Navigation Links
Steps towards the use of adult stem cells for gene therapy
Date:10/13/2011

This research, published on Oct. 12 on the Nature review website, provides evidence of a major concept could pave the way for the future use of these stem cells to treat humans, through perspective gene therapies. For several years now, scientists have been able to produce cells with stem cell properties, by using specialized and mature cells from our body, such as skin cells. These 'iPS' stem cells are said to be "pluripotent': they can provide specialized cells, upon demand, with the same gene pool as the original cells. iPS cells represent a potential basis for the exploration of several therapeutic areas, particularly transplants or gene therapy. However, to date research conducted on these cells had not provided proof of their potential in vivo efficiency for the aforementioned types of use.

For the first time, researchers from the Sanger Institute and the University of Cambridge (United Kingdom), with collaboration from an Institut Pasteur/Inserm team in France, have demonstrated that the cells derived from iPS stem cells may be used within the framework of gene therapy to help counter pathological effects in a mice model with liver failure.

The researchers focussed on a rare genetic disease affecting the liver. It is caused by a point mutation in the a1-antitrypsin gene, which is essential for hepatic cells to function correctly. Children display varying degrees of mild symptoms (jaundice, abdomen distension, etc.), but, in adulthood, these symptoms may progressively develop into a pulmonary emphysema and cirrhosis, where the only hope of a cure is a liver transplant.

Researchers from the University of Cambridge, directed by Ludovic Vallier and David Lomas, and from the Sanger Institute, coordinated by Allan Bradley, began by sampling patients' skin cells, which were then cultured in vitro for "differentiation" before applying the properties of the pluripotent stem cells: this is the "iPS cells" stage. Through genetic engineering, scientists were then able to correct the mutation responsible for the disease. They then engaged the now "healthy" stem cells in the maturation process, leading them to differentiate to liver cells.

Scientists from the Institut Pasteur and Inserm, led by Hlne Strick-Marchand in the mixed Institut Pasteur/Inserm Innate Immunity unit (directed by James Di Santo), then tested new human hepatic cells thus produced on an animal model afflicted with liver failure. Their research showed that the cells were entirely functional and suited to integration in existing tissue and that they may contribute to liver regeneration in the mice treated.

This groundbreaking work, published in Nature, thus strengthened hopes in scientific and medical communities regarding the use of iPS cells to treat humans.


'/>"/>
Contact: Marion Doucet
marion.doucet@pasteur.fr
01-456-88928
INSERM (Institut national de la sant et de la recherche mdicale)
Source:Eurekalert

Related medicine news :

1. Three steps to unbreakable bones on World Osteoporosis Day
2. Simple Safety Steps Can Make Back to School a Breeze
3. Steps Outlined to Screen All Newborns for Heart Defects
4. Kids Urged to Take Steps to Avoid Spreading Illness at School
5. Next steps for Tenofovir gel: CONRAD and TIA sign license agreement
6. Take Steps to Prevent Pulmonary Embolisms
7. Paraplegic man stands, steps with assistance and moves his legs voluntarily
8. Five Steps to a Healthier Heart
9. Early Steps Toward an Alzheimers Blood Test
10. Take Steps to Avoid Winters Indoor Allergies
11. A few steps could lead to big gains for hospitalized seniors
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/13/2017)... (PRWEB) , ... October 13, 2017 , ... ... locations throughout Arkansas that offers insurance and financial preparation services, is providing an ... Rock City Rescue organization. , Rock City Rescue is a locally recognized nonprofit ...
(Date:10/13/2017)... ... October 13, 2017 , ... PurhealthRX , a leading Health ... technology. Applying the Purzorb™process to full spectrum CBD oil will revolutionize the rapidly growing ... form that can be easily incorporated into liquid products, while reducing costs to end ...
(Date:10/13/2017)... ... October 13, 2017 , ... The Visiting Nurse Association ... Featuring a collection of specialty vendors and unique items from across the nation, this ... health and wellness services offered by the VNA. The boutique will be open ...
(Date:10/13/2017)... ... October 13, 2017 , ... Coveros, a leader in agile ... a contract by the Center for Medicare and Medicaid Services (CMS). The Enterprise ... use of Agile methodologies in a consistent and high value manner across CMS ...
(Date:10/12/2017)... ... October 12, 2017 , ... Planet Fitness, one of the largest ... its plans to open a flagship location in Covington, LA at 401 N. U.S. ... Go store next to Office Depot in the Holiday Square shopping center. Its location ...
Breaking Medicine News(10 mins):
(Date:10/12/2017)... , Oct. 12, 2017 West ... in innovative solutions for injectable drug administration, today announced ... the market opens on Thursday, October 26, 2017, and ... results and business expectations at 9:00 a.m. Eastern Time. ... or 253-336-8738 (International). The conference ID is 94093362. ...
(Date:10/11/2017)... -- Caris Life Sciences ® , a leading innovator in ... medicine, today announced that St. Jude Medical Center,s Crosson ... as its 17 th member. Through participation with ... Institute will help develop standards of care and best ... cancer treatment more precise and effective. ...
(Date:10/11/2017)... 2017  Hill-Rom Holdings, Inc. ("Hill-Rom") (NYSE: HRC), today ... Las Piedras, Puerto Rico , where ... Following a comprehensive ... minor structural damage, temporary loss of power and minimal ... completed, manufacturing operations have resumed, and the company expects ...
Breaking Medicine Technology: