CRISPR based gene therapies may still be years away

Jocelyn Kaiser, Science

http://www.sciencemag.org/news/2016/05/gene-editor-crispr-won-t-fully-fix-sick-people-anytime-soon-here-s-why

While gene editing techniques such as CRISPR/Cas9 have changed the way basic research is being conducted, widespread use of the technology in medical treatments could be years away.  As with previous promising gene therapies the largest obstacle is delivery of the required proteins into the organs of interest.    The repair of the mutations behind genetic disorders often necessitates the use of HDR, however as HDR has very low efficiencies most of the treated cells may end up being repaired by NHEJ, presenting a challenging obstacle to researchers.  While many gene therapists are excited for the potential of CRISPR/Cas9 technology, years of research are needed before it sees clinical application.

Author: Advanced Analytical

Advanced Analytical Technologies, Inc. (AATI) simplifies complex genomics workflows to accelerate research and discovery in pharmaceuticals, life science, biofuels, biotechnology and healthcare.

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