LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
- S. Hacein-Bey-Abina, C. von Kalle, M. Cavazzana‐Calvo
- Biology, MedicineScience
- 17 October 2003
Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
- A. Aiuti, F. Cattaneo, M. Roncarolo
- Medicine, BiologyNew England Journal of Medicine
- 10 December 2009
Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible.
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
- S. Hacein-Bey-Abina, A. Garrigue, M. Cavazzana‐Calvo
- Biology, MedicineJournal of Clinical Investigation
- 2 September 2008
These findings functionally specify a genetic network that controls growth in T cell progenitors and led to sustained remission in 3 of the 4 cases of T cell leukemia, but failed in the fourth.
Microtubule-dependent formation of podosomal adhesion structures in primary human macrophages.
- S. Linder, K. Hüfner, U. Wintergerst, M. Aepfelbacher
- BiologyJournal of Cell Science
- 1 December 2000
The findings show that microtubules are essential for podosome formation in primary human macrophages and that WASp and CIP4 may be involved in this phenomenon.
Long-term follow-up and outcome of 39 patients with chronic granulomatous disease.
- J. Liese, S. Kloos, B. Belohradsky
- Medicine, BiologyJornal de Pediatria
- 1 November 2000
Infections with Aspergillus species have become the major cause of infectious complications and death in patients withCGD and prophylactic and therapeutic measures are needed to further increase life expectancy and quality for patients with CGD.
Successful long-term correction of autosomal recessive hyper-IgE syndrome due to DOCK8 deficiency by hematopoietic stem cell transplantation.
- T. C. Bittner, U. Pannicke, M. Albert
- Medicine, BiologyKlinische Pädiatrie
- 1 November 2010
This report presents the long-term outcome of a girl having received a hematopoietic stem cell graft for an at that time genetically undefined combined immunodeficiency associated with severe eczema, multiple food allergies, excessively elevated serum IgE levels and eosinophilia and results in complete immunological correction after HSCT.
X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options.
- M. Albert, T. C. Bittner, H. Ochs
- MedicineBlood
- 22 April 2010
An analysis of the clinical outcome and molecular basis of patients with XLT shows excellent long-term survival but also a high probability of severe disease-related complications, which will allow better decision making when considering treatment options for individual patients withXLT.
PENTA 2009 guidelines for the use of antiretroviral therapy in paediatric HIV‐1 infection
- S. Welch, M. Sharland, D. Bastiaans
- MedicineHIV Medicine
- 1 July 2002
PENTA Guidelines aim to provide practical recommendations for treating children with HIV infection in Europe with expectations of better outcomes following the ongoing success of antiretroviral therapy (ART).
Dysregulation of innate immune receptors on neutrophils in chronic granulomatous disease.
- D. Hartl, N. Lehmann, U. Wintergerst
- Medicine, BiologyJournal of Allergy and Clinical Immunology
- 1 February 2008
Clinical score for nonbacterial osteitis in children and adults.
- A. Jansson, T. Müller, V. Jansson
- MedicineArthritis & Rheumatism
- 1 April 2009
The proposed scoring system helps to facilitate the diagnostic process in patients with suspected NBO and might spare unnecessary invasive diagnostic and therapeutic procedures.
...
...