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ACTA 2. Gene Therapy and Editing for Smooth Muscle Dysfunction Syndrome. Smooth Muscle Dysfunction Syndrome is a rare disease with less than 50 known cases worldwide. It is caused by a specific genetic mutation in the ACTA2 gene that affects smooth muscle cells.
15 Μαρ 2024 · Highlights. Clustered regularly interspaced short palindromic repeats (CRISPR) activation (CRISPRa) technology allows pooled or targeted upregulation of gene expression. This new technical capacity helps to identify genes that, when upregulated, modify cell physiology and/or disease progression.
This gene encodes one of six different actin proteins. Actins are highly conserved proteins that are involved in cell motility, structure, integrity, and intercellular signaling. The encoded protein is a smooth muscle actin that is involved in vascular contractility and blood pressure homeostasis.
The primer mix has been tested to generate satisfactory qPCR data on ABI 7900HT by using the following PCR program: Stage 1: Activation: 50 °C for 2 min; Stage 2: pre-soak:95 °C for 10 min; Stage 3: Denaturation: 95 °C for 15 sec, Annealing: 60°C for 1 min; Stage 4: Melting curve: 95°C for 15 sec, 60°C for 15 sec, 95°C for 15 sec.
ACTA2 CRISPR Activation Plasmid (h) is a synergistic activation mediator (SAM) transcription activation system designed to specifically upregulate gene expression; ACTA2 CRISPR Activation Plasmid (h) consists of three plasmids at a 1:1:1 mass ratio: a plasmid encoding the deactivated Cas9 (dCas9) nuclease (D10A and N863A) fused to the ...
17 Φεβ 2023 · CRISPR-mediated transcriptional activation (CRISPRa) is a powerful technology for inducing gene expression from endogenous loci with exciting applications in high throughput gain-of-function...
α-smooth muscle actin, encoded by ACTA2 gene, is an isoform of the vascular smooth muscle actins, typically expressed in the vascular smooth muscle cells contributing to vascular motility and contraction. ACTA2 gene mutations cause a diversity of ...
