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| A A, le .E-' f :-. Il Text Direction. Shape Fin- usab. . Aa. IA-|, li- Fr Align Text- convert to SmartArt .|Inn( 2 Shape OutineR }EArrangestylek,@shape Enects 'I Font sei Paragraplh Drawing Edit Homework 1-Find an article using crispR/Cas9 to perform knockout, and discuss the discovery/conclusion. Write the reference of the article. Design gDNA sequence for TP53 gene knockout. Design gDNA at this website to https://www.atum.bio/eCommerce/cas9/input 3-Describe your step-wise cloning strategy in construction CRISPR/Cas9 plasmid, transfection, and screening.Explanation / Answer
Using CRISPR/Cas9-Mediated GLA Gene Knockout as an In Vitro Drug Screening Model for Fabry Disease
The Genome-editing system, CRISPR/Cas9 has announced hopeful ability for producing mutation in gene, deletion, and correction in human cells. Application of this useful method in Fabry disease (FD). Enzyme replacement therapy (ERT), a usual control of human recombinant Gal A (rh-GLA), is now available and productive treatment for FD patients to clear the deposited Gb3. Moreover, the short half-life of human body rh-GLA restricts its application. Moreover, absence of a suitable in vitro model of disease limited the drug's high-throughput screening for developing ERT efficacy. Using gene knockout mediated by CRISPR/Cas9 of GLA in HEK-293T cells, we produce GLA-null cells to examine rh-GLA cellular pharmacokinetics.
Together the GLA-knockout HEK-293T cells mediated by CRISPR/Cas9 give invitro model of FD calculating the intracellular rh-GLA pharmacokinetics also for screening candidates to prolong potency of rh-GLA.
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