CRISPR-Cas9

[Hepatology] Researchers summarize the preclinical data obtained in animal models treated with mRNA as a therapeutic agent and discuss the different gene editing strategies applied to the treatment of liver diseases, highlighting both their therapeutic efficacy as well as safety concerns.

[Journal of the American Society of Nephrology] Scientists used CRISPR-Cas9 gene editing of iPSCs to develop humanderived APOL1G0/G0 and APOL1G2/G2 kidney organoids on an isogenic background, and performed bulk RNA sequencing of organoids before and after treatment with IFN-γ.

[Kidney International] Kidney organoids may complement animal models for annotating and predicting the molecular and cellular function of these candidate genes.

[Intellia Therapeutics, Inc.] Intellia Therapeutics, Inc. announced that the first patient has been dosed with NTLA-5001, the company’s ex vivo CRISPR/Cas9 genome editing candidate for the treatment of AML.

[Cell Reports] Scientists used maturity-onset diabetes of a young 3 patient and CRISPR/Cas9-engineered human induced pluripotent stem cells (hiPSC) grown as 3D organoids to investigate how HNF1αp291fsinsC affected hiPSC differentiation during pancreatic development.

[Molecular Therapy-Methods & Clinical Development] Scientists proposed adoptive transfer of SARS-CoV-2-specific T cells made resistant to a common immunosuppressant, Tacrolimus, for optimized performance in the immunosuppressed patient.