Weekly reads: CRISPR sickle cell, Parkinson’s, pig-human chimera concerns

CRISPR gene editing has made rapid progress heading from bench to bedside. Perhaps the fastest has been its progress toward clinical use to combat sickle cell disease. We’ll start with a new paper on one major effort here.

CRISPR, gene editingCRISPR gene editing. This process often involves cutting DNA, which then can be used as an opening to make specific changes to the sequence.

CRISPR sickle cell

CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease, NEJM. There are several approaches to using gene editing for sickle cell disease, including inhibiting BCL11A to boost fetal hemoglobin. This paper reports on a Novartis Phase 1/2 sickle cell trial. The data are encouraging. They conclude: “CRISPR-Cas9 disruption of the HBG1 and HBG2 gene promoters was an effective strategy for induction of fetal hemoglobin. Infusion of autologous OTQ923 into three participants with severe sickle cell disease resulted in sustained induction of red-cell fetal hemoglobin and clinical improvement in disease severity.”

Previous similar clinical trial work using CTX001 by CRISPR Therapeutics and Vertex Pharmaceuticals has already been extremely encouraging as well.

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