Navigation Links
Researchers pioneer new gene therapy technique using natural repair process

Harnessing the strength of a natural process that repairs damage to the human genome, a researcher from UT Southwestern Medical Center has helped establish a method of gene therapy that can accurately and permanently correct mutations in disease-causing genes.

The findings are available online in Nature.

By artificially initiating a DNA repair process known as homologous recombination, Dr. Matthew Porteus of UT Southwestern, working with scientists from Richmond, Calif.-based Sangamo Biosciences, was able to replace a mutated version of the gene that encodes a portion of the interleukin-2 receptor (IL-2R) in human cells, restoring both gene function and the production of the IL-2R protein. Mutations in the IL-2R gene are associated with a rare immune disease called severe combined immunodeficiency disease, or SCID. Children with SCID are unable to successfully fight off infections, and must constantly live in a germ-free environment. Their lifespans are usually shortened by systemic infection, and while bone marrow transplants can be used to treat the disease, they are not always successful.

"SCID is ideal for this sort of therapy because you only need to correct the defect in a small number of immune cells to fix the problem," said Dr. Porteus, assistant professor of pediatrics and biochemistry at UT Southwestern. "This is called selective advantage; the healthy cells grow and divide preferentially over the mutant ones."

Previous gene therapy attempts for SCID have been only moderately successful because of technical difficulties in the delivery method. In one instance, the correct IL-2R gene was delivered to mutant cells of SCID children by a disabled virus, but some subsequently developed leukemia because the virus inadvertently turned on a cancer gene.

Dr. Porteus' strategy differs fundamentally from previous gene therapies because it essentially replicates the natural process, which is more accurate. This accuracy means th
'"/>

Source:University of Texas Southwestern Medical Center at Dallas


Page: 1 2 3

Related biology news :

1. Researchers discover way to make cells in the eye sensitive to light
2. Researchers find how protein allows insects to detect and respond to pheromones
3. Researchers Uncover Key Step In Manufacture of Memory Protein
4. Researchers reveal the infectious impact of salmon farms on wild salmon
5. Researchers identify target for cancer drugs
6. Researchers discover molecule that causes secondary stroke
7. Researchers find missing genes of ancient organism
8. Researchers trace evolution to relatively simple genetic changes
9. Researchers add new tool to tumor-treatment arsenal
10. UF Researchers Map Bacterial Proteins That Cause Tooth Loss
11. VCU Researchers Identify Networks Of Genes Responding To Alcohol In The Brain
Post Your Comments:
(Date:1/22/2015)... , Jan. 13, 2015 /PRNewswire/ - Today, FindBiometrics, the leading source ... launch of its new website design. "When we ... infancy", says Peter O,Neill , founder and CEO of ... industry needs involvement from the key players on a very ...
(Date:1/22/2015)... Conn. , Jan. 20, 2015 NXT-ID, Inc. (NASDAQ: ... mobile commerce market, reports on the recent success of the Wocket™ smart ... Wocket smart wallet was named as one of the "11 ... the "5 Best Products Launched At CES So Far" by Newseveryday.com and ...
(Date:1/22/2015)...   EyeLock, Inc. , a market leader of iris-based identity ... to the new role of Senior Vice President of ... of mobile platforms and wearable solutions for EyeLock,s technologies. Gerber ... the semiconductor industry to his role at EyeLock, with expertise ...
Breaking Biology News(10 mins):Identity Industry Leading Publication Debuts New Look As Biometrics Hit Mainstream 2CES Response for NXT-ID's Wocket Smart Wallet Kicks Off 2015 2CES Response for NXT-ID's Wocket Smart Wallet Kicks Off 2015 3EyeLock Names Steve Gerber as Senior Vice President of Mobile and Wearables 2
... Efforts to understand how the aging process affects the ... and older adults. "Everybody ages differently. By looking ... vascular health, we begin to understand that preventable factors ... State University psychology doctoral student Andrew Bender, lead author ...
... HOUSTON (May 8, 2012) The best doctors strive ... Rice University students to help him do so in the ... a prototype device to literally lift the weight from obese ... breathing. The respiratory assist device built by Team R-Aides ...
... the final stages of approving the use of the country,s ... guidance from scientists funded by PETA. The 3T3 Neutral Red ... when they come into contact with sunlight and is already ... scheduled to be accepted in China by late summer. Until ...
Cached Biology News:WSU researchers say genes and vascular risk modify effects of aging on brain and cognition 2WSU researchers say genes and vascular risk modify effects of aging on brain and cognition 3Rice students work on weighty problem for doctors 2Rice students work on weighty problem for doctors 3Rice students work on weighty problem for doctors 4
(Date:1/22/2015)... 22, 2015   Cypher Genomics, Inc., the ... SQNM ), the leading molecular diagnostics ... generation noninvasive prenatal tests (NIPT). Through this agreement, ... Mantis™, to advance analysis of clinically-relevant fetal sub-chromosomal ...
(Date:12/24/2014)... Dec. 24, 2014  United Therapeutics Corporation (NASDAQ: ... MDT ) has submitted a pre-market approval application ... the use of Medtronic,s SynchroMed ® II implantable ... use with United Therapeutics, Remodulin ® (treprostinil) Injection ...
(Date:12/24/2014)... GMO corn cases filed across the United States, ... of being consolidated in a Kansas federal court for pretrial ... 162 Corn Litigation, MDL No. 2591 in the U.S. District ... Syngenta GMO corn multidistrict litigation (MDL) has been handed over ...
(Date:12/24/2014)... December 23, 2014 The report ... its definition, classification, application and industry overview. This ... product cost structure. Production is separated by regions, ... raw materials, equipment, downstream client survey, marketing channels, ...
Breaking Biology Technology:Cypher Genomics and Sequenom Announce Development Agreement 2Cypher Genomics and Sequenom Announce Development Agreement 3Cypher Genomics and Sequenom Announce Development Agreement 4United Therapeutics Announces Submission Of Pre-Market Approval Application For Implantable Drug Infusion System To Deliver Remodulin 2United Therapeutics Announces Submission Of Pre-Market Approval Application For Implantable Drug Infusion System To Deliver Remodulin 3United Therapeutics Announces Submission Of Pre-Market Approval Application For Implantable Drug Infusion System To Deliver Remodulin 4Carey Danis & Lowe Reports on Syngenta GMO Corn Transfer Order 2Worldwide Methyl Mercaptan Market 2015-2020 Forecasts on Development & Trends Now Available at ReportsnReports.com 2
... somehow survived where other traditional marketing tools and old ... must face harsher realities once again, all to ensure ... Web sites are simply doomed. While billions of Web ... clash into the billions of surfing customers and simply ...
... Ted Kanavas, R-Brookfield, called on the Legislature and the ... Development Fund. , ,The fund would use existing information ... Administration and segregate a portion of the funds for ... would not flow into this fund. , ,This fund ...
... Corporation and First Data Corporation jointly ... provides for the purchase of substantially all of the ... approximately $610 million in cash. As the majority shareholder ... million and NYCE will become a Metavante subsidiary. , ...
Cached Biology Technology:The mysteries and the future of Web sites 2The mysteries and the future of Web sites 3The mysteries and the future of Web sites 4Metavante and First Data to buy PIN-debit company NYCE 2
...
... atoms at the 3, 3', 4, and ... as an internal standard for the quantification ... PGE1 is the theoretical cyclooxygenase metabolite of ... undetectable in the plasma of normal humans ...
Acrylamide, 500 g. Assay: > 99.9%, acrylic acid: < 0.001%. Conductivity (35%): < 2 umhos/cm.Risks: 45, 46, 48, 23, 24, 25 ; Safety Precautions: 53, 44. Category: Nucleotides & Enzymes & Biochemicals...
Direct ELISA Assay Diluent, 10 L...
Biology Products: