Gene editing: DNA versus RNA (Drug Discovery News)


Researchers correcting the genetic causes of disease at the DNA or RNA level make their cases.

Treating disease is often a matter of restoring the function of a specific protein, process, or pathway. But when a disease manifests in cellular chaos too widespread to correct or in drug targets that are lost altogether, researchers may resort to remedying its genetic origin. These gene therapies can deactivate aberrant genes with strategic breakages, replace mutation-ridden regions of genes, and even edit individual nucleotide bases.    

While gene editing technologies such as CRISPR-Cas9 go straight for the DNA source, the often overlooked intermediate in the DNA-to-protein pipeline is attracting attention as an alternate editing canvas. Robert Bell, a gene therapy scientist at Ascidian Therapeutics, is one of a group of researchers developing platforms to rewrite genes at the RNA level. Gregory Davis, a genome engineer at Sangamo Therapeutics, is among scientists advancing DNA editing systems. These two researchers lay out the advantages, disadvantages, and unique applications of each approach, revealing that together, they may broaden the scope of genes researchers can edit and diseases gene therapy can treat.

Read the full article on the Drug Discovery News website