Crisprcas9

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Prompt

The schematic should include: 1. CRISPR-Cas9 Components – Cas9 protein bound to a single guide RNA; PAM (NGG) adjacent to the target sequence. 2. BCL11A Gene Structure – show the erythroid enhancer region within BCL11A. 3. DNA Cleavage Site – indicate the double-strand break within the enhancer sequence. 4. Downstream Effect – reduced BCL11A → derepressed γ-globin → increased fetal hemoglobin (HbF). Safety Consideration – off-target editing is a key FDA concern; genome-editing therapies must demonstrate minimal unintended edits and thorough long-term monitoring