Special Focus Issue Part II: Recruitment of solid lipid nanoparticles for the delivery of CRISPR/Cas9: primary

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A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
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A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

Published on: August 25, 2017

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Hasan Akbaba1, Gulsah Erel-Akbaba2, Serif Senturk3,4

  • 1Department of Pharmaceutical Biotechnology, Faculty of Pharmacy, Ege University, Izmir, 35100, Turkey.

Nanomedicine (London, England)
|May 10, 2021
PubMed

Insights

Researchers developed biocompatible, nonviral solid lipid nanoparticles (SLNs) for CRISPR/Cas9 gene editing. These SLNs show promise for efficient anticancer therapy delivery, demonstrating good transfection efficiency and low toxicity.

Area of Science:

  • Biotechnology
  • Nanomedicine
  • Gene Therapy

Background:

  • CRISPR/Cas9 gene editing holds potential for cancer therapy.
  • Efficient and safe delivery of CRISPR/Cas9 remains a significant challenge.
  • Nonviral, biocompatible delivery systems are crucial for clinical translation.

Purpose of the Study:

  • To develop and characterize solid lipid nanoparticles (SLNs) for CRISPR/Cas9 delivery.
  • To evaluate the cytotoxicity and transfection efficiency of SLNs carrying CRISPR/Cas9.
  • To establish a foundation for targeted anticancer SLN-based therapies.

Main Methods:

  • Solid lipid nanoparticles (SLNs) were formulated using pseudo and 3D ternary plots.
  • SLNs and their complexes with PX458 plasmid DNA were characterized.
  • Cytotoxicity and transfection efficiency were assessed in vitro.

Main Results:

  • Formulated SLNs were nanosized, monodispersed, stable, and exhibited no significant toxicity.
  • SLN/CRISPR-Cas9 complexes demonstrated comparable transfection efficiency to the positive control.
  • The developed SLNs provide a viable platform for gene delivery.

Conclusions:

  • Solid lipid nanoparticles offer a promising, biocompatible, and nonviral delivery system for CRISPR/Cas9.
  • Further optimization of SLN production parameters can lead to targeted anticancer properties.
  • This approach facilitates the advancement of CRISPR/Cas9 gene editing into preclinical anticancer studies.
Keywords:
3D ternary plotCRISPR/Cas9PX458gene deliverysolid lipid nanoparticletransfection

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