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In Vivo Ovarian Cancer Gene Therapy Using CRISPR-Cas9

Zhi-Yao He1, Ya-Guang Zhang1, Yu-Han Yang1

  • 11 Department of Pharmacy, State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University , and Collaborative Innovation Center of Biotherapy, Chengdu, China.

Human Gene Therapy
|January 18, 2018
PubMed

Insights

A novel liposome delivery system effectively targets ovarian cancer cells using CRISPR-Cas9 technology to downregulate DNMT1, inhibiting tumor growth with fewer side effects than traditional chemotherapy.

Area of Science:

  • Biotechnology
  • Gene Therapy
  • Oncology

Background:

  • CRISPR-Cas9 genome editing shows promise for gene therapy but lacks safe delivery systems.
  • Ovarian cancer is a significant health concern, often developing resistance to treatments.

Purpose of the Study:

  • To develop and evaluate a folate receptor-targeted liposome (F-LP) for delivering CRISPR-Cas9 targeting DNA methyltransferase 1 (DNMT1) in ovarian cancer.
  • To assess the efficacy and safety of this novel therapeutic approach in preclinical models.

Main Methods:

  • A folate receptor-targeted liposome (F-LP) was engineered to encapsulate CRISPR plasmid DNA (gDNMT1) expressing Cas9 and single-guide RNA.
  • The F-LP/gDNMT1 complex was characterized for stability and safety.
  • In vitro and in vivo studies were conducted to evaluate DNMT1 mutation, downregulation, and therapeutic effects on ovarian cancer models.

Main Results:

  • F-LP efficiently complexed with gDNMT1, forming a stable and safe F-LP/gDNMT1 delivery system.
  • F-LP/gDNMT1 demonstrated successful in vitro mutation of endogenous DNMT1 and in vivo downregulation of DNMT1 expression.
  • Treatment with F-LP/gDNMT1 significantly inhibited tumor growth in both paclitaxel-sensitive and -resistant ovarian cancer models, with reduced adverse effects compared to paclitaxel.

Conclusions:

  • CRISPR-Cas9-mediated DNMT1 downregulation via F-LP delivery is a promising therapeutic strategy for ovarian cancer.
  • Lipid-based delivery systems offer a viable platform for precise genome editing therapeutics, advancing CRISPR-Cas9 applications in medicine.
Keywords:
CRISPRDNA methyltransferasegene deliveryovarian cancertargeted therapy

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