Bionano: Alternative to KT and CMA for Evaluating CRISPR-Edited Cells in Stem Cell Therapy

 Bionano Genomics Inc. (Nasdaq: BNGO) today announced a publication in Nature Communications demonstrating the utility of optical genome mapping (OGM) as an alternative to karyotyping (KT) and chromosomal microarray (CMA) for the evaluation of CRISPR-Cas9 edited human induced pluripotent stem cell (iPSC) lines in order to uncover possible pathogenic structural alterations that may limit their usefulness for stem cell therapy.

The study authors found that approximately 15% of CRISPR-Cas9 edited genomes (2 of 13) had potentially pathogenic large chromosomal deletions at unexpected off-target sites. In addition to those two off-target deletions, the authors reported a large, unexpected deletion at the target site.

“This study is an example of how OGM can be used as part of developing cell and gene therapies, including stem cell therapies.  The expansion of iPSC-mediated cell therapy faces risks due to off-target structural variations that may be introduced during CRISPR-Cas9 genome editing. Since genome aberrations caused by CRISPR-Cas9 editing could lead to unforeseen adverse effects, we believe careful and comprehensive analysis of edited genomes is important in developing these therapies and their manufacture," commented Erik Holmlin, PhD, president and chief executive officer of Bionano.

The paper is available at: https://www.nature.com/articles/s41467-023-40901-x.

https://www.biospace.com/article/releases/bionano-announces-publication-demonstrating-utility-of-ogm-as-an-alternative-to-kt-and-cma-for-evaluating-crispr-edited-cells-as-part-of-stem-cell-therapy-development/