Navigation Links
Genome editing, a next step in genetic therapy, corrects hemophilia in animals
Date:6/26/2011

advantage over conventional gene therapy techniques that may randomly deliver a replacement gene into an unfavorable location, bypassing normal biological regulatory components controlling the gene. This imprecise targeting carries a risk of "insertional mutagenesis," in which the corrective gene causes an unexpected alteration, such as triggering leukemia.

In hemophilia, an inherited single-gene mutation impairs a patient's ability to produce a blood-clotting protein, leading to spontaneous, sometimes life-threatening bleeding episodes. The two major forms of the disease, which occurs almost solely in males, are hemophilia A and hemophilia B, caused respectively by a lack of clotting factor VIII and clotting factor IX. Patients are treated with frequent infusions of clotting proteins, which are expensive and sometimes stimulate the body to produce antibodies that negate the benefits of treatment.

In the current study, the researchers used genetic engineering to produce mice with hemophilia B, modeling the disease in people. Before treatment, the mice had no detectable levels of clotting factor IX.

Previous studies by other researchers had shown that ZFNs could accomplish genome editing in cultured stem cells that were then injected into mice to treat sickle cell disease. However, this ex vivo approach is not feasible for many human genetic diseases, which affect whole organ systems. Therefore the current study tested whether genome editing was effective when directly performed in vivo (in a living animal).

High and colleagues designed two versions of a vector, or gene delivery vehicle, using adeno-associated virus (AAV). One AAV vector carried ZFNs to perform the editing, the other delivered a correctly functioning version of the F9 gene. Because different mutations in the same gene may cause hemophilia, the process replaced seven different coding sequences, covering 95 percent of the disease-carrying mutations in hemophilia B.'/>"/>

Contact: John Ascenzi
ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
Source:Eurekalert  

Page: 1 2 3

Related biology news :

1. The human genome -- now on an iPad near you
2. The complete map of the Germany E. coli O104 genome released
3. Entomologists launch the 5,000 Insect Genome Project (i5k)
4. Genome offers clue to functions of destructive wheat fungus
5. Genome sequence could reveal Achilles heels of important wheat disease
6. Gerry A. Higgins, Ph.D. Joins GenomeQuest to Foster Development of Whole Genome Diagnostics
7. Einstein offers easy-to-use genome analyzer to scientific community
8. BGI releases a complete de novo E. coli O104 genome and details of their detection kit
9. BGI sequences genome of the deadly E. coli in Germany and reveals new super-toxic strain
10. Of mice and men: UNC-led team solves mouse genome dilemma
11. Comparison of genomes of plant parasites provides solid clues for response
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Genome editing, a next step in genetic therapy, corrects hemophilia in animals
(Date:12/22/2014)... 2014  The 2014 Holiday Season may be ... Acuity Market Intelligence reports that the long anticipated ... Acuity forecasts that intensifying demand for smart phones, ... will drive a global market of 2.5 billion ... 2020.   According to Maxine ...
(Date:12/17/2014)... 15, 2014 Research and Markets ... the "Samsung Galaxy S5 - Home Button ... their offering. http://photos.prnewswire.com/prnh/20130307/600769 ... sensing technology than the iPhone 5S, Samsung introduces ... its product. The Galaxy S5 home ...
(Date:12/17/2014)... -- Automation is fundamentally transforming the travel experience and ... borders. Over the past decade, ePassports, biometric readers, ... to self process through border control via eGates ... increasing number of airports, seaports, and land borders ... Maxine Most , Principal at Acuity Market ...
Breaking Biology News(10 mins):First Season of Holiday Shopping with Mobile Biometric Payments Wraps Up With a Present for Biometrics Industry: A Rosy Forecast for More Than 2 Billion Users of 4.8 Billion Mobile Biometric Devices by 2020 2Samsung Galaxy S5 - Home Button Synaptics Fingerprint Sensor Technology Analysis 2Automated Border Control (ABC) Transforms the Global Travel Experience With More Than 2500 ABC eGates and APC Kiosks Deployed In Airports, Seaports, and Land Borders Worldwide 2
... is not a steady march towards ever more sophisticated beings ... pointless, according to findings published by a team of researchers ... Department of Biochemistry. "Aristotle was the first to classify organisms ... of evolution continued this idea, with the concept of a ...
... Green technologies developed at The University of Nottingham have ... aimed at reducing global warming and tackling climate change. ... Rushlight Awards, attracting two prizes for innovations that will ... the developing world and help to make homes of ...
... are related to complex species including vertebrates (such as humans) ... an evolutionary link between simple animals such as jellyfish and ... implies that they have not always been as simple as ... and Acoelomorpha are very simple animals ...
Cached Biology News:New evolutionary research disproves living missing link theories 2Nottingham success in green technologies awards 2Nottingham success in green technologies awards 3Simple marine worms distantly related to humans 2
(Date:12/22/2014)... VIEJO, Calif. , Dec. 22, 2014  ( ... that CoPatient, an online medical bill review and advocacy ... offering in the FREE WellCard Savings discount health services ... medical bill that was more than they expected to ... members reduce health care costs, WellCard Savings is pleased ...
(Date:12/22/2014)... Fertility Associates of Memphis has announced a ... a network of more than 20 contributing fertility ... fast access to a national pool of frozen ... Associates of Memphis is the only fertility clinic in ... Dr. William Kutteh , director of Fertility Associates ...
(Date:12/19/2014)... BioPlus Specialty Pharmacy (BioPlus), one ... of Nick Maroulis, Pharm.D. to the newly created position ... In this position, Dr. Maroulis will continue to manage ... of our multi-site pharmacies as the company expands. Dr. ... that time he has served in many different capacities ...
(Date:12/19/2014)... 19, 2014 Bina Technologies, Inc. (Bina, ... they have been acquired by Roche (SIX: RO, ROG; ... held company that provides a big data platform for ... data for the academic and translational research markets.  Bina ... will continue to focus on development of their innovative ...
Breaking Biology Technology:WellCard Savings Announces CoPatient Medical Bill Review Service for Members 2Fertility Associates of Memphis Announces Partnership with Donor Egg Bank USA 2Fertility Associates of Memphis Announces Partnership with Donor Egg Bank USA 3BioPlus Specialty Pharmacy Promotes Dr. Nick Maroulis to Vice President of Specialty Pharmacy Services 2Bina Technologies is acquired by Roche. 2Bina Technologies is acquired by Roche. 3
... (AIM: NWBS and NWBT; OTC Bulletin Board: NWBO) (the,"Company") ... Rajhi Holdings,through the subscription of 1,000,000 new shares in ... share. The new stock is expected to be ... shares will not be tradable on the,Company,s OTC bulletin ...
... Relations, a Top 25 independent PR agency announced today the ... Group and the Yan Center for Corrective & Cosmetic Surgery, ... clientele. Both medical centers have tasked 5WPR to proactively direct ... Kent Holtorf and the Yan Center have selected 5W PR ...
... iCo Therapeutics Inc. (TSX-V: ICO) is pleased to announce ... financing provides the company with sufficient capital to develop ... clinical trial. iCo-007 is targeting the treatment of Diabetic ... company with additional working capital. iCo,s partner Isis Pharmaceuticals ...
Cached Biology Technology:Northwest Biotherapeutics Secures $700,000 Equity Financing 2The Holtorf Medical Group and Yan Center for Corrective & Cosmetic Surgery Tap 5W Public Relations 2iCo Therapeutics Closes $1.3 Million Private Placement 2iCo Therapeutics Closes $1.3 Million Private Placement 3
... comprehensive microarray service for systematic discovery ... non-coding RNAs (ncRNAs) using a novel ... which enables highly sensitive and specific ... This comprehensive service includes custom probe ...
... The AccuSpot automates LC micro-fractionation, ... With the AccuSpot system, LC eluent ... onto target plates, in trace amounts. ... target plates for MALDI-TOF-MS measurements. When ...
... Sigmas 10 Capillary Electrophoresis Running Buffer ... and high throughput demands of todays sequencing ... supplier of TBE to the gel-based sequencing ... large scale capacity to meet your buffer ...
... and far-infrared range FT-IR spectrometers give you ... class. Yet all this comes at an ... Having a larger and faster mirror ... conventional FT-IRs provides 300%,larger solid angle of ...
Biology Products: