Title |
Preconditioning of mesenchymal stem cells for improved transplantation efficacy in recessive dystrophic epidermolysis bullosa
|
---|---|
Published in |
Stem Cell Research & Therapy, November 2014
|
DOI | 10.1186/scrt511 |
Pubmed ID | |
Authors |
Christopher Perdoni, John A McGrath, Jakub Tolar |
Abstract |
The use of hematopoietic cell transplantation (HCT) has previously been shown to ameliorate cutaneous blistering in pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), an inherited skin disorder that results from loss-of-function mutations in COL7A1 and manifests as deficient or absent type VII collagen protein (C7) within the epidermal basement membrane. Mesenchymal stem cells (MSCs) found within the HCT graft are believed to be partially responsible for this amelioration, in part due to their intrinsic immunomodulatory and trophic properties and also because they have been shown to restore C7 protein following intradermal injections in models of RDEB. However, MSCs have not yet been demonstrated to improve disease severity as a stand-alone systemic infusion therapy. Improving the efficacy and functional utility of MSCs via a pre-transplant conditioning regimen may bring systemic MSC infusions closer to clinical practice. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Oman | 1 | 25% |
Russia | 1 | 25% |
Unknown | 2 | 50% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 3 | 75% |
Science communicators (journalists, bloggers, editors) | 1 | 25% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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United States | 2 | 4% |
Japan | 1 | 2% |
Austria | 1 | 2% |
Unknown | 50 | 93% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Ph. D. Student | 10 | 19% |
Researcher | 10 | 19% |
Student > Bachelor | 6 | 11% |
Other | 3 | 6% |
Student > Doctoral Student | 2 | 4% |
Other | 10 | 19% |
Unknown | 13 | 24% |
Readers by discipline | Count | As % |
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Agricultural and Biological Sciences | 11 | 20% |
Medicine and Dentistry | 11 | 20% |
Biochemistry, Genetics and Molecular Biology | 7 | 13% |
Engineering | 3 | 6% |
Immunology and Microbiology | 2 | 4% |
Other | 6 | 11% |
Unknown | 14 | 26% |