Genome editing technologies that install diverse edits can widely enable genetic studies and new therapeutics. Here we develop click editing, a genome writing platform that couples the advantageous properties of DNA-dependent DNA polymerases with RNA-programmable nickases (e.g. CRISPR-Cas) to permit the installation of a range of edits including substitutions, insertions, and deletions. Click editors (CEs) leverage the "click"-like bioconjugation ability of HUH endonucleases (HUHes) with single stranded DNA substrates to covalently tether "click DNA" (clkDNA) templates encoding user-specifiable edits at targeted genomic loci. Through iterative optimization of the modular components of CEs (DNA polymerase and HUHe orthologs, architectural modifications, etc.) and their clkDNAs (template configurations, repair evading substitutions, etc.), we demonstrate the ability to install precise genome edits with minimal indels and no unwanted byproduct insertions. Since clkDNAs can be ordered as simple DNA oligonucleotides for cents per base, it is possible to screen many different clkDNA parameters rapidly and inexpensively to maximize edit efficiency. Together, click editing is a precise and highly versatile platform for modifying genomes with a simple workflow and broad utility across diverse biological applications.
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http://dx.doi.org/10.1101/2023.09.12.557440 | DOI Listing |
Pharmaceut Med
January 2025
New York State Psychiatric Institute, 1051 Riverside Drive, New York, NY, 10032, USA.
Background And Objectives: Drug development in schizophrenia is limited by the differential scaling of the active treatment and placebo arms of a study, such that, as the number of sites increases, the magnitude of placebo response disproportionately increases. The objective of this article was to identify factors conducive to efficient recruitment as a step towards trial designs allowing recruitment of more participants per site, leading to reduced variability, and potentially a smaller placebo effect.
Patients And Methods: Using the information of 554 individuals, we calculated the percentage of individuals who were screened, consented, and retained in our research, along with rationale for nonconsent.
Cell Chem Biol
October 2024
Key Laboratory of Biomedical Polymers of Ministry of Education, College of Chemistry and Molecular Sciences, The Institute of Molecular Medicine, Wuhan University People's Hospital, Hubei Province Key Laboratory of Allergy and Immunology, Wuhan University, Wuhan 430072, Hubei, China.
A photocatalytic click chemistry approach, offering a significant advancement over conventional methods in RNA function modulation is described. This innovative method, utilizing light-activated small molecules, provides a high level of precision and control in RNA regulation, particularly effective in intricate cellular processes. By applying this strategy to CRISPR-Cas9 gene editing, we demonstrate its effectiveness in enhancing gene editing specificity and markedly reducing off-target effects.
View Article and Find Full Text PDFHealth Aff (Millwood)
October 2024
Laura M. Hahn is a general internist in Baltimore, Maryland. Patient names have been excluded or changed to protect privacy. Acknowledgments to Katherine A. Miller and Elizabeth McCloskey Miller for technical editing, language editing, and proofreading. To access the author's disclosures, click on the Details tab of the article online.
A physician reflects on what drove her out of primary care, and how it might be fixed.
View Article and Find Full Text PDFNpj Ment Health Res
September 2024
Center for Technology and Behavioral Health, Geisel School of Medicine, Dartmouth College, Lebanon, NH, USA.
Depression and anxiety frequently co-occur with opioid use disorder (OUD) yet are often overlooked in standard OUD treatments. This study evaluated the feasibility, acceptability, and preliminary effectiveness of a mobile application designed to address these symptoms in individuals receiving medications for OUD (MOUD). A randomized controlled trial recruited N = 63 adults with OUD who received MOUD and screened positive for moderate depression or generalized anxiety.
View Article and Find Full Text PDFCereb Circ Cogn Behav
August 2024
Department of Neuroimaging, Institute of Psychiatry, Psychology & Neuroscience, Kings College London. SE5 8AF, UK.
Brain health means optimal physiological brain function across the normal life-course. It encompasses not only healthy brain aging but also brain diseases, their diagnosis and treatment. In all these areas, molecular science has advanced our understanding.
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