By MaryAnn Labant – Genetic Engineering & Biotechnology News –
As genome editing therapies move through clinical trials to regulatory approval, scientists continue the quest for the holy grail of large-scale DNA editing
Although genome editing was not a new concept, as zinc finger and TALEN platforms were already in use, the discovery of CRISPR-Cas9 shifted genome-editing research and clinical translation into high gear. But just like other platforms, this new kid on the block was not applicable to every editing situation for every genetic disease.
A longstanding desire in the field is a one-and-done, mutation-agnostic cure for genetic diseases that result from numerous mutations in a gene or from large-scale chromosomal structural variations, including deletions, duplications, inversions, and translocations. Gene therapies for these genetic indications require large-scale DNA manipulation, presenting different technical and regulatory challenges than correcting single-nucleotide point mutations.
