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Delivering on the promise of gene editing

Drug Discovery World

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Likewise, Lessard et al.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Incorporating in vivo pooled CRISPR screens has allowed for the interrogation of thousands of genetic perturbations in cells, leading to the determination of the involvement of molecules (PTPN2, ADAR1, or DHX37) in immune checkpoint inhibitor (ICI) responsiveness and T cell activation.

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