How fast can CRISPR be used to cure muscular dystrophy?

Antonio Regalado, 17 October 2016, MIT Technology Review,

Muscular dystrophy (MD) is the result of defects in the dystrophin gene and results in the loss of muscle strength and mass.  CRISPR has been proposed as a possible way to cure individuals suffering from MD. However, progressing from basic research to treatment can take years.  This leaves many like Benjamin Dupree in the position of waiting for a potential cure.  This article examines how the hope for a cure impacts MD individuals and just how fast one may be available.

Engineering guide RNAs

Nowak, C. M., et. al. (2016) Nucleic Acids Research

The CRISPR/Cas9 system is composed of a nuclease, Cas9, and a guide RNA that directs Cas9.  While various Cas9 modifications have been created, very few options of which Cas9 to use exist.  In contrast, many guide RNA modifications have been proposed to help target Cas9 and decrease off-target effects.  This review surveys the different guide RNA modifications and provides guidance on when to use each modification.

CRISPR could provide relief for sickle-cell anemia patients

Heidi Ledford, Nature News, 12 October 2016

Sickle cell anemia is the result of a single base change; however, treatments have been limited.  Now researchers out of UC-Berkeley have reported being able to correct the sickle mutation in up to 25% of cells taken from sickle cell patients.   The modified cells were introduced into mice where, despite the initially positive results, only 5% where able to produce normal hemoglobin, demonstrating that a clinical treatment could still be years away.

CRISPR v TALEN: Where each gene editing technique shines and falls short

Jon Chesnut, GEN News, 13 Oct 2016

While CRISPR gene editing is the hot new technology TALENs may still play a pivotal role in gene editing.  This article breaks down the differences in how TALENs and CRISPR gene editing technologies work and describes when it may be beneficial to use one over the other.

Modeling large structural variations in iPSCs with CRISPR/Cas9

Park, C. Y. et. al. (2016) Nature Protocols 11:2154-2169

While CRISPR/Cas9 gene editing has become an indispensable tool to model genetic disease, it has been limited to the creation and correction of simple mutations.   Presented here is a method developed by Park et. al. for the creation/correction of large chromosomal rearrangements in human induced pluripotent stem cells using CRISPR/Cas9 in approximately 2 weeks.

Myths and Realities of CRISPR Gene Editing

Ellen Jorgensen, TEDSummit 2016,

CRISPR has been revolutionary due to how “cheap” and “easy” it is to use.  However, what do these terms actually mean?  Ellen Jorgensen – founder of the DIY lab Genspace – addresses these questions by discussing the resources and skills required for use of the CRISPR technology.

New revelations in CRISPR patent battle

Jon Cohen, Science Magazine, 5 October 2016,

Two new developments in the CRISPR patent battle may alter who gains control of the lucrative technology.  The Broad Institute has asked patent officials to separate four patents from the current dispute with UC Berkeley, which would allow them to be judged in separate proceedings.  This may ultimately allow both UC Berkeley and the Broad Institute to gain control of CRISPR intellectual property.  Additionally, the pharmaceutical company Cellectis is claiming an umbrella patent covering “most of the gene editing procedures done with a nuclease” including CRISPR/Cas9, zinc fingers, and TALENs.  Other parties are disputing Cellectis’s claims.

Noncoding regions of genome targeted by CRISPR

Anna Azvolinsky, The Scientist, 29 September 2016,

Researchers from Harvard, MIT and the Broad Institute have used CRISPR technology to study regulatory elements of the genome.  The overriding goal of these studies is to develop rules for how non-coding sections of the genome impact coding regions, as well as discover potential drug targets and how genetic changes in the non-coding regions can affect drug resistance.

Monsanto obtains non-exclusive license from the Broad Institute

Jeff Akst 27 September 2016

In the latest CRISPR/Cas9 licensing agreement, Monsanto has acquired a non-exclusive world-wide license from the Broad Institute.  Monsanto plans to use CRISPR for “site-directed integration of specific genes as well as the opportunity to enhance beneficial [plant characteristics] or remove undesired plant characteristics.”