New CRISPR Target: RNA


NYSCF – Robertson Stem Cell Investigator and NYSCF – Robertson Stem Cell Prize recipient Feng Zhang, PhD, Core Member of the Broad Institute of Harvard and MIT, shared yet another novel update of CRISPR gene editing technology. Published in Science, an exciting paper describes a new CRISPR-based gene editing system that targets RNA, the molecule responsible for translating DNA into protein, instead of DNA.

Watch a presentation by NYSCF – Robertson Investigator, Feng Zhang, PhD, who helped pioneer the revolutionary CRISPR gene editing tool.

DNA comprises the human genome, the set of genetic information or instructions that make each of us who we are. DNA consists of codes for proteins that are the building blocks of all parts of life, but it needs RNA to carry out these instructions. If a change is made in the DNA of an organism, that change will be passed down from generation to generation. However, if a change is made in RNA, it is not passed on.

This breakthrough technology, dubbed the “REPAIR” system, allows transient changes to the interpretation of DNA, instead of a permanent DNA change. This achievement also eliminates one of the persistent concerns about CRISPR usage, that permanent changes to the genome are not always the right approach. This system could enable the correction of a genetic mutation, and therefore a potential cure for a wide number of diseases, without actually changing the genome.

Read the paper in Science

Read more in the LA Times

Read more in Vox

Diseases & Conditions:

Genomics & Gene Editing

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