New Gene Tool Could Succeed Where CRISPR Fails In Vivo
Stanford researchers have developed TIGRa, an ultracompact gene activation tool designed to overcome the size limitations of CRISPR systems. The tool successfully improved vision in a mouse model of glaucoma, showing potential for future in vivo gene therapies.
Why it matters
TIGRa offers a more efficient and versatile method for gene therapy, potentially enabling treatments for complex diseases that current CRISPR technology cannot address.
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.
When delivered by viral vectors, for example, which are packed with the DNA instruction manual for cells to build CRISPR themselves, the lengthy instructions for CRISPR hardly fit inside a single virus. For therapies that may require adjusting multiple genes, delivering multiple CRISPR components separately becomes complicated and inefficient.
Now, Stanford Medicine scientists have developed a travel-sized solution - an ultracompact gene activation tool, called TIGRa (pronounced "tiger A"), small enough that its short DNA instructions can be packed inside viral vectors with room to spare.
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