CRISPR Gene Editing

Review articles on the subject have appeared in Nature magazine.   Subsequently a webinar on Perspectives on the potential for CRISPR Editing of embryos was presented (last week) to interested members of ASRM (The American Society of Reproductive Medicine) including speakers: Steven Palter, MD, Art Caplan, PhD, Paula Amato, MD, Dieter Egli, PhD, Jun Lu PhD

Discussion included use of CRISPR to knock out Genes, repair genes, and alter genes in basic research.   CRISPR can also be used to create a library of genes to enable gene screening.    CRISPR has been used to correct a germ cell (reproductive cell) in an animal model, in other words gene editing to correct a mutation. 

In a human setting, CRISPR was used to correct a gene for genetic IHSS (a heart abnormality: idiopathic hypertrophic sub aortic stenosis) in a majority of cells  (embryos)  treated.   In these cases,  sperm with the genetic mutation were injected into eggs along with the engineered CRISPR. 

Risks include:  Mosaicism (more than one type of cell- some corrected and some not or some corrected in a different way),  “off-target edits”  which is in effect inducement of changes or mutations in other places in the embryo genome.

There was agreement that this technology is not yet ready for clinical use due to safety considerations.    There was also agreement that further and additional work should be continued given the potential benefit for people if the safety concerns can be addressed.

In addition:  Ethical issues were raised including regarding experimentation with genetics.  In addition the idea that the embryo cannot consent to the modification being applied was raised.    The ethics of eventually being able to genetically modify adult individuals was also considered.   Currently this type of research is not permitted in the US.