CRISPR (clustered regularly interspaced short palindromic repeats)-mediated gene modification presents a powerful avenue for the treatment of patients with genetic diseases. During this webinar, we will discuss the application of CRISPR/Cas9 gene editing using a non-viral cell engineering technology as a viable means of patient treatment.

X-linked chronic granulomatous disease (X-CGD) patients have recurrent infections and autoinflammation resulting from CYBB (encodes gp91phox) mutations that impair phagocyte oxidase activity. We demonstrate efficient CRISPR-mediated repair of a mutation in the CYBB gene of CD34⁺ hematopoietic stem and progenitor cells (HSPCs) from patients with X-CGD using MaxCyte’s cGMP-compliant, electroporation-based delivery platform.

Characterization of gene-corrected patient HSPC cells was performed including in vitro NADPH (nicotinamide adenine dinucleotide phosphate) oxidase functional assays and in vivo mouse engraftment studies. The level of targeted gene correction persisting long term in our mouse xenograft studies suggests that CRISPR-mediated gene correction of hematopoietic stem cell using a non-viral method of delivery is a feasible therapeutic option for treatment of X-CGD patients with the c.676C>T mutation.

Learning Objectives:

  • Efficient delivery of CRISPR/Cas9 gene editing components into HSPCs using MaxCyte’s non-viral cell engineering technology
  • Optimization of CRISPR/Cas9 gene correction of patient CD34⁺ HSPCs
  • Successful gene correction of a mutation in the CYBB gene of HSPCs from patients with X-CGD
  • Short and long term analysis of in vivo engraftment of corrected HSPCs

Presenters: Jessica Carmen, PhD., Director of Business Development Cellular Therapies & Linhong Li, PhD., Director of Cell Engineering, MaxCyte, Inc.

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