Immune cells edited with CRISPR/Cas9 to fight cancer seem to be safe and long-lasting. WorldCancerDay
CRISPR/Cas9 is a two-part molecular tool for cutting DNA. One part, a snippet of genetic material called a guide RNA, leads the DNA-cutting enzyme Cas9 to specific spots in DNA where researchers want to make a change. In this case, the team altered three genes in immune cells called T cells. The edits were aimed toward making the T cells more efficient than usual in killing cancer cells.
Most of the cuts were right on target, but the gene editor made some cuts the researchers didn’t intend. These “off-target” cuts plus deletions and rearrangements of some DNA were found in a few edited cells. For instance, the sloppiest guide RNA caused 7,778 on-target edits and only 38 off-target edits. In seven of these off-target instances, the unwanted edits landed in thegene. Those edits are probably not dangerous as that gene is not active in T cells anyway, the authors say.
Scientists worry that editing mistakes, deletions and rearrangements may inactivate genes that restrict cell growth or create cancer-promoting mutations. But edited T cells containing off-target edits in the study didn’t appear to grow abnormally. The researchers used versions of CRISPR/Cas9 that were available in 2016, but the gene editor has since been improved to increase efficiency and decrease off-target cutting. So future trials might have more accurate editing, the researchers say.
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