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| Content Provider | PubMed Central |
|---|---|
| Author | Lin, Ruei-zeng Alexandra, Dreyzin Aamodt, Kristie Li, Dan Jaminet, Shou-ching S. Dudley, Andrew C. Melero-martin, Juan M. |
| Copyright Year | 2011 |
| Abstract | For decades, autologous ex vivo gene therapy has been postulated as a potential alternative to parenteral administration of recombinant proteins. However, achieving effective cellular engraftment of previously retrieved patient cells is challenging. Recently, our ability to engineer vasculature in vivo has allowed for the introduction of instructions into tissues by genetically modifying the vascular cells that build these blood vessels. In the present study, we genetically engineered human blood–derived endothelial colony-forming cells (ECFCs) to express erythropoietin (EPO) under the control of a tetracycline-regulated system, and generated subcutaneous vascular networks capable of systemic EPO release in immunodeficient mice. These ECFC-lined vascular networks formed functional anastomoses with the mouse vasculature, allowing direct delivery of recombinant human EPO into the bloodstream. After activation of EPO expression, erythropoiesis was induced in both normal and anemic mice, a process that was completely reversible. This approach could relieve patients from frequent EPO injections, reducing the medical costs associated with the management of anemia. We propose this ECFC-based gene-delivery strategy as a viable alternative technology when routine administration of recombinant proteins is needed. |
| Related Links | http://dx.doi.org/10.1182/blood-2011-08-372946 |
| Ending Page | 5428 |
| Page Count | 9 |
| Starting Page | 5420 |
| File Format | |
| ISSN | 00064971 |
| e-ISSN | 15280020 |
| Journal | Blood |
| Issue Number | 20 |
| Volume Number | 118 |
| Language | English |
| Publisher | American Society of Hematology |
| Publisher Date | 2011-11-17 |
| Access Restriction | Open |
| Rights Holder | American Society of Hematology |
| Subject Keyword | Immunology Cell Biology Biochemistry Hematology Research in Higher Education |
| Content Type | Text |
| Resource Type | Article |
| Subject | Cell Biology Hematology Biochemistry Immunology |
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