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

LA JOLLA, CAA study led by researchers at the Salk Institute for Biological Studies, has catapulted the field of regenerative medicine significantly forward, proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology. The study, published in the May 31, 2009 early online edition of Nature, is a major milestone on the path from the laboratory to the clinic.

"It's been ten years since human stem cells were first cultured in a Petri dish," says the study's leader Juan-Carlos Izpisa Belmonte, Ph.D., a professor in the Gene Expression Laboratory and director of the Center of Regenerative Medicine in Barcelona (CMRB), Spain. "The hope in the field has always been that we'll be able to correct a disease genetically and then make iPS cells that differentiate into the type of tissue where the disease is manifested and bring it to clinic."

Although several studies have demonstrated the efficacy of the approach in mice, its feasibility in humans had not been established. The Salk study offers the first proof that this technology can work in human cells.

Belmonte's team, working with Salk colleague Inder Verma, Ph.D., a professor in the Laboratory of Genetics, and colleagues at the CMRB, and the CIEMAT in Madrid, Spain, decided to focus on Fanconi anemia (FA), a genetic disorder responsible for a series of hematological abnormalities that impair the body's ability to fight infection, deliver oxygen, and clot blood. Caused by mutations in one of 13 Fanconi anemia (FA) genes, the disease often leads to bone marrow failure, leukemia, and other cancers. Even after receiving bone marrow transplants to correct the hematological problems, patients remain at high risk of developing cancer and other serious health conditions.

After taking hair or skin cells from patients with Fanconi anemia, the investigators corrected the defective gene in the 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  

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:2/9/2016)... 2016 Vigilant Solutions announces today that an agency ... develop a lead in a difficult homicide case. The agency ... locate the suspect vehicle. Due to the ongoing investigation, the ... omitted at the agency,s request. --> ... victim was found deceased at an intersection here in the ...
(Date:2/3/2016)... February 3, 2016 --> ... research report "Automated Fingerprint Identification System Market by Component ... Application (Banking & Finance, Government, Healthcare, and Transportation) and ... the market is expected to be worth USD 8.49 ... between 2015 and 2020. The transformation and technology evolution ...
(Date:2/2/2016)... , Feb. 2, 2016 Checkpoint Inhibitors ... Rising Market Are you interested in the ... revenues for checkpoint inhibitors. Visiongain,s report gives those ... product and national level. Avoid falling behind ... progress, opportunities and revenues those emerging cancer therapies ...
Breaking Biology News(10 mins):
(Date:2/11/2016)... ... February 11, 2016 , ... ... new stem cell treatment clinic in Quito, Ecuador. The new facility will provide ... applications to patients from around the world. , The new GSCG clinic ...
(Date:2/10/2016)... ASAE is introducing a hybrid membership model ... the option of joining or renewing through an organizational ... staff size, every employee in any size association or ... all available member benefits.   John H. ... options will allow organizations of any size and their ...
(Date:2/10/2016)... ... 2016 , ... Benchmark Research, a fully-integrated network of ... principal investigators (PI) to the roles of Chief Medical Officer, Clinical Research and ... Chu, a Benchmark Research PI in the Austin office, will assume the role ...
(Date:2/10/2016)... (PRWEB) , ... February 10, 2016 , ... ... now available on Microsoft Azure. On Azure, Arvados provides capabilities for managing and ... clear demand for Microsoft Azure from major institutions collecting and analyzing genomic data,” ...
Breaking Biology Technology: