Mammalian artificial chromosomes and clinical applications for genetic modification of stem cells

an overview.

R. Katona, Sandra L. Vanderbyl, Carl F. Perez

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

Modifying multipotent, self-renewing human stem cells with mammalian artificial chromosomes (MACs), present a promising clinical strategy for numerous diseases, especially ex vivo cell therapies that can benefit from constitutive or overexpression of therapeutic gene(s). MACs are nonintegrating, autonomously replicating, with the capacity to carry large cDNA or genomic sequences, which in turn enable potentially prolonged, safe, and regulated therapeutic transgene expression, and render MACs as attractive genetic vectors for "gene replacement" or for controlling differentiation pathways in progenitor cells. The status quo is that the most versatile target cell would be one that was pluripotent and self-renewing to address multiple disease target cell types, thus making multilineage stem cells, such as adult derived early progenitor cells and embryonic stem cells, as attractive universal host cells. We will describe the progress of MAC technologies, the subsequent modifications of stem cells, and discuss the establishment of MAC platform stem cell lines to facilitate proof-of-principle studies and preclinical development.

Original languageEnglish
Pages (from-to)199-216
Number of pages18
JournalMethods in molecular biology (Clifton, N.J.)
Volume738
DOIs
Publication statusPublished - 2011

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Mammalian Artificial Chromosomes
Stem Cells
Genetic Vectors
Embryonic Stem Cells
Cell- and Tissue-Based Therapy
Transgenes
Genes
Complementary DNA
Technology
Cell Line

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Mammalian artificial chromosomes and clinical applications for genetic modification of stem cells : an overview. / Katona, R.; Vanderbyl, Sandra L.; Perez, Carl F.

In: Methods in molecular biology (Clifton, N.J.), Vol. 738, 2011, p. 199-216.

Research output: Contribution to journalArticle

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