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Related Concept Videos

CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...

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Updated: Jun 27, 2026

CIRCLE-Seq for Interrogation of Off-Target Gene Editing
08:23

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Published on: November 1, 2024

Building CRISPR-Based Gene-Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics.

Sebastian Hernandez Rodriguez1, Toshifumi Yokota1

  • 1Department of Medical Genetics, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Genes
|June 26, 2026
PubMed
Summary

Advanced CRISPR gene editing tools like base and prime editors offer precise genetic therapies for rare diseases. This review explores their potential, challenges, and the future of interventional genetics for personalized treatments.

Keywords:
CRISPRgene editinginterventional geneticsmonogenic disorderspersonalized medicine

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Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Molecular Biology

Background:

  • CRISPR technology has evolved from double-strand break methods to precise base and prime editors.
  • This shift enables targeting a wide range of disease-causing mutations, particularly in rare monogenic disorders.
  • Emerging CRISPR modalities represent a move towards platform-based therapeutic systems.

Purpose of the Study:

  • To review the technological progression of CRISPR systems, including base and prime editors.
  • To explore the applications, limitations, and translation of these technologies into personalized gene therapies.
  • To introduce the concept of interventional genetics and outline strategies for scalable, regulatory-ready gene-editing platforms.

Main Methods:

  • Review of recent advancements in CRISPR-based genome editing technologies.
  • Analysis of principles, applications, and limitations of base and prime editing.
  • Discussion of challenges in gene therapy delivery, safety, and regulatory pathways.

Main Results:

  • CRISPR base and prime editors allow precise, programmable DNA sequence modifications.
  • These advanced editors hold significant promise for treating a broad spectrum of rare monogenic diseases.
  • The review highlights key challenges including delivery, off-target effects, and regulatory hurdles.

Conclusions:

  • CRISPR technology is advancing towards versatile, platform-based therapeutic systems for rare genetic disorders.
  • Interventional genetics offers a framework for linking genomic diagnosis to gene-editing therapies.
  • Developing scalable, regulatory-ready platforms is crucial for accelerating clinical implementation of individualized gene therapies.