Curing diseases with CRISPR

Over the past several years, huge advances in genome editing technologies have fueled an almost palpable excitement about the future of genome engineering. Beginning with the discovery of zinc-finger nucleases, and followed recently by the description of TALEN and CRISPR genome-editing systems, the possibility of literally rewriting a genome has quickly gone from dream to reality.

An incredibly exciting potential use of genome-editing technologies is to correct genetic mutations that cause diseases. Everyone aware of CRISPR and TALEN technology has obviously considered this possibility, and the medical and biotechnology fields are working towards the development of the first genome editing-based treatments.

Now, for the first time, researchers have demonstrated that CRISPR can be used to eliminate a disease-causing mutation from cells in an animal model of a human disease. These three studies, published in the most recent issue of Science, show that the CRISPR system can effectively rewrite the genome of mice carrying a genetic defect causing muscular dystrophy. They demonstrated that editing can be done in cell culture or directly in an animal, to effectively cure the disease.

It now seems like only a matter of time before genome-editing is applied to human patients, and hopes are high for the medical potential of CRISPR and TALEN.

Cyagen Biosciences provides custom mouse and rat models, including transgenics, knockouts and knockins, and CRISPR/Cas9 or TALEN genome editing. We also have an extensive line of stem cells and cell culture reagents, as well as custom virus packaging. Our VectorBuilder platform provides a wide variety of molecular engineering services. Using our innovative online tools, you can design and order custom DNA constructs specific to your experimental needs. Right now, you can choose from lentiviruses, AAV vectors, shRNA expression vectors, CRISPR/Cas9 vectors, and more. Soon, VectorBuilder will also offer AAV-based CRISPR vectors!

Bibliography

1. Tabebordbar M, Zhu K, Cheng JK, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ. (2016) In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351:407-11.
2. Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D, Gersbach CA. (2016) In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351:403-7. 
3. Long C, Amoasii L, Mireault AA, McAnally JR, Li H, Sanchez-Ortiz E, Bhattacharyya S, Shelton JM, Bassel-Duby R, Olson EN. (2016) Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351:400-3.