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An Anonymous Coward writes:

Here we demonstrate safe intravenous and intra-amniotic administration of polymeric nanoparticles to fetal mouse tissues at selected gestational ages with no effect on survival or postnatal growth. In utero introduction of nanoparticles containing peptide nucleic acids (PNAs) and donor DNAs corrects a disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia, yielding sustained postnatal elevation of blood hemoglobin levels into the normal range, reduced reticulocyte counts, reversal of splenomegaly, and improved survival, with no detected off-target mutations in partially homologous loci.

[...] Unlike gene editing technologies that rely on the activity of exogenously delivered nucleases18,19—such as zinc finger nucleases, TAL effector nucleases, and CRISPR/Cas9—PNA/DNA NPs can be readily administered in vivo and have been shown to have extremely low to undetectable off-target effects in the genome because the PNA editing molecules lack inherent nuclease activity5,6,7.

[...] Unlike other gene editing technologies that rely on activity of exogenous nucleases (CRISPR/Cas, TAL effector nucleases and zinc finger nucleases) that can create extraneous double-stranded breaks, PNA-mediated gene editing makes use of endogenous, high fidelity repair pathways, which reduces the risk of error-prone end-joining causing additional mutations. With continuing concern regarding off-target effects of CRISPR/Cas951 and the finding that Cas9 proteins can illicit an adaptive immune response52, the safety profile of PNA/DNA editing may be particularly attractive, as avoiding off-target mutations is of exceptional importance during fetal development.

https://www.nature.com/articles/s41467-018-04894-2

Original Submission