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Bardet Biedl Syndrome Family Association

Connecting families and sharing information on research, treatment, and therapies for Bardet Biedl Syndrome.

February 2014 Newsletter: CRISPR, A huge leap forward for gene therapy

A new technology holds promise for rapidly accelerating progress on gene therapy. Gene therapy in use in current approaches to treating retinal degeneration use a modified, harmless virus to deliver a “good” gene to cells. While that technique has shown promise, it’s slow because researchers have to carefuuly calibrate the right amount of the good gene to deliver—too much of a good thing can definitely be bad. The technique also leaves the defective gene in place, which can produce other complications.

The new technique is called CRISPR. It’s based on a process that bacteria use to defend themselves. CRISPR allows a precise gene to be cut out of a strand of dna and replaced with a different gene. Targeting and replacing specific genes solves some of the problems of delivering good genes via virus. Since researchers know which gene is bad and what a good one is, they can simply replace the bad gene with a good one. That means there is less worry about getting the dose right or possible harmful interactions with the defective gene.

These are still very early days—CRISPR was only developed in the last 18 months. A series of papers published in scientific journals this summer verified that the technique works in a wide variety of applications. That has led to rapid adoption of the technique in labs. Dr. Sheffield and Dr. Drack are already working with CRISPR at the University of Iowa. In November, a new company was founded by the scientists who discovered CRISPR specifically to focus on using the technique for gene therapy. We’ll be hearing more about CRISPR and
its application to the development of therapies for BBS this summer at our conference.