Remove In-Vivo Remove Packaging Remove Reagent Remove RNA
article thumbnail

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.

DNA 98
article thumbnail

Delivering on the promise of gene editing

Drug Discovery World

Prime editing, while also employing a single strand nick, expands this scope to all 12 possible changes using a more complex prime editing guide RNA (pegRNA). Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo.