CRISPR-Cas9

[Molecular Psychiatry] Investigators used iPSC technology to obtain hippocampal neurons to investigate the neuropathology of IQSEC2-mediated disease. The neurons were characterized at three-time points during differentiation to assess developmental progression.

[Intellia Therapeutics, Inc.] Intellia Therapeutics, Inc. announced that the US FDA has accepted the investigational new drug application for NTLA-5001, the company’s first wholly-owned ex vivo CRISPR genome editing candidate for the treatment of cancer.

[Stem Cell Reports] Researchers demonstrated efficient editing of master transcription factor (TF)Myod1 locus using the CRISPR/Cas9/AAV9-sgRNA system in juvenile satellite cells (SCs). Application on two key TFs, MYC and BCL6, unveiled distinct functions in SC activation and muscle regeneration.

[Scientific Reports] Scientists developed a non-cancer lineage with CRISPR-Cas9-mediated H19 knockdown and observe the changes in a cellular context. To edit the promoter region of H19, two RNA guides were designed, and the murine C2C12 myoblast cells were transfected.

[Scientific Reports] The authors optimized a CRISPR/Cas9 gene edition protocol and used it in wild type immortalised myoblasts to delete exon 52 of the dystrophin gene, modeling a common Duchenne muscular dystrophy mutation; and in patient’s immortalised cultures they deleted an inhibitory microRNA target region of the utrophin UTR, leading to utrophin upregulation.

[Molecular Therapy] Researchers co-delivered CRISPR-Cas9 and a donor DNA sequence to insert the missing human exon 52 into its corresponding position within the Duchenne muscular dystrophy gene and achieved full-length dystrophin correction in skeletal and cardiac muscle.