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Pediatric Diseases and Stem Cells: Recent Advances and Challenges

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Stem Cells: Current Challenges and New Directions

Part of the book series: Stem Cell Biology and Regenerative Medicine ((STEMCELL))

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Abstract

Stem cell treatments hold promise in the pediatric field. Children have increased regenerative potential when compared with adults. In addition, the number of stem cells needed for therapeutic efficacy is much lower in children based on their lower body weights. Therefore, sufficient number of cells may readily be collected from donors and/or may be expanded ex vivo in lesser number of passages. All of these factors are expected to enable more efficient, less expensive, and timely application of stem cells in clinical practice. In this review, we will cover the areas potentially suitable for stem cell therapies in children including inborn errors of metabolism, transplantation, and autoimmune/inflammatory conditions. Hematopoetic and mesenchymal stromal/stem cells will be emphasized as the most available stem cell sources for clinical application at present. In addition, the invaluable role of doing research with the use of induced pluripotent stem cell lines obtained from the cells/tissues of inherited rare diseases is highlighted and future application areas are included.

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Abbreviations

ALD:

Adrenoleukodystrophy

BMT:

Bone marrow transplantation

BPD:

Bronchopulmonary dysplasia

cGMP:

Current good manufacturing practices

CD:

Crohn’s disease

EBMT:

European Group for blood and marrow transplantation

ESC:

Embryonic stem cell

GLD:

Globoid cell leukodystrophy

GCSF:

Granulocyte colony stimulating factor

GVHD:

Graft versus host disease

HSCs:

Hematopoetic stem cells

HSCT:

Hematopoetic stem cell transplantation

HIV:

Human immunodeficiency virus

i.v.:

Intravenous

iPSC:

Induced pluripotent stem cell

MIOP:

Malignant infantile osteopetrosis

MLD:

Metachromatic leukodystrophy

MPS:

Mucopolysaccharidosis

MSC:

Mesenchymal stem cell/mesenchymal stromal cell

NEC:

Necrotizing enterocolitis

OI:

Osteogenesis imperfecta

RDEB:

Recessive dystrophic epidermolysis bullosa

SDF-1 alpha:

Stromal-derived factor 1 alpha

UCB:

Umbilical cord blood

UCBT:

Umbilical cord blood transplantation

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Authors and Affiliations

  1. PEDI-STEM Center for Stem Cell Research and Development, Hacettepe University, Ankara, Türkiye

    Duygu Uçkan

  2. Bone Marrow Transplantation Unit, Ihsan Doğramacı Children’s Hospital Pediatric Hematology, Ankara, Türkiye

    Duygu Uçkan

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  1. Duygu Uçkan
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Correspondence to Duygu Uçkan .

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Editors and Affiliations

  1. Regenerative Medicine Program, Ottawa Hospital Research Institute Sprott Centre for Stem Cell Research, Ottawa, Ontario, Canada

    Kursad Turksen

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Uçkan, D. (2013). Pediatric Diseases and Stem Cells: Recent Advances and Challenges. In: Turksen, K. (eds) Stem Cells: Current Challenges and New Directions. Stem Cell Biology and Regenerative Medicine. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4614-8066-2_7

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