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:4/23/2014)... Alzheimer,s disease is the first cause of dementia and ... effective cure has yet been found. One of the ... the cellular mechanisms which cause alterations in nerve transmissions ... of the disease., Researchers from the Institute of Neuroscience ... cellular mechanism involved in memory consolidation and were able ...
(Date:4/23/2014)... University of Liverpool is leading a 2 million Food ... norovirus in food premises and industry workers. , ... In 2012 a batch of frozen strawberries infected ... in the authorities, understanding of which strains cause infection ... the bacteria. , Researchers will produce data ...
(Date:4/22/2014)... the University of Montana,s Wildlife Biology Program, co-wrote a research ... National Academy of Sciences on how streamwater chemistry varies ... Lowe and ... of Washington, the Cary Institute of Ecosystem Studies, the University ... examined 664 water samples collected every 10 meters along 32 ...
Breaking Biology News(10 mins):Loss of memory in Alzheimer's mice models reversed through gene therapy 2New research focuses on streamwater chemistry, landscape variation 2
... on a unique vertebrate called the sea lamprey shows that ... development. These genes are paradoxically lost all throughout the developing ... or PGCs. The PGCs can be thought of as embryonic ... generation of lampreys. Based on computational analysis, a significant number ...
... An inexpensive antifungal drug, thiabendazole, slows tumor growth and shows ... of Natural Sciences at The University of Texas at Austin ... frogs, mice and humans. Thiabendazole is an FDA-approved, generic ... 40 years as an antifungal. It is not currently used ...
... 2012) What comes naturally to most people to ... who have lost the full use of their limbs. New ... TIRR Memorial Hermann aims to help victims recover that ability ... from the National Institutes of Health (NIH) and the President,s ...
Cached Biology News:Research reveals unique solution to gene regulation 2Common antifungal drug decreases tumor growth and shows promise as cancer therapy 2Brain-wave-reading robot might help stroke patients 2Brain-wave-reading robot might help stroke patients 3
(Date:1/15/2014)... , Jan. 15, 2014  Bill Jacobs Automotive, a group of ... teaming up with the Heartland Blood Center and offering free ... The drive takes place Saturday, Jan. 18 at the Bill ... p.m. (Photo: http://photos.prnewswire.com/prnh/20140115/MN46637 )  The ...
(Date:1/15/2014)... FALLS, N.Y., Jan. 15, 2014   Niagara Thermal Products ... selected by Buffalo BioBlower Technologies LLC ("B3") as the ... Phase 2 award from the Army Corps of Engineers.  ... that kills all biological contaminants and destroys VOCs and ...
(Date:1/15/2014)... (PRWEB) January 15, 2014 Cynvenio, a ... management through the genomic analysis of tumor cells in ... Finder™, a web-based cancer decision support application powered by ... with Cynvenio’s ClearID™ genomic test, Therapy Finder will provide ...
(Date:1/15/2014)... Jan. 15, 2014 ­ RedBrick Health , a ... technology, today announces that EmblemHealth , the ... company, is now providing the RedBrick Compass health assessment, ... to all of its members. EmblemHealth is among the ...
Breaking Biology Technology:Join the Bill Jacobs Auto blood drive and receive a free oil change 2Niagara Thermal Products LLC Selected as Critical Supplier for Buffalo BioBlower Air Purification Systems 2Cynvenio Offers New Breast Cancer Therapy Finder Application for its ClearID Breast Cancer Genomic Test 2Cynvenio Offers New Breast Cancer Therapy Finder Application for its ClearID Breast Cancer Genomic Test 3EmblemHealth Selects RedBrick Health to Power its Next-Generation Health Engagement and Behavior Change System 2EmblemHealth Selects RedBrick Health to Power its Next-Generation Health Engagement and Behavior Change System 3
... not just because they sparkle. Engineers and physicists ... is as hard as an ice ball to the ... making it an ideal material or coating for seals, ... Carpick, associate professor in the Department of Mechanical Engineering ...
... Physicists at UC Riverside have made an accidental ... change how information in computers can be transported ... a property electrons possess that makes them behave ... the development of spin-based semiconductor technology such as ...
... Inc.,(Nasdaq: RDYN ) today announced that it has ... Ltd. for faropenem medoxomil (faropenem).,In conjunction with this decision, ... agreement with Asubio Pharma Co., Ltd. and Nippon,Soda Co. ... as a,result of the Company being unable to secure ...
Cached Biology Technology:University of Pennsylvania engineers reveal what makes diamonds slippery at the nanoscale 2Discovery by UC Riverside physicists could enable development of faster computers 2Discovery by UC Riverside physicists could enable development of faster computers 3Replidyne Terminates Faropenem Agreements 2Replidyne Terminates Faropenem Agreements 3
Rabbit polyclonal to HMGB1 (rating: ****) ( Abpromise for all tested applications). entrezGeneID: 3146 SwissProtID: P09429...
Pooled normal sreum with 0.1% sodium azide as preservative...
Mouse CXCR3 Allophycocyanin MAb (Clone 220803)...
Mouse P-Selectin/CD62P MAb (Clone 127933)...
Biology Products: