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Updated: Jun 5, 2025

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A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
Published on: September 2, 2021
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CRISPR CLIP: comprehensive reviews on interventional studies using precision recombinant technologies: clinical
Swarali Yatin Chodnekar1, Zurab Tsetskhladze1
1Faculty of Medicine, University Geomedi, 3, King Solomon Street, Tbilisi 0114, Georgia.
Oxford Open Immunology
|December 11, 2024
Summary
This systematic review shows CRISPR gene editing is increasingly used in clinical trials for genetic diseases, primarily targeting cancer and blood disorders. Research is concentrated in the US and China, highlighting a need for broader global participation.
Area of Science:
- Biotechnology and Genetic Engineering
- Clinical Research
- Genomic Medicine
Background:
- CRISPR gene editing technology offers revolutionary potential for treating genetic diseases.
- Understanding the current landscape of CRISPR-based clinical trials is crucial for future research and development.
Purpose of the Study:
- To systematically review and consolidate clinical trials utilizing CRISPR technology for genetic diseases.
- To provide insights into the progress, trends, and geographical distribution of CRISPR clinical research.
Main Methods:
- Systematic review of randomized controlled trials (RCTs) from seven major databases up to March 2024.
- Inclusion criteria focused on therapeutic CRISPR applications for genetic diseases; exclusion criteria removed non-relevant studies.
- Methodological quality assessed using the CASP tool; data analyzed thematically.
Main Results:
- Identified 82 RCTs using CRISPR, with the United States and China leading in trial numbers.
- Cancer and monogenetic blood diseases (e.g., Thalassemia, sickle cell anemia) are the most targeted conditions.
- CTX001 and Cyclophosphamide were prominent biological agents; 2018 marked a peak year for clinical trials.
Conclusions:
- CRISPR technology shows burgeoning interest in therapeutic applications for genetic diseases.
- Geographical concentration of trials in specific countries and potential study oversights warrant attention.
- Current trials represent only a fraction of CRISPR's potential for treating genetic disorders, necessitating expanded global research efforts.
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