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:9/18/2014)... are the ideal animal in which to study the ... ocean into tens of thousands of freshwater streams and ... to adapt to the new environment. , Breeding studies ... turned up one of the genes that controls tooth ... gene,s regulation in a freshwater population is associated with ...
(Date:9/17/2014)... creation of the Santa Ana Wildfire Threat Index ... of the powerful, hot, dry Santa Ana wind, which ... was introduced Sept. 17 by the U.S. Forest Service, ... Electric. , The index includes four classification levels ranging ... help fire agencies and other emergency responders, the media ...
(Date:9/17/2014)... USA -- "Nature has developed, very cleverly, some lessons ... in optical design," said Joseph Shaw, director of the ... explore surfaces and structures at the nanoscale, we,ll discover ... San Diego in August during a conference called "The ... and Rongguang Liang of the University of Arizona College ...
Breaking Biology News(10 mins):Counting fish teeth reveals regulatory DNA changes behind rapid evolution, adaptation 2Counting fish teeth reveals regulatory DNA changes behind rapid evolution, adaptation 3UCLA scientists play key role in developing new Santa Ana Wildfire Threat Index 2Nature's designs inspire research into new light-based technologies 2Nature's designs inspire research into new light-based technologies 3
... LA Research led by Lauren Cole, a public ... of Public Health and Director of the Epidemiology Program ... the incidence of head and neck cancer has risen ... with the greatest increase among middle-aged white men. At ...
... gene called XRCC2 cause increased breast cancer risk, according to ... Human Genetics . The study looked at families that have ... in the currently known breast cancer susceptibility genes. Sean ... professor in the Department of Oncological Sciences at the University ...
... such as the Iberian lynx, are under an increasingly serious ... that can even survive in extremely arid environments. A study ... to adapt according to climate. The life cycle of ... patterns of people and goods. These parasites and the pathogens ...
Cached Biology News:LSUHSC research finds HPV-related head & neck cancers rising, highest in middle-aged white men 2A new breast cancer susceptibility gene 2Ticks can adapt to the Spain's climatic diversity 2
(Date:9/18/2014)... , Sept. 18, 2014 Research ... "Global Human Albumin Industry Report 2014" report ... Albumin Industry Report 2014 is a professional and in-depth ... albumin industry. The report provides a basic ... industry chain structure. The human albumin market analysis is ...
(Date:9/18/2014)... Ky. , Sept. 18, 2014  Neogen Corporation ... has entered into a strategic agreement with Merck Animal ... Program. The Igenity Dairy Heifer Program ... essential information on the genetic potential of replacement dairy ... a calf is born, and is an excellent tool ...
(Date:9/18/2014)... Woburn, MA (PRWEB) September 18, 2014 ... Single Molecule Sequencing (tSMS™) technology, announced today the ... round of investment, led by Genomic Diagnostic Technologies, ... scientific and diagnostic applications. , Founded in March ... Scientist and Bioinformatics Analyst at Helicos BioSciences, SeqLL ...
(Date:9/18/2014)... the course of a day - car, phone, ... the chemical industry to turn raw materials such ... valuable chemicals that are the ingredients of life,s ... who achieved outstanding progress in driving this energy ... light instead of fossil fuels, has won the ...
Breaking Biology Technology:Global Human Albumin Industry Report 2014 2Global Human Albumin Industry Report 2014 3Merck Animal Health to market Neogen's dairy genomic program 2SeqLL Closes Series-A Funding Round for Expansion of Single Molecule Sequencing 2Using solar energy to turn raw materials into ingredients for every day life 2
... University of Wisconsin Medical Foundation is initiating ... Foundation's clinical data and to improve the reliability of ... QDX Platform V Integration Services from Quovadx, ... in Englewood, Colorado. , ,The Medical Foundations growth ...
... of single-molecule DNA analysis technology, announced Thursday it has commenced ... tumor, known as oligodendroglioma. OpGen, based in Madison, is working ... of genetics and chemistry at the University of Wisconsin-Madison and ... mapping, invented by Schwartz, will enable researchers to determine how ...
... weeks ago, I referred to offshore outsourcing as an ... an issue de lannee (an election year at that ... questions about the economic future of the U.S. Prominent ... the contenders for the Democratic presidential nomination to the ...
Cached Biology Technology:UW Medical Foundation Integrates Medical Records 2OpGen joins UW, Henry Ford Heath in Improving Brain Tumor Treatment 2The Great Offshore Outsourcing Debate 2
Hybridizer (220 V) For more information, please visit Dako' web-site. EU regulatory status: CE US regulatory status: IVD...
... The involvement of integrins in vascular proliferation, ... The integrin family of cell adhesion receptors ... composed of an a and b subunit ... structure and functional diversity of the integrin ...
... exchange media are rigid macroporous hydrophilic media. ... rates at moderate pressures, while the macroporous ... the hydrophilic matrix reduces nonspecific binding. Macro-Prep ... anion exchanger with very high flow rates ...
... siRNA --- a cocktail of 3 siRNAs ... interestis our most popular product. The siTrio ... knockdown of your target gene when used ... transfection and confirmation of optimal transfection with ...
Biology Products: