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Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein

Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR–Cas9 ribonucleoprotein

Technology tamfitronics Main Clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9-based genome editing1,2,3 has the potential to provide wide-ranging treatments for genetic diseases4,5,6 if safe and effective methods for delivering CRISPR-based therapeutics can be developed7,8. Although viral delivery of CRISPR genome editors is the most widely used method for in vivo cell editing9,10,11, viral vectors can […]

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Click editing enables programmable genome writing using DNA polymerases and HUH endonucleases

Click editing enables programmable genome writing using DNA polymerases and HUH endonucleases

Technology tamfitronics Doudna, J. A. The promise and challenge of therapeutic genome editing. Nature 578229–236 (2020). Article CAS PubMed PubMed Central Google Scholar Anzalone, A. V., Koblan, L. W. & Liu, D. R. Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors. Nat. Biotechnol. 38824–844 (2020). Article CAS PubMed Google Scholar van Overbeek, […]

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Precise in vivo RNA base editing with a wobble-enhanced circular CLUSTER guide RNA

Precise in vivo RNA base editing with a wobble-enhanced circular CLUSTER guide RNA

Technology tamfitronics Main Site-directed adenosine-to-inosine (A-to-I) RNA base editing is a very promising technology with a clear path for clinical application1,2. Hydrolytic deamination of A by enzymes of the ADAR family (adenosine deaminases acting on RNA) produces an inosine, which is biochemically interpreted as G in many cellular processes such as splicing or translation and, […]

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