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CRISPR-mediated Gene Correction of Hematopoietic Stem Cells from Patients with X-linked Chronic Granulomatous Disease Using Non-Viral Cell Engineering






March 8, 2017
11am EST to 12pm EST

 

Sponsored by
MaxCyte

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Webinar Description:

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

Speakers:

Linhong LiLinhong Li

Director of Cell Engineering

MaxCyte, Inc.


Linhong Li is Director of Cell Engineering at MaxCyte, Inc., where he has worked for the past sixteen years. Dr. Li received a Bachelor of Science degree in Physics from the Changsha Institute of Technology, a Master degree in Biomedical Science in Hua Zhong University of Technology and Science, and a PhD in Biophysics from the State University of New York at Buffalo. During his time at MaxCyte, Linhong has spearheaded the development and clinical application of a non-viral gene delivery platform for use in human cell therapy.

Jessica CarmenJessica Carmen, PhD.

Director of Business Development Cellular Therapies

MaxCyte, Inc.


Dr. Carmen is the Director of Business Development for Cellular Therapy Partnerships at MaxCyte, Inc. MaxCyte is driving the next generation of cell-based medicines with its best in-class cell modification technology which is used in the discovery, development, and manufacture of small molecule, biologic, and cell-based medicines. She is responsible for the growth of partnerships with developers of ex vivo-modified cellular therapies. Dr. Carmen joined MaxCyte earlier this year after nearly 6 years at Lonza Inc.; a global leader of custom manufacturing for the pharmaceutical industry. In her previous role she was responsible for establishing partnerships with developers of cell-based medicines in order to expand Lonza’s cell therapy contract manufacturing business.

Dr. Carmen is co-chair of the Standards Coordinating Body (SCB) for Regenerative Medicines task force and has been instrumental in the establishment of the SCB. Currently, she is Secretary of the SCB and a member of the Board of Directors. She is an active member of the Science and Technology Committee of the Alliance for Regenerative Medicine (ARM) and has served on the Commercialization Committee for the International Society of Cellular Therapy (ISCT). Dr. Carmen earned a Doctorate from the Johns Hopkins University School of Public Health and a Bachelor of Science from the University of Florida.



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