The -15/WT genotype will have both, a normal and a slightly shortened CCR5 giving no HIV protection at all. The -4/+1 genotype may suffer the fate of non-sense mediated decay. Here are the amino acid predictions for each genotype (I reverted to Topcocns as Protter had some problems in generating correct plots).
From the original AJHG paper, however, also delta 32 carriers may be HIV infected. As there exists also non-CCR5 dependent virus replication, also Nana is at risk of HIV infection when being virus exposed.
Now Mitalipov is believed to have broken new ground both in the number of embryos experimented upon and by demonstrating that it is possible to safely and efficiently correct defective genes that cause inherited diseases.
Although none of the embryos were allowed to develop for more than a few days—and there was never any intention of implanting them into a womb—the experiments are a milestone on what may prove to be an inevitable journey toward the birth of the first genetically modified humans. […] Reached by Skype, Mitalipov declined to comment on the results, which he said are pending publication. But other scientists confirmed the editing of embryos using CRISPR. “So far as I know this will be the first study reported in the U.S.”
For a few moments of fame, scientists take every risk, even “mass destruction and proliferation”.
It reminds me to the first gene therapy trial by French Anderson who was later stripped of tenure, fired from his faculty position and barred from the campus of his university.
First of all, the current gene therapies are not being entered into the clinical trial databases on a regular basis. Maybe as the number of treated patients go down to a single individual or as there is no control group. I would really like the recommendation of a priori entry into clinicaltrials.gov or clinicaltrialsregister.eu (or some newly designed gene therapy databases). Just to get towards a clear risk/ benefit ratio.
Second, there should be some way to recognize gene editing. Some barcode as we used it already long ago, an artificial sequence that indicates the new insert. epigenie.com summarized the current approaches by last summer: Gestalt/Jay Shendure, barcode/Stephen R. Quake, scartrace/Jan Philipp Junker, hgRNA/Prasant Mali, mScribe/Tim Lu. Something like the FLAG-tag. Or more recently
R. Kalhor et al., “Rapidly evolving homing CRISPRbarcodes,” Nature Methods, doi:10.1038/nmeth.4108, 2016.
S.D. Perli et al., “Continuous genetic recording with self-targeting CRISPR-Cas in human cells,” Science, doi: 10.1126/science.aag0511, 2016.
It would be irresponsible to proceed with any clinical use of germline editing unless and until (i) the relevant safety and efficacy issues have been resolved, based on appropriate understanding and balancing of risks, potential benefits, and alternatives, and (ii) there is broad societal consensus about the appropriateness of the proposed application.