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| Name | Class |
|---|---|
| Goethe University | OTHER |
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The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| retroviral SF71-gp91phox transduced CD34+ cells | Genetic | autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells |
| Measure | Description | Time Frame |
|---|---|---|
| eradication of pre-existing therapy refractory bacterial and/or fungal infections | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Reconstitution of ROS production by peripheral blood cells | 1 month |
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| Name | Affiliation | Role |
|---|---|---|
| Reinhard Seger, Prof Dr med | University Children's Hospital, Zurich | Principal Investigator |
| Janine Reichenbach, PD Dr med | University Children's Hospital, Zurich | Study Chair |
| Ulrich Siler, Dr rer nat | University Children's Hospital, Zurich | Study Chair |
| Manuel Grez, Dr rer nat | Georg Speyer Research Institute, Frankfurt a.M. | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Children's Hospital | Zurich | CH-8032 | Switzerland |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 16582916 | Background | Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006 Apr;12(4):401-9. doi: 10.1038/nm1393. Epub 2006 Apr 2. |
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| ID | Term |
|---|---|
| D006105 | Granulomatous Disease, Chronic |
| ID | Term |
|---|---|
| D010585 | Phagocyte Bactericidal Dysfunction |
| D007960 | Leukocyte Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |