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Stem Cell Therapy for Fanconi Anemia

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Stem Cells: Biology and Engineering

Part of the book series: Advances in Experimental Medicine and Biology ((ICRRM,volume 1083))

Abstract

Stem cell therapy is the administration of stem cells to a patient to treat or prevent a disease. Since stem cells possess the long-term self-renewal capacity and provide daughter cells that differentiate into the specialized cells of each tissue, stem cell therapy will theoretically improve the disease condition for the lifetime of the patient. As the most widely used stem cell therapy, bone marrow transplantation is the treatment of choice for many kinds of blood disorders, including anemias, leukemias, lymphomas, and rare immunodeficiency diseases. For the fatal genetic blood disorder Fanconi anemia, allogeneic bone marrow transplantation has remained the only curative treatment. But the recent advances in stem cell and gene therapy fields may provide promising opportunities for an alternative or even better management of Fanconi anemia. Many of these new ideas and opportunities are also useful for treating other blood diseases that affect hematopoietic stem cells, such as sickle cell anemia, severe combined immunodeficiencies, and beta-thalassemias. In this chapter, these advances along with their challenges and limitations will be thoroughly discussed.

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Abbreviations

AAV:

Adeno-associated virus

FA:

Fanconi anemia

GVHD:

Graft-versus-host disease

HLA:

Human leukocyte antigens

HSCT:

Hematopoietic stem cell transplantation

HSPC:

Hematopoietic stem and progenitor cell

iPS:

Induced pluripotent stem cell

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  1. Oregon Stem Cell Center, Department of Pediatrics, Oregon Health & Science University, 3181 SW Sam Jackson Park Road, Portland, OR, 97239, USA

    Qing-Shuo Zhang

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  1. Qing-Shuo Zhang
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Correspondence to Qing-Shuo Zhang .

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  1. Laboratory of Stem Cell Research and Application, University of Science, Vietnam National University, Ho Chi Minh City, Vietnam

    Phuc Van Pham

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Zhang, QS. (2017). Stem Cell Therapy for Fanconi Anemia. In: Van Pham, P. (eds) Stem Cells: Biology and Engineering. Advances in Experimental Medicine and Biology(), vol 1083. Springer, Cham. https://doi.org/10.1007/5584_2017_67

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