Navigation Links
Combined stem cell-gene therapy approach cures human genetic disease in vitro
Date:5/31/2009

patients' cells using gene therapy techniques pioneered in Verma's laboratory. They then successfully reprogrammed the repaired cells into induced pluripotent stem (iPS) cells using a combination of transcription factors, OCT4, SOX2, KLF4 and cMYC. The resulting FA-iPS cells were indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.

Since bone marrow failure as a result of the progressive decline in the numbers of functional hematopoietic stem cells is the most prominent feature of Fanconi anemia, the researchers then tested whether patient-specific iPS cells could be used as a source for transplantable hematopoietic stem cells. They found that FA-iPS cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.

"We haven't cured a human being, but we have cured a cell," Belmonte explains. "In theory we could transplant it into a human and cure the disease."

Although hurdles still loom before that theory can become practicein particular, preventing the reprogrammed cells from inducing tumorsin coming months Belmonte and Verma will be exploring ways to overcome that and other obstacles. In April 2009, they received a $6.6 million from the California Institute Regenerative Medicine (CIRM) to pursue research aimed at translating basic science into clinical cures.

"If we can demonstrate that a combined iPSgene therapy approach works in humans, then there is no limit to what we can do," says Verma.


'/>"/>

Contact: Gina Kirchweger
Kirchweger@salk.edu
858-453-410-01340
Salk Institute
Source:Eurekalert  

Page: 1 2

Related biology news :

1. Extended wakefulness, combined with alcohol, severely impairs driving performance
2. Measurement Specialties, Inc. Announces Impact of German Business Tax Reform 2008 Resulting in Lower Combined German Tax Rate - Impacts 2Q08 Reported Financial Results
3. Bee species outnumber mammals and birds combined
4. Sociological research shows combined impact of genetics, social factors on delinquency
5. A combined tooth-venom arsenal revealed as key to Komodo dragons hunting strategy
6. A new radiation therapy treatment developed for head and neck cancer patients
7. St. Jude finds factors that accelerate resistance to targeted therapy in lymphoblastic leukemia
8. UC health news: molecular pathway may predict chemotherapy effectiveness
9. MIT works toward safer gene therapy
10. Intravenous gene therapy protects normal tissue of mice during whole-body radiation
11. Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Combined stem cell-gene therapy approach cures human genetic disease in vitro
(Date:9/15/2014)... lowland tapir populations may continue to drop in French ... the Office National de la Chasse et de la ... Global reviewed data retrieved from camera traps in the ... compared this data to current harvest rates in the ... in French Guiana," said Matthias Tobler, a scientist with ...
(Date:9/15/2014)... INDIANAPOLIS -- Adding a common epilepsy drug to a ... fewer side effects. Moreover, the combination can reduce the ... to a team of pain researchers at Indiana University. ... patients with neuropathic pain, a difficult-to-treat condition often felt ... tissue damage. , "There is a huge unmet need ...
(Date:9/15/2014)... a vital source of energy for both plants and ... way into the cell could lead to the design ... in the amount of fruits and vegetables farmers are ... one of these "pathways" into the cell by piecing ... strand of spider silk. , To determine the size, ...
Breaking Biology News(10 mins):IU study: Combining epilepsy drug, morphine can result in less pain, lower opioid doses 2X-rays unlock a protein's SWEET side 2X-rays unlock a protein's SWEET side 3
... diabetes during pregnancy is directly controlled by serotonin, a ... a neurotransmitter, and is influenced by the amount of ... to new findings of an international team led by ... to simple dietary solutions and possible therapeutics for the ...
... rich? Smithsonian researchers have new evidence that the answer ... to a study published in the journal, Nature ., ... not grow and survive well under their mothers or other ... fellow at the University of WisconsinMilwaukee and the Smithsonian Tropical ...
... available in Spanish . , At a time ... Service (ARS)-funded scientists have contributed to a limited but growing ... cognitive function. Cognitive function is measured by the ... use available information for activities of daily life. Alzheimer,s disease, ...
Cached Biology News:Gestational diabetes linked to serotonin and dietary protein 2Gestational diabetes linked to serotonin and dietary protein 3Biodiversity's holy grail is in the soil 2Vitamin D and mental agility in elders 2
(Date:9/11/2014)... stem cells (hiPSCs), researchers have gained new insight ... altered patterns of neuronal signaling associated with this ... from the hiPSCs of healthy individuals and of ... these stem cells to release neurotransmitters, such as ... are linked to various disorders. By discovering a ...
(Date:9/11/2014)... at Chalmers University of Technology are first to show ... atom. They can thereby demonstrate phenomena from quantum physics ... results will be published in the journal Science ... well known and has been studied extensively in the ... kind of interaction with sound waves has been a ...
(Date:9/11/2014)... paper published this week in Science , the ... Manchester,s Nobel laureate Sir Andre Geim report a material ... applied fields, similar to sailboats driven diagonally to the ... chicken wire made from carbon but with a ... state by placing it on top of boron nitride, ...
(Date:9/11/2014)... Fla. , Sept. 11, 2014 /PRNewswire/ ... anti-infective pharmaceuticals, announced today that the United ... a ,Notice of Allowance, for patent application ... Uses,.  The ,486 patent application relates to ... through gene-silencing and can be designed to ...
Breaking Biology Technology:Stem cells help researchers understand how schizophrenic brains function 2The sound of an atom has been captured 2New species of electrons can lead to better computing 2Lakewood-Amedex Receives Notice Of Allowance From The United States Patent and Trademark Office (USPTO) For A New Patent Covering Its nanoRNA Gene-Silencing Technology 2Lakewood-Amedex Receives Notice Of Allowance From The United States Patent and Trademark Office (USPTO) For A New Patent Covering Its nanoRNA Gene-Silencing Technology 3
... Sector, TROY, Mich., Nov. 10 Automation ... data from its,Fourth Annual Technology Industry Report: Driving ... on Wednesday, Nov. 12 at the,Troy Marriott. The ... regarding Automation Alley,s rapidly growing presence in the,life ...
... -- China Medicine,Corporation (OTC Bulletin Board: CHME) ... and distributor of prescription and over the ... supplements, and medical devices and medical formulations, ... call at 9:00 a.m. Eastern Time,on Thursday, ...
... The West Michigan Science,& Technology Initiative and ... a new medical device company, TransCorp, Inc. TransCorp ... Technology offices in,Grand Valley State University,s Cook-DeVos Center ... and commercialize novel surgical instruments,and implants. Other participants ...
Cached Biology Technology:Automation Alley Releases Report on State of Southeast Michigan's Technology Economy 2Automation Alley Releases Report on State of Southeast Michigan's Technology Economy 3China Medicine Announces Conference Call to Discuss Third Quarter 2008 Results 2West Michigan's Newest Medical Device Company Receives Support from the West Michigan Science & Technology Initiative and EDF Ventures 2
RayBio Human Chemokine Antibody Array 1.1 (8) with Accessory, detects 38 chemokine factors (for lysate) Class: Antibody Array Products Product Group: Antibody Array...
... The CultureWell cell culture chambered ... cultured cells for staining and ... medical-grade silicone gaskets with standard ... sterile and ready to use. ...
Adhesive seal-tab, for HybriWell™ hybridization sealing system *set of 400*...
...
Biology Products: