Researchers helped develop a specially engineered retinal patch to treat people with sudden, severe sight loss.
The results of this groundbreaking clinical study, published in Nature Biotechnology, describe the effective implantation of a specially engineered patch of retinal pigment epithelium cells derived from stem cells to treat people with sudden severe sight loss from wet AMD -- the form that afflicted Waters. This is the first description of a completely engineered tissue that has been successfully used in this way.
The authors used CRISPR/Cas9 to correct β-Thal iPSCs, gene-corrected cells exhibit normal karyotypes and fully pluripotency as hES, showed no off-targeting effects. Their results shown that the gene-corrected β-Thal iPS cell lines restored HBB expression and reduced reactive oxygen species (ROS) production.
Multiple strategies are developed to enable multispectral and combinatorial mosaic gene-function analysis in mice, allowing comparison of molecular phenotypes on a cell-by-cell basis.
BigField GEG Tech's insight:
Here, the scientists describe several methods for the rapid generation of transgenic or gene-targeted mice and embryonic stem (ES) cell lines containing all the necessary elements for inducible, fluorescent, and functional genetic mosaic (ifgMosaic) analysis. This technology enables the interrogation of multiple and combinatorial gene function with high temporal and cellular resolution.
A new primate model has been developed to test treatments that might cure HIV/AIDS and suggests answers to questions raised by the 'Berlin patient,' the only human thought to have been cured so far.
The authors used CRISPR-Cas9 and TALEN to edit the genome of human pluripotent stem cell clones and they performed whole-genome sequencing at high coverage in order to assess the degree of mutagenesis across the entire genome. In both types of clones, they found that off-target mutations attributable to the nucleases were very rare. From this analysis, they suggest that, although some cell types may be at risk for off-target mutations, the incidence of such effects in human pluripotent stem cells may be sufficiently low and thus not a significant concern for disease modeling and other applications.
Welcome page of SkinStemDev INSERM Laboratory on Epithelial pathophysiology , Stem cells and Tissue repair Aberdam's lab Daniel Aberdam
BigField GEG Tech's insight:
Daniel Aberdam was recognized for his work on iPS, his team has reprogrammed hair cells of patients with blindness to generate cornea cells. An important step about hope to restore the vision of patients with this type of disease!
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