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

p>

The researchers injected mice with the gene therapy vector, which was designed to travel to the liverwhere clotting factors are produced. The mice that received the ZFN/gene combination then produced enough clotting factor to reduce blood clotting times to nearly normal levels. Control mice receiving vectors lacking the ZFNs or the F9 minigene had no significant improvements in circulating factor or in clotting times.

The improvements persisted over the eight months of the study, and showed no toxic effects on growth, weight gain or liver function, clues that the treatment was well-tolerated.

"We established a proof of concept that we can perform genome editing in vivo, to produce stable and clinically meaningful results," said High. "We need to perform further studies to translate this finding into safe, effective treatments for hemophilia and other single-gene diseases in humans, but this is a promising strategy for gene therapy." She continued, "The clinical translation of genetic therapies from mouse models to humans has been a lengthy process, nearly two decades, but we are now seeing positive results in a range of diseases from inherited retinal disorders to hemophilia. In vivo genome editing will require time to mature as a therapeutic, but it represents the next goal in the development of genetic therapies."


'/>"/>

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:11/21/2014)... MOUNTAIN VIEW, Calif. , Nov. 20, 2014 /PRNewswire/ ... for higher driver efficiency are piloting the North American ... the number of accidents growing, gesture recognition systems that ... will make a mark in the industry. ... of the Automotive Gesture Recognition Market in ...
(Date:11/18/2014)... BOSTON , Nov. 17, 2014   ... HealthCare and EMC collaborate to develop The Partners ... , The Partners Data Lake will allow ... to improve diagnostics, treatment and the lives of ... support research and clinical activities across the Partners ...
(Date:11/12/2014)... Nov. 12, 2014 Crossmatch™, a leading provider ... U.are.U ® fingerprint readers have been deployed throughout ... Central Mexico . The bakery chain implemented ... payroll issues caused by employees clocking in for each ... fingerprint readers, Montparnasse relied on paper timecards and a ...
Breaking Biology News(10 mins):Gesture Recognition Intensifies with the Rise of In-car Smartphone Integration, Says Frost & Sullivan 2Gesture Recognition Intensifies with the Rise of In-car Smartphone Integration, Says Frost & Sullivan 3Gesture Recognition Intensifies with the Rise of In-car Smartphone Integration, Says Frost & Sullivan 4Partners HealthCare and EMC Unite to Solve Health Care's Toughest Challenges Using Big Data 2Partners HealthCare and EMC Unite to Solve Health Care's Toughest Challenges Using Big Data 3Partners HealthCare and EMC Unite to Solve Health Care's Toughest Challenges Using Big Data 4Partners HealthCare and EMC Unite to Solve Health Care's Toughest Challenges Using Big Data 5Montparnasse Pasteleria Achieves Time and Attendance Transparency with Crossmatch U.are.U Fingerprint Readers 2
... have found a way to project future habitat ... for threatened biodiversity. Associate Professor Grant Wardell-Johnson ... Institute for Biodiversity and Climate, along with lead ... now at Wageningen University in the Netherlands, developed ...
... on undersea vehicles that have fish-like sensations, advanced ship ... of Naval Research (ONR) on Dec. 23 learned they ... and engineers. The researchers have been selected for ... and will be honored at a ceremony at the ...
... in a quest to correct a faulty chromosome through cellular ... used stem cells to correct a defective "ring chromosome" with ... chromosome abnormalities that give rise to birth defects, mental disabilities ... be possible to use this approach to take cells from ...
Cached Biology News:Safe havens revealed for biodiversity in a changed climate 2White House lauds ONR-funded researchers for early success 2Nature study discovers chromosome therapy to correct a severe chromosome defect 2Nature study discovers chromosome therapy to correct a severe chromosome defect 3
(Date:11/24/2014)... 24, 2014 Continental Clinical Solutions, ... into clinical trials at its mid-Atlantic late-phase research ... was created with the idea of closing patient ... role in medical advances," said Continental's CEO, ... to the immortal cell line of Henrietta Lacks, ...
(Date:11/24/2014)... 24, 2014 The new video, ... METTLER TOLEDO demonstrates how to reduce the number ... and sample panel design can be complicated, requiring ... protect against corrosion and deposition. Dedicated single and ... amount of panel space. Additionally, installing and maintaining ...
(Date:11/24/2014)... Senior Vice President, General Manager for Clinical ... acumen Elsevier , a world-leading provider ... congratulates Diane Bartoli , Senior Vice President and ... Solutions, for being recognized in the 13 th ... Profiles in Diversity Journal ® . ...
(Date:11/22/2014)... Two new educational webinars hosted by ... ALS research and how the pharmaceutical industry can make life ... Challenge: Where Does the ALS Money Go? , Featuring guest ... Time: 1:30pm ET, Register to attend , This ... Cudkowicz, Julianne Dorn Professor of Neurology at Massachusetts General Hospital ...
Breaking Biology Technology:At Continental Clinical Solutions, CEO Saleh Stevens Focuses on Diversity in Clinical Trial Recruitment at Maryland Site 2At Continental Clinical Solutions, CEO Saleh Stevens Focuses on Diversity in Clinical Trial Recruitment at Maryland Site 3New Video Shows How to Simplify Cycle Chemistry Sample Panels to Save Time and Costs 2Elsevier Clinical Solutions' Diane Bartoli Featured In Profiles in Diversity Journal's 13th Annual Women Worth Watching Issue 2Elsevier Clinical Solutions' Diane Bartoli Featured In Profiles in Diversity Journal's 13th Annual Women Worth Watching Issue 3DrugDev Webinars in December to Explore What Comes Next for ALS Research and How We Can Make Life Easier for Clinical Sites 2
... SHENYANG, China, May 3, 2011 /PRNewswire-Asia-FirstCall/ -- 3SBio ... "the Company"), a leading China-based biotechnology company focused ... today announced that it will release its first ... stock market  closes on Wednesday, May 11, 2011 ...
... 3, 2011 At this year,s American Academy of AntiAging ... named the leading factors in premature aging and disease. AGEs ... result of sugar that attaches to a protein or lipid ... AGEs are completely irreversible—and cause accelerated damage to the tissues ...
... Adeona Pharmaceuticals, Inc. (AMEX: AEN ), a ... diseases, announced today that after scientific review, the ongoing ... for multiple sclerosis (MS) has received an additional $1,594,553 ... Institute of Neurological Disorders and Stroke (NIH/NINDS). The clinical ...
Cached Biology Technology:3SBio Inc. Schedules Unaudited First Quarter 2011 Results 2At This Year's American Academy of AntiAging (A4M) Conference, Glycation and AGEs (Advanced Glycation End Products) Were Named the Leading Factors in Premature Aging and Disease 2Adeona's Multiple Sclerosis Clinical Trial Receives $1,594,553 Grant 2Adeona's Multiple Sclerosis Clinical Trial Receives $1,594,553 Grant 3Adeona's Multiple Sclerosis Clinical Trial Receives $1,594,553 Grant 4
... isoprostanes produced from arachidonic acid during lipid peroxidation. ... rat. 8-iso PGE2 inhibits U-46619 or I-BOP-induced platelet ... µM, respectively. When infused into the renal artery ... mg/kg/min, 8-iso PGE2 decreases the GFR and renal ...
... centrifuge offers both high-speed and high-volume ... of rotor options, making it ideal for ... Maximum Versatility , High-speed (up to ... or high-volume (4 x 250mL) swinging bucket ...
...
... submarine gel systems used gasketed gel trays. Turn the ... tank. Turn it back and run the gel - ... Turn & Cast tray is UV transparent. Each tray ... the tray and one in the center. This allows ...
Biology Products: