Navigation Links
Gene therapy for human skin disease produces long-term benefits
Date:12/26/2013

Stem cell-based gene therapy holds promise for the treatment of devastating genetic skin diseases, but the long-term clinical outcomes of this approach have been unclear. In a study online December 26th in the ISSCR's journal Stem Cell Reports, published by Cell Press, researchers evaluated a patient with a genetic skin disorder known as epidermolysis bullosa (EB) nearly seven years after he had undergone a gene therapy procedure as part of a clinical trial. The study revealed that a small number of skin stem cells transplanted into the patient's legs were sufficient to restore normal skin function, without causing any adverse side effects.

"These findings pave the way for the future safe use of epidermal stem cells for combined cell and gene therapy of epidermolysis bullosa and other genetic skin diseases," says senior study author Michele De Luca of the University of Modena and Reggio Emilia.

EB is a painful condition that causes the skin to be very fragile and to blister easily, and it can also cause life-threatening infections. Because there is no cure for the disease, current treatment strategies focus on relieving symptoms. To evaluate stem cell-based gene therapy as a potential treatment, De Luca and his colleagues previously launched a phase I/II clinical trial at the University of Modena and recruited an EB patient named Claudio. The researchers took skin stem cells from Claudio's palm, corrected the genetic defect in these cells, and then transplanted them into Claudio's upper legs.

In the new study, De Luca and his team found that this treatment resulted in long-term restoration of normal skin function. Nearly seven years later, Claudio's upper legs looked normal and did not show signs of blisters, and there was no evidence of tumor development. Remarkably, a small number of transplanted stem cells was sufficient for long-lasting skin regeneration.

Even though Claudio's skin had undergone about 80 cycles
'/>"/>

Contact: Mary Beth O'Leary
moleary@cell.com
617-397-2802
Cell Press
Source:Eurekalert

Page: 1 2

Related biology news :

1. Combination of cell transplantation and gene therapy for Alzheimers disease
2. Blue light phototherapy kills antibiotic-resistant bacteria, according to new studies
3. National award honors CHOP scientist for career work in gene therapy for hemophilia
4. Deep sequencing of breast cancer tumors to predict clinical outcomes after single dose of therapy
5. New gene therapy proves promising as hemophilia treatment
6. How can we improve the efficacy of antipsychotics in the era of personalized pharmacotherapy?
7. T cell immunotherapy: Promising results in children and adults with leukemia
8. International gene therapy trial for bubble boy disease shows promising early results
9. Penn Medicine team reports on study of first 59 leukemia patients who received cell therapy
10. Top 12 Pioneer Awards for seminal work in gene and cell therapy selected by blue ribbon panel
11. Hormone therapy could enhance the therapeutic effect of head and facial bone grafts
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/18/2014)... patients referred for evaluation of suspected genetic conditions, ... 25 percent, including detection of a number of ... disease, according to a study appearing in ... coincide with the American Society of Human Genetics ... or coding regions of thousands of genes simultaneously ...
(Date:10/17/2014)... Researchers from the University of Copenhagen have shown for ... the lungs of Cystic fibrosis patients, giving them the ... growth in chronic infections. , The study also discovered ... fibrosis (CF) patients was halted or slowed down by ... consumed all the oxygen and helped "suffocate" the bacteria, ...
(Date:10/16/2014)... Oregon and Washington land managers have a new synthesis ... eastside moist mixed-conifer (MMC) forests in the two states. ... Forests in Eastern Oregon and Washington: A Synthesis of ... Management, a general technical report published by the U.S. ... response to a request from managers for a synthesis ...
Breaking Biology News(10 mins):Whole-exome sequencing shows potential as diagnostic tool 2Scientists opens black box on bacterial growth in cystic fibrosis lung infection 2New report synthesizes best available science on management of moist mixed-conifer forests 2
... at the Smithsonian and partnering organizations have discovered a remarkably ... the Republic of Palau. This fish exhibits many primitive anatomical ... 800 species of living eels, resulting in its classification as ... The team,s research is published online in the Proceedings ...
... Sons of male mice exposed to prenatal stress are ... in the August 17 issue of The Journal of ... lead to individual differences in stress response that may be ... of Pennsylvania, and colleagues previously found that male mice were ...
... and Switzerland have investigated the remarkable distance that microorganisms ... about their potential to colonise new lands and also ... John Moores University (LJMU), Swiss Federal Institute for Forest, ... Fdrale de Lausanne (EPFL) the University of Neuchtel published ...
Cached Biology News:Scientists discover the most primitive living eel 2Effects of prenatal stress passed across generations in mice 2Life on the wind: Study reveals how microbes travel the Earth 2
(Date:10/20/2014)... , Oct. 20, 2014  GenVec, Inc. (NASDAQ: ... Horovitz , Ph.D., from its board of directors effective on ... August 2003, and served as its chairman from June 2006 ... the Nominating and Corporate Governance and Audit Committees of the ... than a decade of dedicated service to GenVec, and its ...
(Date:10/19/2014)... The Latin American hardware encryption display market ... with analysis and forecast of revenue. This market was ... to reach $2,366.8 million by 2018, at a CAGR ... the TOC of the Latin American hardware encryption market ... provided. It also provides a glimpse of the segmentation ...
(Date:10/19/2014)... The Asia-Pacific Speech Analytics market report defines and ... and forecast of revenue. This market is estimated to ... by 2019, at a Compound Annual Growth Rate (CAGR) ... the TOC of the Asia-Pacific Speech Analytics market report ... It also provides a glimpse of the segmentation in ...
(Date:10/18/2014)... The “Human Insulin Market- by Product Type, ... Human Insulin (Rapid Acting, Long Acting, Premixed)], by ... - Forecast to 2018” provides a detailed overview ... market trends, and strategies impacting the global human ... of the revenue and share analysis. , The ...
Breaking Biology Technology:Zola P. Horovitz To Retire From GenVec Board 2The Latin American hardware encryption display market is expected to reach $2,366.8 million by 2018 - New Report by MicroMarket Monitor 2The Latin American hardware encryption display market is expected to reach $2,366.8 million by 2018 - New Report by MicroMarket Monitor 3The Asia-Pacific Speech Analytics market is estimated to reach $208 million by 2019 - New Report by MicroMarket Monitor 2The Asia-Pacific Speech Analytics market is estimated to reach $208 million by 2019 - New Report by MicroMarket Monitor 3The Asia-Pacific Speech Analytics market is estimated to reach $208 million by 2019 - New Report by MicroMarket Monitor 4Medical Human Insulin Market Is Expected to Reach $42 billion by 2019 - New Research Report by MarketsandMarkets 2Medical Human Insulin Market Is Expected to Reach $42 billion by 2019 - New Research Report by MarketsandMarkets 3Medical Human Insulin Market Is Expected to Reach $42 billion by 2019 - New Research Report by MarketsandMarkets 4Medical Human Insulin Market Is Expected to Reach $42 billion by 2019 - New Research Report by MarketsandMarkets 5
... Informex , the leading meeting place for buyers ... the Global Pharma Sourcing Conference to be held December ... Philadelphia, Pennsylvania. The two day program will be jointly ... address key issues that global chemical and pharmaceutical industry ...
... Corp. (IDC), a privately held biotechnology company developing ... and allergy, announced today that it has entered ... investigate the use of IDC,s Glucopyranosyl Lipid Adjuvant ... in the field of allergy. (Logo: ...
... Inc. (Nasdaq: SGMO ) today reported third quarter ... third quarter ended September 30, 2011, Sangamo reported a consolidated ... to a net loss of $8.7 million, or $0.19 per ... 30, 2011, the Company had cash, cash equivalents and marketable ...
Cached Biology Technology:Informex Introduces a Thorough Global Pharma Perspectives Conference 2Sangamo BioSciences Reports Third Quarter 2011 Financial Results 2Sangamo BioSciences Reports Third Quarter 2011 Financial Results 3Sangamo BioSciences Reports Third Quarter 2011 Financial Results 4Sangamo BioSciences Reports Third Quarter 2011 Financial Results 5Sangamo BioSciences Reports Third Quarter 2011 Financial Results 6
Although each Beacon 2000 System includes one preinstalled sample chamber (size specified at time of order), optional chambers are available and easily retrofit by the user....
Whole blood is collected from healthy guinea pigs of either sex from US source which are free of visible diseases at time of blood collection. Hemolytic titer: CH50 = 435 units...
...
... serial preparation of four ... cytosol/particulate/cytoskeleton/nuclear fractions from one ... can directly be used ... as 2-D gel/Western blot/gel-shift/translocation/enzyme ...
Biology Products: