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 of renewal during this time period, the transplanted stem cells still retained molecular features of palm skin cells and did not adopt features of leg skin cells. "This finding suggests that adult stem cells primarily regenerate the tissue in which they normally reside, with little plasticity to regenerate other tissues," De Luca says. "This calls into question the supposed plasticity of adult stem cells and highlights the need to carefully chose the right type of stem cell for therapeutic tissue regeneration."


'/>"/>

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

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:5/9/2016)... , UAE, May 9, 2016 ... it comes to expanding freedom for high net worth ... Even in today,s globally connected world, there is still ... system could ever duplicate sealing your deal with a ... second passports by taking advantage of citizenship via investment ...
(Date:4/26/2016)... , April 27, 2016 ... the  "Global Multi-modal Biometrics Market 2016-2020"  report to ... ) , The analysts forecast the ... CAGR of 15.49% during the period 2016-2020.  ... number of sectors such as the healthcare, BFSI, ...
(Date:4/13/2016)... physicians supporting Medicaid patients in Central Florida ... telehealth thanks to a new partnership with higi.   ... can routinely track key health measurements, such as blood ... they opt in, share them with IMPOWER clinicians through ... location at no cost. By leveraging this data, IMPOWER ...
Breaking Biology News(10 mins):
(Date:6/23/2016)... ... 23, 2016 , ... Mosio, a leader in clinical research ... Recruitment and Retention Tips.” Partnering with experienced clinical research professionals, Mosio revisits the ... tools, and strategies for clinical researchers. , “The landscape of how patients receive ...
(Date:6/23/2016)... Mass. , June 23, 2016   ... development of novel compounds designed to target cancer ... napabucasin, has been granted Orphan Drug Designation from ... the treatment of gastric cancer, including gastroesophageal junction ... stemness inhibitor designed to inhibit cancer stemness pathways ...
(Date:6/23/2016)... , June 23, 2016 A person commits a ... crime scene to track the criminal down. An ... Food and Drug Administration (FDA) uses DNA evidence to track ... Sound far-fetched? It,s not. The FDA has increasingly ... support investigations of foodborne illnesses. Put as simply as possible, ...
(Date:6/23/2016)... ... June 23, 2016 , ... ... and Mold) microbial test has received AOAC Research Institute approval 061601. , “This ... introduced last year,” stated Bob Salter, Vice President of Regulatory and Industrial Affairs. ...
Breaking Biology Technology: