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:5/22/2015)... OXFORD, CT , May 22, 2015  NXT-ID, Inc. ... from June 1st, it will be ending its promotional pricing ... www.wocketwallet.com . Laurence Savin , Head of ... our exclusive promotional pricing until the end of May. To ... at wocketwallet.com/invite and when we open the store on June ...
(Date:5/20/2015)... , May 20, 2015 /PRNewswire/ ... http://www.researchandmarkets.com/research/h8t4x6/5year ) has announced the addition ... the Global Iris Recognition Market"  report ... , ,This research service analyses trends ... the government and commercial sectors, globally. ...
(Date:5/19/2015)... , May 19, 2015 ... has announced the addition of the  "Genetic ... offering.  ,     (Logo: http://photos.prnewswire.com/prnh/20130307/600769) , ,A recent ... an in-depth analysis of the current and ... of gene-based tests, their working principles and ...
Breaking Biology News(10 mins):NXT-ID, Inc Announces Retail Price For Wocket Smart Wallet And Extends Discounted Promotional Price Till End Of May 2NXT-ID, Inc Announces Retail Price For Wocket Smart Wallet And Extends Discounted Promotional Price Till End Of May 3Global Iris Recognition Market Report 2015 - 5-Year Opportunity Analysis 2Global Genetic Testing Market Outlook 2018 2
(Date:5/26/2015)... May 26, 2015 Tianyin Pharmaceutical Inc. (NYSE ... in the patented biopharmaceutical, modernized traditional Chinese medicine (mTCM), ... the Company,s Annual Meeting of Shareholders for fiscal year ... held at the Company,s office located in ... 9:00 AM (local time in Chengdu, China ...
(Date:5/26/2015)... 26, 2015 Pharmaco-Kinesis Corporation (PKC) announced today ... at Shutters on the Beach in Santa ... of the Company,s innovative Metronomic Biofeedback Pump (MBP), a ... chemotherapy over time to a target site of the ... biofeedback system in real-time.  ...
(Date:5/26/2015)... May 26, 2015 Oramed Pharmaceuticals ... clinical-stage pharmaceutical company focused on the development of oral ... the study protocol for the company,s Phase IIb trial ... Food and Drug Administration (FDA). The submission was made ... The Phase IIb study of ORMD-0801 for ...
(Date:5/26/2015)... (PRWEB) May 26, 2015 Just the ... a long-standing unmet biomedical need does not guarantee immediate ... problem that has seen many prior failures is often ... acceptance and use is an irony that is often ... small, new start-up company, Asymmetrex has found ...
Breaking Biology Technology:TPI Adjourned Its 2014 Annual Shareholder Meeting and Received Notice of Non-Compliance with NYSE MKT Continued Listing Requirements 2TPI Adjourned Its 2014 Annual Shareholder Meeting and Received Notice of Non-Compliance with NYSE MKT Continued Listing Requirements 3Pharmaco-Kinesis Corporation Convenes Third Neuro-Oncology Clinical Advisory Board Meeting 2Pharmaco-Kinesis Corporation Convenes Third Neuro-Oncology Clinical Advisory Board Meeting 3Pharmaco-Kinesis Corporation Convenes Third Neuro-Oncology Clinical Advisory Board Meeting 4Pharmaco-Kinesis Corporation Convenes Third Neuro-Oncology Clinical Advisory Board Meeting 5Oramed Submits Protocol to U.S. FDA for Its Phase IIb Oral Insulin Study 2Oramed Submits Protocol to U.S. FDA for Its Phase IIb Oral Insulin Study 3In August Asymmetrex Will Present Details of Its Crowdsourcing Campaign to Determine the Extent of Application of New Technologies for Counting Adult Tissue Stem Cells 2In August Asymmetrex Will Present Details of Its Crowdsourcing Campaign to Determine the Extent of Application of New Technologies for Counting Adult Tissue Stem Cells 3
Cached News: