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10.1136/bmjdrc-2021-002363 14 BurchallG

American Journal of Cardiology, 51(9), 14891491

146,147,148,149 The CRISPR-mediated gene-editing system primarily involves Cas9 and single guide RNA (sgRNA) to introduce DNA double-strand breaks (DSBs) at specific positions in the genome

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Tip of the iceberg The tip of the metabolic iceberg conveys the clinical, anatomical, and pathological features for each neurodegenerative disorder, the most visible of which are the hallmark features