CRISPR 2.0: Breakthrough in Gene Editing
CRISPR is a powerful gene-editing technique that functions like a molecular cut-and-paste tool, removing and replacing defective gene segments in our DNA. It seems, now, that the revolutionary biotechnology is getting an upgrade.
Almost half of all genetic diseases are caused by point mutations. These come down to mix-ups in the nucleobase pairs between the chemicals that make up DNA: adenine (A), cytosine (C), guanine (G), and thymine (T). The efficacy of CRISPR is therefore limited since it targets entire sections of DNA, not single base pairs.
As a potential solution, researchers from the Broad Institute at MIT and Harvard present modified CRISPR technologies. One of these is the Adenine Base Editor (ABE) which makes neater edits possible by rearranging A atoms to resemble G ones, prompting A-T base pairs to becomes G-C instead.
The other is the development of Cas13 - a CRISPR protein that makes editing of RNA possible. Unlike DNA editing, which makes permanent changes to the genome, RNA editing has shorter lasting effects. It is achieved by swapping adenosine with inosine, recognized as guanine by cells.
These recent advances in gene-editing create optimism for the future of gene therapy. It is a promising field which also fosters progress in areas such as nanotechnology, biomolecular engineering, and molecular biology. As a result, pharmaceutical companies are investing billions into the industry, and the cost of gene therapy is declining.
Professor Liu from the Broad Institute is hopeful that "CRISPR 2.0" has a broad set of potential therapeutic applications. His lab next plans to explore base-editing technology in the treatment of blood disorders, neurological disorders, hereditary deafness, and hereditary blindness.