The new paper published in PNAS includes four authors from the UC San Diego Department of Bioengineering: Athurva Gore, Zhe Li, Ho-Lim Fung and bioengineering professor Kun Zhang, who leads the Laboratory of Integrative Genomics at the Jacobs School of Engineering.
The press release from the University of Wisconsin-Madison: Study shows patient's own cells may hold therapeutic promise after reprogramming, gene correction
In the study, the researchers used a technique called episomal reprogramming to generate the induced pluripotent stem cells. In contrast to techniques that use retroviruses, episomal reprogramming doesn't involve inserting DNA into the genome. This technique allowed them to produce cells that were free of potentially harmful transgene sequences.
The scientists then corrected the actual retinal disease-causing gene defect using a technique called homologous recombination. The stem cells were extensively "characterized" or studied before and after the process to assess whether they developed significant additional mutations or variations. The results showed that the culture conditions required to correct a genetic defect did not substantially increase the number of mutations.
Brad Fikes from the North County Times covered the story: Induced Pluripotent Stem Cells Pass Gene Therapy Test
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