In vivogene editing has the potential to permanently cure genetic diseases. Unlike some editing technologies that have been designed as research tools and subsequently engineered for therapeutic use, ARCUS is a precise and versatile genome editing technology with the distinct potential to insert, delete...
Eligo Bioscience is deploying in vivo gene editing technologies against microbiome targets to address diseases with high unmet needs.
In vivo gene editing in post-mitotic neurons of the adult brain may be a useful strategy for treating neurological diseases. Here, we develop CRISPR–Cas9 nanocomplexes and show they were effective in the adult mouse brain, with minimal off-target effects. Using this system to targetBace1suppres...
Removing one or more exons from the mutated transcript can produce an in-frame mRNA and a truncated, but still functional, protein. In this study, we developed and tested a direct gene-editing approach to induce exon deletion and recover dystrophin expression in the mdx mouse model of DMD. ...
Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits ...
single mouse. Horizontal bars indicate mean, statistical analysis by Student’s unpairedt-test, *P<0.05. (c) Deep-sequencing analysis to quantify the frequency of targeted gene editingin vivoin CD117+ cells from BM and spleen of β-globin/GFP mice treated as above. Error bars indicate ...
Alternatively, Cas9 orthologs from other species such as SaCas9 (Fig.2g) with a longer PAM requirement of 5′-NNGRRT (R = A or G) can be used to mediate gene editing in vivo with a similar efficiency and specificity to SpCas9 [76,77]. The advantage of a smaller-sized SaCas9 ...
Non-viral CRISPR/Cas gene editing in vitro and in vivo enabled by synthetic nanoparticle co-delivery of Cas9 mRNA and sgRNA Angew Chem Int Ed Engl, 56 (2017), pp. 1059-1063 CrossrefView in ScopusGoogle Scholar 28 Y. Li, T. Thambi, D.S. Lee Co-delivery of drugs and genes using pol...
Researchers and drug developers are increasingly leveraging this flexibility to deliver other types of molecules with therapeutic value—alone or sometimes in combination—including regulatory RNAs (for example, short hairpin RNAs [shRNAs]), vectorized antibodies, and substrates for gene editi...
mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy Large genes require dual adeno-associated viral (AAV) vectors for in vivo delivery/expression, but current methods have limitations. Here the authors develop and functionally evaluate REVeRT, an efficient and flexible dual...