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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...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
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...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
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...

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Related Experiment Video

Updated: Jul 2, 2026

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

CIRCLE-Seq for Interrogation of Off-Target Gene Editing

Published on: November 1, 2024

Precision gene editing: From proof-of-concept to curative therapies.

Tongtong Cui1, Bojin Li2, Bingyu Cai2

  • 1Key Laboratory of Organ Regeneration an Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China; Beijing Institute for Stem Cell and Regenerative Medicine, Beijing 100101, China.

Trends in Molecular Medicine
|June 30, 2026
PubMed
Summary

Gene therapy advances from gene addition to precise genome editing, correcting mutations with technologies like CRISPR-Cas. This evolution promises safer, personalized cures for genetic diseases.

Keywords:
AIbase editinggene therapypersonalized medicineprecision genome editingprime editing

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Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Medical Science

Background:

  • Gene therapy is transitioning from simple gene addition to sophisticated genome editing.
  • Technologies like CRISPR-Cas, base editors, and prime editors enable precise correction of disease-causing mutations.

Purpose of the Study:

  • To review the progression of precision genome editing technologies.
  • To discuss their clinical applications from ex vivo to in vivo treatments.
  • To highlight the role of personalized medicine and emerging challenges.

Main Methods:

  • Review of advancements in gene editing technologies (CRISPR-Cas, base editors, prime editors, transposases).
  • Analysis of clinical applications, including ex vivo and in vivo strategies.
  • Discussion of personalized medicine approaches for rare diseases.
  • Examination of clinical trial progress, delivery methods, and AI's role.

Main Results:

  • Precision editing technologies are reshaping the therapeutic landscape for genetic diseases.
  • Ex vivo and in vivo applications are expanding, targeting various organs.
  • Personalized medicine, including N-of-1 therapies, is emerging for rare conditions.
  • AI is being integrated to optimize editing tools and predict outcomes.

Conclusions:

  • Genome editing technologies offer the potential for safer, more durable, and personalized cures.
  • Addressing delivery and accessibility challenges is crucial for widespread clinical adoption.
  • The field is rapidly advancing, transforming genetic medicine and offering new hope for patients.