Navigation Links
Steps towards the use of adult stem cells for gene therapy

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
INSERM (Institut national de la sant et de la recherche mdicale)

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:
(Date:6/25/2016)... ... 25, 2016 , ... Austin residents seeking Mohs surgery services, can now turn ... to Dr. Russell Peckham for medical and surgical dermatology. , Dr. Dorsey brings specialization ... selective fellowship in Mohs Micrographic Surgery completed by Dr. Dorsey was under the direction ...
(Date:6/25/2016)... Beach, CA (PRWEB) , ... June 25, 2016 , ... ... UCLA with Magna Cum Laude and his M.D from the David Geffen School of ... Diego and returned to Los Angeles to complete his fellowship in hematology/oncology at the ...
(Date:6/24/2016)... ... ... A recent article published June 14 on E Online details ... to state that individuals are now more comfortable seeking to undergo not only the ... and cheek reduction. The Los Angeles area medical group, Beverly Hills Physicians (BHP) notes ...
(Date:6/24/2016)... , ... June 24, 2016 , ... ... and Scientific Sessions in Dallas that it will receive two significant new grants ... grants came as PHA marked its 25th anniversary by recognizing patients, medical professionals ...
(Date:6/24/2016)... ... ... Topical BioMedics, Inc, makers of Topricin and MyPainAway Pain Relief Products, join The ‘Business for ... $12 an hour by 2020 and then adjusting it yearly to increase at the same ... wage, assure the wage floor does not erode again, and make future increases more predictable. ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... , June 24, 2016  Collagen Matrix, ... design, development and manufacturing of collagen and mineral ... announced today that Bill Messer has ... Marketing to further leverage the growing portfolio of ... devices. Bill joins the Collagen Matrix ...
(Date:6/24/2016)... Va. , June 24, 2016 The ... set of recommendations that would allow biopharmaceutical ... (HCEI) with entities that make formulary and coverage decisions, ... the "value" of new medicines. The recommendations ... does not appear on the drug label, a prohibition ...
(Date:6/24/2016)... 24, 2016   Pulmatrix, Inc ., (NASDAQ: ... inhaled drugs, announced today that it was added to ... its comprehensive set of U.S. and global equity ... an important milestone for Pulmatrix," said Chief Executive Officer ... of our progress in developing drugs for crucial unmet ...
Breaking Medicine Technology: