CRISPR-Cas9 gen düzenleme sistemi ve hastalıklar için tedavi yaklaşımı
Anahtar Kelimeler:
CRISPR-Cas9, genom düzenleme, insan hastalıkları, terapötiklerÖzet
Kümelenmiş, düzenli aralıklarla kısa palindromik tekrar (CRISPR'ler) dizileri, birçok bakterinin ve neredeyse tüm arkelerin genomlarında yaygındır. CRISPR/Cas9 gen düzenlemesi için güçlü bir teknolojidir. Yüksek özgüllüğü ve etkinliği nedeniyle, CRISPR/Cas9, çoklu genetik değişiklikleri içeren insan hastalıklarının tedavisinde, özellikle kanser için yaygın olarak kullanılmaktadır. CRISPR/Cas9 tarafından farklı hastalıkların tedavisi kavramları oluşturulmuştur. Bununla birlikte, klinik uygulamaları için önemli zorluklar devam etmektedir. Terapötik potansiyelin yanı sıra, CRISPR-Cas9 aracı, ilaç keşfi ve geliştirilmesi için genetik olarak engellenmiş hayvan modelleri oluşturmak için de uygulanabilir. Bu derleme makalesi, CRISPR-Cas9 sistemlerinin kökenini ve kanser, alerji, immünolojik bozukluklar, Duchenne kas distrofisi, kardiyovasküler bozukluklar, nörolojik bozukluklar, kistik fibroz, gözle ilgili bozukluklar dahil olmak üzere çeşitli genetik bozukluklara karşı terapötik potansiyellerini tartışmaktadır.
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