Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease

Bhattacharya, Arpita; Slatter, Mary; Curtis, Ann; Chapman, C.; Barge, D; Jackson, Antony P.; Flood, Terry; Abinun, Mario; Cant, Andrew J.; Gennery, Andrew R.

doi:10.1038/sj.bmt.1703863

Cited by 37 publications

(19 citation statements)

References 13 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The majority of these transplants have occurred from 2000 on and have a median follow-up of 25 months. (personal communication) An additional 50 patients outside of the US and Canada are reported in various publications including single case reports and a survey of the European experience [31][32][33][34][35][36][37][38][39][40][41][42][43][44] with an overall survival rate of 94% and a success rate of 43 out of the 50 achieving stable donor engraftment. The longest reported follow up to date from the Europeans is 5 years in a patient transplanted using a HLA-matched sibling and a Busulfan/Cytoxan-based conditioning regimen.…”

Section: Allogeneic Transplantationmentioning

confidence: 97%

Advances in treatment for chronic granulomatous disease

Kang

Malech

2008

Immunol Res

View full text Add to dashboard Cite

Chronic granulomatous disease (CGD) is a rare congenital disorder resulting from a failure of neutrophils to produce oxidases. Patients are therefore prone to recurrent infections from various organisms including fungi and atypical bacteria. The mortality in patients with the X-linked form of CGD, the most common type, ranges from 3% to 5% per year and although management of infections has improved with advances in antimicrobial therapies, better methods are needed to be able to cure these patients. Peripheral blood stem cell or bone marrow transplantation, while curative, is not widely used due to the episodic nature of the infections and the belief by many that conservative management is preferable to the risks of transplantation. Still, as will be discussed, improvements in the field are making allogenic transplantation more desirable and tilting the risk benefit ratio in favor of this modality. Additionally, gene therapy, which has been a long touted method to cure CGD, has within the last 5-10 years become more and more of a reality and may be realized by the end of this decade.

show abstract

Section: Allogeneic Transplantationmentioning

confidence: 97%

Advances in treatment for chronic granulomatous disease

Kang

Malech

2008

Immunol Res

View full text Add to dashboard Cite

show abstract

“…A number of individual case reports have been published but only one small case series describing the outcome of UCSCT for primary immunodeficiency in eight patients. [9][10][11][12][13][14] We report our 5-year single centre experience using UCSCT for patients with primary immune deficiency.…”

mentioning

confidence: 99%

Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency

Bhattacharya

Slatter

Chapman

et al. 2005

Bone Marrow Transplant

Self Cite

View full text Add to dashboard Cite

“…In contrast to BM aspiration, human UCB is obtained by a simple, safe, and painless procedure when the baby is delivered. Since the late 1980s, UCB has become an indispensable source of hematopoietic stem/progenitor cells for transplantation of hematopoietic stem cells to treat some hematological disorders [9][10][11][12][13][14]. However, human UCB has been controversial for the presence of MSCs; some researchers successfully isolated MSCs from UCB, whereas others have not [15][16][17][18].…”

mentioning

confidence: 99%

Skeletal Myogenic Differentiation of Mesenchymal Stem Cells Isolated from Human Umbilical Cord Blood

Gang¹,

Jeong²,

Hong³

et al. 2004

STEM CELLS

279

212

View full text Add to dashboard Cite

Human umbilical cord blood (UCB) has been regarded as an alternative source for cell transplantation and cell therapy because of its hematopoietic and nonhematopoietic (mesenchymal) potential. Although there has been debate about whether mesenchymal stem cells (MSCs) are invariably present in UCB, several reports showed that MSC‐like cells could be consistently derived from human UCB and, moreover, could differentiate into various cells of a mesodermal origin. However, it remains unclear whether these UCB‐derived MSCs are also capable of differentiating into skeletal muscle cells. In this study, we isolated MSCs from human UCB and induced them to differentiate into skeletal muscle cells. During cell culture expansion, UCB‐derived mononuclear cells gave rise to adherent layers of fibroblast‐like cells expressing MSC‐related antigens such as SH2, SH3, α‐smooth muscle actin, CD13, CD29, and CD49e. More important, when these UCB‐derived MSCs were incubated in promyogenic conditions for up to 6 weeks, they expressed myogenic markers in accordance with myogenic differentiation pattern. Both flow cytometric and reverse transcriptase–polymerase reaction analyses showed that two early myogenic markers, MyoD and myogenin, were expressed after 3 days of incubation but not after 2 weeks. At week 6, more than half of UCB‐derived MSCs expressed myosin heavy chain, a late myogenic marker. Our results demonstrate that UCB‐derived MSCs possess a potential of skeletal myogenic differentiation and also imply that these cells could be a suitable source for skeletal muscle repair and a useful tool of muscle‐related tissue engineering.

show abstract

Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease

Cited by 37 publications

References 13 publications

Advances in treatment for chronic granulomatous disease

Advances in treatment for chronic granulomatous disease

Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency

Skeletal Myogenic Differentiation of Mesenchymal Stem Cells Isolated from Human Umbilical Cord Blood

Contact Info

Product

Resources

About