Mesenchymal stem cells for trinucleotide repeat disorders

Geralyn Annett, Gerhard Bauer, Jan Nolta

Research output: Chapter in Book/Report/Conference proceedingChapter

2 Citations (Scopus)

Abstract

Mesenchymal stem cells/marrow stromal cells (MSCs) are ideally suited for cellular therapy due to their ease of isolation, manipulation, and strong safety profile in the clinic. They can be expanded from normal qualified human donors in large quantities and can be infused without tissue matching, since they shield themselves from the immune system. The ability to be transplanted without tissue matching has allowed large multicenter trials to be conducted with direct comparison of the same batches of MSCs, without adverse events or rejection reactions. MSCs are now approved as drugs in several countries outside of the USA. MSCs can be genetically modified to provide sustained and long-term delivery of growth factors at supraphysiological levels. Gene-modified MSCs are in clinical trials for the treatment of stroke and are under consideration for the treatment of neurodegenerative disorders such as Huntington's disease.

Original languageEnglish (US)
Title of host publicationMethods in Molecular Biology
Pages79-91
Number of pages13
Volume1010
DOIs
StatePublished - 2013

Publication series

NameMethods in Molecular Biology
Volume1010
ISSN (Print)10643745

Fingerprint

Trinucleotide Repeats
Stromal Cells
Mesenchymal Stromal Cells
Bone Marrow
Aptitude
Huntington Disease
Neurodegenerative Diseases
Multicenter Studies
Immune System
Intercellular Signaling Peptides and Proteins
Stroke
Clinical Trials
Safety
Pharmaceutical Preparations
Genes

Keywords

  • BDNF
  • Clinical trials
  • Human mesenchymal stem cells
  • Huntington's disease
  • Neurite outgrowth
  • Neurodegenerative diseases
  • Tissue repair

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics

Cite this

Annett, G., Bauer, G., & Nolta, J. (2013). Mesenchymal stem cells for trinucleotide repeat disorders. In Methods in Molecular Biology (Vol. 1010, pp. 79-91). (Methods in Molecular Biology; Vol. 1010). https://doi.org/10.1007/978-1-62703-411-1-6

Mesenchymal stem cells for trinucleotide repeat disorders. / Annett, Geralyn; Bauer, Gerhard; Nolta, Jan.

Methods in Molecular Biology. Vol. 1010 2013. p. 79-91 (Methods in Molecular Biology; Vol. 1010).

Research output: Chapter in Book/Report/Conference proceedingChapter

Annett, G, Bauer, G & Nolta, J 2013, Mesenchymal stem cells for trinucleotide repeat disorders. in Methods in Molecular Biology. vol. 1010, Methods in Molecular Biology, vol. 1010, pp. 79-91. https://doi.org/10.1007/978-1-62703-411-1-6
Annett G, Bauer G, Nolta J. Mesenchymal stem cells for trinucleotide repeat disorders. In Methods in Molecular Biology. Vol. 1010. 2013. p. 79-91. (Methods in Molecular Biology). https://doi.org/10.1007/978-1-62703-411-1-6
Annett, Geralyn ; Bauer, Gerhard ; Nolta, Jan. / Mesenchymal stem cells for trinucleotide repeat disorders. Methods in Molecular Biology. Vol. 1010 2013. pp. 79-91 (Methods in Molecular Biology).
@inbook{7a5dc139e2234d36b9d9802e74525d11,
title = "Mesenchymal stem cells for trinucleotide repeat disorders",
abstract = "Mesenchymal stem cells/marrow stromal cells (MSCs) are ideally suited for cellular therapy due to their ease of isolation, manipulation, and strong safety profile in the clinic. They can be expanded from normal qualified human donors in large quantities and can be infused without tissue matching, since they shield themselves from the immune system. The ability to be transplanted without tissue matching has allowed large multicenter trials to be conducted with direct comparison of the same batches of MSCs, without adverse events or rejection reactions. MSCs are now approved as drugs in several countries outside of the USA. MSCs can be genetically modified to provide sustained and long-term delivery of growth factors at supraphysiological levels. Gene-modified MSCs are in clinical trials for the treatment of stroke and are under consideration for the treatment of neurodegenerative disorders such as Huntington's disease.",
keywords = "BDNF, Clinical trials, Human mesenchymal stem cells, Huntington's disease, Neurite outgrowth, Neurodegenerative diseases, Tissue repair",
author = "Geralyn Annett and Gerhard Bauer and Jan Nolta",
year = "2013",
doi = "10.1007/978-1-62703-411-1-6",
language = "English (US)",
isbn = "9781627034104",
volume = "1010",
series = "Methods in Molecular Biology",
pages = "79--91",
booktitle = "Methods in Molecular Biology",

}

TY - CHAP

T1 - Mesenchymal stem cells for trinucleotide repeat disorders

AU - Annett, Geralyn

AU - Bauer, Gerhard

AU - Nolta, Jan

PY - 2013

Y1 - 2013

N2 - Mesenchymal stem cells/marrow stromal cells (MSCs) are ideally suited for cellular therapy due to their ease of isolation, manipulation, and strong safety profile in the clinic. They can be expanded from normal qualified human donors in large quantities and can be infused without tissue matching, since they shield themselves from the immune system. The ability to be transplanted without tissue matching has allowed large multicenter trials to be conducted with direct comparison of the same batches of MSCs, without adverse events or rejection reactions. MSCs are now approved as drugs in several countries outside of the USA. MSCs can be genetically modified to provide sustained and long-term delivery of growth factors at supraphysiological levels. Gene-modified MSCs are in clinical trials for the treatment of stroke and are under consideration for the treatment of neurodegenerative disorders such as Huntington's disease.

AB - Mesenchymal stem cells/marrow stromal cells (MSCs) are ideally suited for cellular therapy due to their ease of isolation, manipulation, and strong safety profile in the clinic. They can be expanded from normal qualified human donors in large quantities and can be infused without tissue matching, since they shield themselves from the immune system. The ability to be transplanted without tissue matching has allowed large multicenter trials to be conducted with direct comparison of the same batches of MSCs, without adverse events or rejection reactions. MSCs are now approved as drugs in several countries outside of the USA. MSCs can be genetically modified to provide sustained and long-term delivery of growth factors at supraphysiological levels. Gene-modified MSCs are in clinical trials for the treatment of stroke and are under consideration for the treatment of neurodegenerative disorders such as Huntington's disease.

KW - BDNF

KW - Clinical trials

KW - Human mesenchymal stem cells

KW - Huntington's disease

KW - Neurite outgrowth

KW - Neurodegenerative diseases

KW - Tissue repair

UR - http://www.scopus.com/inward/record.url?scp=84880349302&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84880349302&partnerID=8YFLogxK

U2 - 10.1007/978-1-62703-411-1-6

DO - 10.1007/978-1-62703-411-1-6

M3 - Chapter

C2 - 23754220

AN - SCOPUS:84880349302

SN - 9781627034104

VL - 1010

T3 - Methods in Molecular Biology

SP - 79

EP - 91

BT - Methods in Molecular Biology

ER -