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  1. This work involves extensive characterization of disease models as well as optimizing gene targeted therapy administration. We are also working to investigate the use of gene editing for in vivo application to edit the ACTA2 gene to provide the diseased cells with a correct and permanent ACTA2 gene.

  2. MINDY2/CINDY is an algorithm for the genome-wide discovery of modulators of transcriptional interactions. It offers a new information theoretic method to identify multivariate statistical dependencies between a transcription factor and one or more of its targets, conditional on the presence (or absence) of a candidate modulator gene.

  3. 10 Απρ 2017 · Therapeutic gene editing can be administered through two basic strategies: (1) direct in vivo delivery of a gene-editing nuclease and (2) delivery of cells engineered ex vivo to contain a...

  4. 1 Ιουλ 2024 · The purpose of this paper is three-fold: first, a chronological description of the history of CRISPR-Cas9-sgRNA-based gene editing; second, a brief description of the current state of clinical research in hematologic diseases, including selected applications in treating hematologic diseases with CRISPR-based gene therapy, preceded by a brief des...

  5. 10 Μαρ 2015 · Individuals with ACTA2 mutations present with both type A and type B aortic dissections at significantly younger ages than individuals enrolled in the International Registry of Acute Aortic Dissection (IRAD) study 22 and are likely to report a family history of thoracic aortic disease.

  6. 29 Δεκ 2021 · Liu et al. review the development of the CRISPR-Cas toolbox and gene editing technologies, including the Cas nucleases used for gene editing as well as CRISPR-Cas-mediated DSB-dependent and DSB-independent gene editing tools, with an emphasis on precision tools and CRISPR-Cas-related technologies.

  7. Genome-editing technologies. Cartoons illustrating the mechanisms of targeted nucleases. From top to bottom: homing endonucleases, zinc-finger nucleases (ZFNs), transcription activator-like effector (TALE) nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9).

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