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"Electroporation-based delivery for gene editing: A strategy for familial hypercholesterolemia"

Tuesday, October 14, 2025, 9:00 AM Pacific Daylight Time

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder marked by severely elevated cholesterol and early atherosclerotic cardiovascular disease. Liver transplantation is the only curative option, but it is limited by donor shortages, procedural risks, and lifelong immunosuppression.

Over three decades ago, the first trial of ex vivo hepatocyte transplantation for HoFH failed to achieve sufficient lipid lowering. Although CRISPR–Cas9 now enables efficient gene editing, robust hepatocyte engraftment remains a major barrier.

To overcome this, our laboratory is developing non-viral ex vivo strategies to edit hepatocytes and applying selective pressure to expand corrected cells in the liver. We used electroporation to deliver Cas9 ribonucleoproteins targeting Cypor into hepatocytes, followed by transplantation with transient acetaminophen administration in a mouse model of HoFH.

Hepatocytes isolated using the gentleMACS Octo Dissociator showed strong engraftment, underscoring its potential to advance cell-based gene editing therapies in preclinical models. 

In this webinar, you’ll learn:

• The challenges and limitations of current therapies for homozygous familial hypercholesterolemia
• Insights into innovative CRISPR-mediated gene editing strategies 
• How the gentleMACS Octo Dissociator system enhances preclinical studies of ex vivo gene editing in mouse models of liver disease

Who should attend:

• Gene therapy scientists
• Gene editing scientists
• Liver disease experts
• Liver cell therapy researchers
• Hereditary tyrosinemia researchers
• Hepatocytes and liver researchers

Offered Free by: Miltenyi Biotec
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