Abstract
A platform has been devised in which autologous stem cells are the targets of both long-term and transient gene therapy culminating in synergistic combinations. Gene targeting and cybridization drive long-term gene therapy: they mediate gene repair/alteration or targeted transgene integration, and mitochondrial (mt)DNA transfer, respectively. Transient regenerative gene therapy drives regenerative medicine sensu stricto (aging/degenerative pathologies) and synergizes long-term gene therapy, thereby magnifying the repopulating/ regenerative ability of target stem cells/cybrids (inherited/acquired disorders including mtDNA diseases). Transient gene therapy has an epigenetic arm for long-term gene inactivation mediated by promoter-specific siRNAs (Morris et al, 2004); conversely, gene activation might possibly be amenable to non-coding antisense RNAs. Such a universal platform is aimed at eliminating hazardous random- integration of therapeutic DNA, at reversing inherited diseases by reestablishing wild-type genomic homeostasis and at tackling most pathologies through stem cell repopulation dynamics into appropriate niches (long-term engraftment) and tissues (cell turn- over).
Published Version
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