New gene activation tool may do what CRISPR cannot: Work inside the body
The ability to precisely adjust the expression of genes inside the human body—ramping up protective ones and tamping down harmful ones—holds enormous potential for treating and preventing disease. But the standard molecular tools used to tune genes, such as the CRISPR system, are too bulky to package and deliver into the body's cells.
New gene activation tool TIGRa may offer benefits over CRISPR-based therapies:
Stanford researchers have developed TIGRa, an ultracompact gene activation tool that can be packaged inside viral vectors for delivery into human cells. Unlike traditional CRISPR tools, TIGRa is more versatile and efficient at activating multiple genes simultaneously. In mouse models, TIGRa successfully activated two protective genes in retinal ganglion cells, helping retain partial vision in mice with retinal damage.
TIGRa's compact size, based on the TIGR-Tas gene-targeting system, makes it suitable for therapeutic use. Multiple variants of TIGRa show promise in activating diverse therapeutic genes, including those for retinal degeneration, muscular dystrophy, and obesity. When packed into viral vectors and injected into mice, TIGRa improved retinal ganglion cell survival and preserved retinal structure after injury.
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