Volume 5 Issue 2, February 2021

By using CRISPR and transposon constructs, pigs have been genetically modified to inactivate endogenous retroviruses and to enhance the compatibility of their organs with the human immune and coagulation systems.

The sustained expression of RNA-targeting Cas9 delivered intramuscularly or systemically by adeno-associated viral vectors eliminates pathogenic foci of expanded-repeat transcripts and reverses muscle-disease phenotypes in mouse models of myotonic dystrophy type 1.

Subretinal delivery of lentiviruses bearing Cas9 mRNA and a guide RNA targeting the Vegfa gene reduces the development of choroidal neovascularization in a mouse model of wet age-related macular degeneration.

Pigs can be genetically modified to inactivate endogenous retroviruses and to display enhanced compatibility with the human immune system using a combination of CRISPR–Cas9 and transposon technologies.

Lentiviruses co-packaging SpCas9 mRNA and an expression cassette encoding for a guide RNA targeting the Vegfa gene prevent mice from developing wet age-related macular degeneration induced by Vegfa. [Summary amended to correct the description of the packaged cargo.]

Molecular and physiological phenotypes in mouse models of myotonic dystrophy type I can be sustainably reversed by CRISPR-mediated targeting of toxic RNA repeats.

Subretinal viral delivery, in mice, of an adenine base editor and a single-guide RNA targeting a nonsense mutation in the Rpe65 gene restores near-normal levels of retinal and visual functions.

Cytidine base editors delivered to the liver of mice with phenylketonuria via adeno-associated viruses or lipid nanoparticles do not lead to detectable off-target edits in the RNA and DNA of hepatocytes.

A web tool enables the rapid design of prime-editing guide RNAs from reference and edited DNA sequences, for diverse prime-editing applications.