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CRISPR-Cas9: Targeted Genome Editing

CRISPR-0 D Cas9 TARGETED GENOME EDITING This powerful genome editing technique uses "genomic scissors" to add, disrupt, or delete sequences of specific genes. Cas9 PAM Target Guide RNA DNA DNA-cutting enzyme Complement to DNA Target DNA Modified DNA Protospacer Cas9 Protein DNA-cutting enzyme Cas9 binds toa guide RNA strand that is complementary to the DNA sequence of interest. The RNA-guided CRISPR/Cas9 complex binds to the target DNA and makes site specific breaks in the DNA, achieving targeted mutagenesis. Guide RNA Transfect into cell DNA-cutting Enzyme Target DNA Double-Stranded Break Disrupt a gene of interest Modify a specific part of genome DNA Template (No insert provided) Knock-out mutation Knock-in or gene replacement Therapeutics: This technology has the potential to cure many human genetic diseases, including sickle cell anemia, clotting diseases, muscular dystrophy, and blindness. Most recently, this technique was proposed to treat people with "bubble boy" disease, a genetic immunodeficiency dis order that could be reversed with gene therapy. Animal models: This technique offers a faster and easier way to create mice and other animals with specific genetic differences compared to traditional genetic manipulations. This technology works in many different organisms, but an effective delivery method still needs to be developed in order for it to be safe and effective, especially inhuman cells. REFERENCES: - Hsu PD, Lander ES, Zhang F. Development and applicatians of CRISPR Cas9 for gename engineering. Cel. 2014Jun S157(6:1262-78. - Fineran PC, Dy RL Gene regulation by engineered CRISPR-Cas systems. Cur Opin Marobiol 2014 Apr1883-9. doi 10106/jmb.2014.02.007. Epub 2014 Mar 15. Review. - Doudna JA, Charpenier E Genome editing. The new frontier of gename engineering with CRISR-Cas9. Science. 2014 Nov28,346(6213)1258096. doilan26Acience1258096. Review. © Knowing Neurons http://knowingneurons.com Current Hurdles: Applications: ..... .......

CRISPR-Cas9: Targeted Genome Editing

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This neuroscience technology may be the key to treating disease

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