A number of diseases, including glaucoma and multiple sclerosis, attack the retina in the human eye, destroying ganglion cells or nerve cells and causing vision loss and even blindness. A group of researchers at Johns Hopkins have developed a technique to use stem cells derived from human embryos to create retina nerve cells. The technique has the potential for therapies that could cure the ravages of diseases that damage the retina.
The team used a tool called CRISPR-Cas9 to insert DNA into the stem cells. Part of the genetic manipulation involves having the cells glow bright red to differentiate them from other cells. Then the researchers used a cell-sorting technique to separate the newly formed retina ganglion cells from the rest of the material.
The production of these ganglion cells will allow the scientist to study their biology in detail. The research, in turn, could lead to new drug therapies that can slow or even stop the destruction of retina ganglion cells.
The holy grail of the research would be to develop transplant procedures so that grown ganglion cells could be transplanted into human eyes that have been damaged by disease. In this fashion, people who suffer from low vision or blindness because their retinas have been damaged can have some measure of their sight restored.
For the purpose of developing new therapies for the biotechnology industry, Donald Zack, M.D., Ph.D., the Guerrieri Family Professor of Ophthalmology at the Johns Hopkins University School of Medicine is working closely with Peter Calabresi, M.D., professor of neurology and director of the Johns Hopkins Multiple Sclerosis Center.
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