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The degree of muscle phenotypic rescue in mosaic mice exceeded the efficiency of gene correction, likely reflecting an advantage of the corrected cells and their contribution to regenerating muscle.
The reprogrammed, genetically corrected cells functioned normally.
So the numbers of corrected cells rapidly outstrip those of uncorrected cells.
In contrast, the corrected cells live the length of normal red blood cells, about 120 days.
The corrected cells produced healthy hemoglobin, which mutated cells do not make at all.
And corrected cells have about a tenfold advantage over uncorrected cells, he said.
Similar(19)
Gene-corrected cells retained full pluripotency and a normal karyotype following removal of reprogramming factor and drug-resistance genes.
Stably EGFP-corrected cells were generated at a frequency of \(\sim\ 0.05% with an optimized oligonucleotide design combining modified bases and reduced number of phosphorothioate bonds.
We provide evidence from comparative RNA-seq analysis suggesting cellular immunity induced by the oligonucleotides might contribute to the low viability of oligo-corrected cells.
Thus, linking this selection strategy to therapeutic gene expression offers the potential to reconstitute diseased hematopoietic tissue with gene-corrected cells.
However, all patients were maintained on enzyme-replacement therapy, which prevented the evaluation of its efficacy and abolished the selective advantage for gene-corrected cells.
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