Nature: CRISPR/Cas9 system saves blind mice

The British Journal of Natural Communications published an important study of genetics. A genome editing method can prevent the retinal degeneration of mice and save blind mice. The method described utilizes the CRISPR/Cas9 gene therapy system and is applicable to a variety of potential genetic defects that cause retinitis pigmentosa, the leading cause of blindness.

Retinitis pigmentosa is incurable and difficult to detect, causing great harm to the patient's eyes and is hereditary. The disease is characterized by retinal degeneration, but since retinitis pigmentosa may be caused by mutations in more than 60 genes, it is very difficult to develop targeted therapies to repair each specific gene.

The retinal includes rod cells and cones, and mutations in the gene that cause retinitis pigmentosa first cause rod cell death, which in turn causes cone death, which ultimately leads to blindness in the patient.

Wu Zhijian, a researcher at the National Eye Institute of the United States, and colleagues did not treat diseases that caused genetic mutations. Instead, they tested a method for retaining cones. They use the most popular "star" technology in the biomedical field - the CRISPR/Cas9 system, which disables the genes that determine the identity of rod cells and induces rod cells to acquire cone characteristics that protect them from harmful pathogenesis. The impact of mutations.

《Nature》:CRISPR/Cas9系统可拯救失明小鼠

The results of the experiment showed that in three retinal degeneration mouse models (a total of 30 mice), the therapy prevented retinal degeneration and improved vision.

This study suggests that this method may be suitable for the treatment of retinal degenerative diseases and may be widely applicable without being affected by different potential genetic mutations.

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