue that enhances ... enabling them to easily share client-level information; earned a ... to expand to organizations serving seniors aging in community ... Diego" partner on December 11 th 4-6p. ...
(Date:12/5/2014)... , Dec. 4, 2014  Tute Genomics, a leader ... million in Series A1 funding led by UK-based Eurovestech. ... participated in the investment round. "We are ... community adopts next-generation sequencing and seeks new approaches for ... Reid Robison , MD MBA, and CEO of Tute ...
Breaking Biology News(10 mins):New telemetry implants expected to change how large animal toxicology studies are conducted 2Community Information Exchange San Diego Launches Pilot, Secures $1 Million Expansion Grant, and Becomes a "Live Well San Diego" Partner 2Community Information Exchange San Diego Launches Pilot, Secures $1 Million Expansion Grant, and Becomes a "Live Well San Diego" Partner 3Community Information Exchange San Diego Launches Pilot, Secures $1 Million Expansion Grant, and Becomes a "Live Well San Diego" Partner 4Tute Genomics Raises $2.3 Million to Scale Up Cloud-Based Genome Analytics and Accelerate Personalized Medicine 2Tute Genomics Raises $2.3 Million to Scale Up Cloud-Based Genome Analytics and Accelerate Personalized Medicine 3
(Date:12/17/2014)... December 16, 2014 The Activated ... 2013, which is why IBISWorld updated its original industry ... to benefit from an intensified focus on environmental policy. ... new emission standards for power plants and a range ... to IBISWorld Industry Analyst Sarah Kahn, “a range of ...
(Date:12/17/2014)... Washington, USA (PRWEB) December 16, 2014 ... Baum, manager at Philipps- Universität Marburg’s Department of ... and photonics, in the newly released SPIE ... “And never be too shy to ask questions.” ... (science, technology, engineering, and mathematics) occupations ranging from ...
(Date:12/17/2014)... December 17, 2014 Ipsen Biopharmaceuticals, ... IPN; ADR: IPSEY), today announced that ... as Somatuline®) was approved by the U.S. Food ... gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in adult patients with ... metastatic disease to improve progression-free survival (PFS). , ...
(Date:12/17/2014)... This report is a professional ... For an overview analysis, the report introduces quartz ... structure, industry overview, policy analysis, and news analysis, ... has witnessed rapid development with key manufacturers increasing ... report mentions quartz tubing upstream raw materials and ...
Breaking Biology Technology:Activated Carbon Manufacturing in the US Industry Market Research Report Has Been Updated from IBISWorld 2Activated Carbon Manufacturing in the US Industry Market Research Report Has Been Updated from IBISWorld 3Network, Multitask, Persevere: SPIE Women in Optics Planner Subjects Share from Experience 2Network, Multitask, Persevere: SPIE Women in Optics Planner Subjects Share from Experience 3Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 2Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 3Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 4Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 5Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 6Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 7Ipsen Announces the FDA Approval of Somatuline® Depot® (lanreotide) Injection for the Treatment of Gastroenteropancreatic Neuroendocrine Tumors 82020 Quartz Tubing Market: Global Development & Forecasts Analysis Now at DeepResearchReports.com 2
Cached News: