Gene-edited human stem cell–derived β cells fro
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Posted On: 04/22/2020 4:15:58 PM
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Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice
https://stm.sciencemag.org/content/12/540/eaax9106
2. Complete preclinical studies for gene therapy for optic nerve atrophy in Wolfram.
We have established six new adeno-associated virus constructs for gene therapy. We can transfer and express normal WFS1 gene and MANF gene (regeneration factor) in human cells. We are still at the preclinical stage, and I want to accelerate the speed. I have a few strategies I plan to share with you soon.
https://wolframsyndrome.dom.wustl.edu/rare-disease-day-2020/
https://stm.sciencemag.org/content/12/540/eaax9106
2. Complete preclinical studies for gene therapy for optic nerve atrophy in Wolfram.
We have established six new adeno-associated virus constructs for gene therapy. We can transfer and express normal WFS1 gene and MANF gene (regeneration factor) in human cells. We are still at the preclinical stage, and I want to accelerate the speed. I have a few strategies I plan to share with you soon.
https://wolframsyndrome.dom.wustl.edu/rare-disease-day-2020/
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