IN THE LABORATORY, CRISPR OR GENE EDITING has proven to be a highly useful tool in genome analyses and engineering such as the creation of model organisms and studying diseases such as cancer that result from multiple mutations. In humans, CRISPR certainly has the potential to cure some genetic diseases. Some of the challenges currently faced include the fact that the editing may not be 100% or that there could be off-target effects. In addition, CRISPR has the same challenges that are faced by the RNAi field. This centers around targeting the system to specific cells/ tissues. If the genetic change required is only in one organ or only in very specific cell types (e.g., hematopoetic stem cells), this could be more difficult. Significant progress has been made over time in the RNAi (RNA interference) field, for example, in liver targeting. There are numerous academic research groups and biotech companies worldwide who are using CRISPR, the field will certainly advance, and as a result, many of these challenges could be solved.
LAURA HALES, PH.D.
Laura has nearly 20 years of industry experience, the bulk of which as a biologics discovery researcher. She is a founder of Extend Biosciences and The Isis Group.