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Published on: September 2, 2021
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.
Abstract:
To consolidate clinical trials that utilized the CRISPR technology to synthesise cures for various genetic diseases as a means to provide a window into the progress made so far while paving the way forward for future research and practices. Systematic review (PROSPERO CRD42023479511). Trials from seven databases' (ClinicalTrials.gov, European Union Clinical Trials Registry, ISRCTN registry, ICTRP/trialsearch.who.int, ChiCTR.org.cn, Clinical Trial Registry India, and Cochrane Library/Trials) inception to 9 March 2024, were considered. Exclusion criteria were unrelated, duplicated, non-English, unavailable full texts, diagnostic studies, correlational studies, observational studies, abstract-only papers, reviews or conference papers. Included studies were appraised using the ten-item CASP tool to assess methodological quality. The review identified 82 RCTs utilizing CRISPR and revealed four main themes: Diseases targeted, Countries of Clinical trials, Type of interventions, and Trial trends over the years. Geographically, the United States and China lead in the number of CRISPR clinical trials, followed by the European Union. However, Africa, Asia, and South America have very few trials. Among disease classes, cancer is the most prevalent focus with 39 studies, followed by monogenetic blood diseases, like Thalassemia and sickle cell anaemia. The biological agent CTX001 and Cyclophosphamide each feature in 11 studies. The peak year for clinical trials was 2018, marked by a significant increase with 16 studies conducted. Despite conducting a comprehensive search, the majority of trials were concentrated in the United States and China. Additionally, potential oversights due to vague titles, English-only studies, and indexing issues may have occurred. Nonetheless, by incorporating data from seven distinct databases, this review significantly contributes to understanding CRISPR's utilization in therapeutic clinical trials, paving the way for future research directions. The review underscores the burgeoning interest in CRISPR-based interventions. Current trials barely tap CRISPR's potential for treating genetic diseases.
Insights
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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