| Title |
Curative haploidentical BMT in a murine model of X-linked chronic granulomatous disease
|
|---|---|
| Published in |
International Journal of Hematology, April 2015
|
| DOI | 10.1007/s12185-015-1799-8 |
| Pubmed ID | |
| Authors |
Yasuo Takeuchi, Emiko Takeuchi, Takashi Ishida, Masafumi Onodera, Hiromitsu Nakauchi, Makoto Otsu |
| Abstract |
Chronic granulomatous disease (CGD) is a primary immunodeficiency disorder characterized by defective microbial killing in phagocytes. Long-term prognosis for CGD patients is generally poor, highlighting the need to develop minimally toxic, curative therapeutic approaches. We here describe the establishment of a mouse model in which X-linked CGD can be cured by allogeneic bone marrow transplantation. Using a combination of non-myeloablative-dose total body irradiation and a single injection of anti-CD40 ligand monoclonal antibody, transplantation of whole bone marrow cells achieved long-lasting mixed chimerism in X-linked CGD mice in a haploidentical transplantation setting. Stable mixed chimerism was maintained for up to 1 year even at a low range (<20 % donor cells), indicating induction of donor-specific tolerance. The regimen induced mild myelosuppression without severe acute complications. Stable chimerism was therapeutic, as it suppressed cutaneous granuloma formation in an in vivo test suited for evaluation of treatment efficacy in murine CGD models. These results warrant future development of a simplified allogeneic hematopoietic cell transplantation regimen that would benefit CGD patients by allowing the use of haploidentical donor grafts without serious concerns of severe treatment-related toxicity. |
X Demographics
The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 1 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 1 | 100% |
Mendeley readers
The data shown below were compiled from readership statistics for 17 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 17 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Other | 2 | 12% |
| Professor > Associate Professor | 2 | 12% |
| Student > Bachelor | 1 | 6% |
| Student > Ph. D. Student | 1 | 6% |
| Professor | 1 | 6% |
| Other | 2 | 12% |
| Unknown | 8 | 47% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 3 | 18% |
| Engineering | 2 | 12% |
| Nursing and Health Professions | 1 | 6% |
| Agricultural and Biological Sciences | 1 | 6% |
| Biochemistry, Genetics and Molecular Biology | 1 | 6% |
| Other | 0 | 0% |
| Unknown | 9 | 53% |
Attention Score in Context
This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 26 February 2016.
All research outputs
#20,311,744
of 22,852,911 outputs
Outputs from International Journal of Hematology
#1,084
of 1,394 outputs
Outputs of similar age
#222,887
of 264,475 outputs
Outputs of similar age from International Journal of Hematology
#8
of 13 outputs
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