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Dual CRISPR RNA Method May Help Treat Duchenne Muscular Dystrophy
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KyotoJapanAkitsu-hottaKyoto-universityStem-cell-reportsGene editing system restores dystrophin function in stem cells from patients with Duchenne muscular dystrophy
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KyotoJapanAkitsu-hottaKyoto-universityStem-cell-reportsKyoto University The Hidetoshi Sakurai laboratory reports two new markers that should advance iPS cell therapies for muscle degeneration. Muscular dystrophy describes a group of muscle degenerative congenital diseases. The severity and rates differ between disease and patient, but in all cases the degeneration progresses, sometimes leaving patients in wheelchairs and unable to breathe without artificial respiration. With no present cure, scientists are experimenting with the transplantation of muscle progenitor cells, but the amounts needed can only be made from stem cells such as iPS cells. A new study by CiRA Associate Professor Hidetoshi Sakurai and colleagues report two cell surface receptors, CDH13 and FGFR4, that are expected to advance iPS cell therapies for these diseases.
Japan
Kyoto
Mingming-zhao
Akitsu-hotta
Naoshi-sugimoto
Antonio-lucena-cacace
Tatsuya-jonouchi
Hidetoshi-sakurai
Takuma-mizusawa
Mitsuru-sasaki-honda
Minas-nalbandian
Masahiko-yasuda
Kyoto University
The Hotta lab shows that protection from cellular RNA enhances the genome editing of iPS cells. The ability for scientists to edit human genomic DNA has been around for decades. Such research has revealed how a single mutation in just one of the billions of base pairs in the human genome can cause destructive diseases. In the past decade, advances in CRISPR-Cas9 technology have taken DNA editing a major step farther and promise to revolutionize regenerative medicine. One of the biggest challenges for therapeutic application is the efficiency of precise genome editing. A new study by Junior Associate Professor Akitsu Hotta and colleagues reports a simple but key step in the delivery of CRISPR-Cas9 into iPS cells that enhances this efficiency.
Japan
Kyoto
Takahiro-iguchi
Peter-gee
Miyuki-ono
Akitsu-hotta
Yuto-kita
Huaigeng-xu
Xiouh-wang
Noriko-sasakawa
Akihiro-kagita
Ps-cell-research