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Related Concept Videos

CRISPR01:59

CRISPR

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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...
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CRISPR/Cas9 Genome Editing01:28

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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...
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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
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Updated: Mar 12, 2026

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
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Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges.

Piao Yang1, Mohadeseh Khoshandam2,3, Iman Bhia4

  • 1Department of Molecular Genetics, College of Arts and Sciences, The Ohio State University, Columbus OH 43210, USA.

Asian Journal of Pharmaceutical Sciences
|March 11, 2026
PubMed
Summary

CRISPR gene editing offers revolutionary potential for treating genetic disorders, neurological diseases, and cancer. This review highlights innovative therapeutic designs and efficiency improvements for CRISPR-based treatments, addressing future challenges in clinical applications.

Keywords:
CRISPR/Cas systemsClinical trialsGene editingGenetic diseasesGenetic therapy

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Area of Science:

  • Biotechnology
  • Genomics
  • Molecular Biology

Background:

  • CRISPR gene editing technology has revolutionized genetic modification.
  • Its application holds significant promise for treating genetic disorders, neurological diseases, infectious diseases, and cancer.

Purpose of the Study:

  • To review current clinical trials utilizing the CRISPR/Cas system for various diseases.
  • To emphasize innovative targeting strategies and therapeutic designs for CRISPR applications.
  • To explore advancements in CRISPR editing efficiency, challenges, and future potential in managing genetic disorders and cancer.

Main Methods:

  • Systematic review of current clinical trials involving CRISPR/Cas technology.
  • Analysis of recent research focusing on CRISPR editing efficiency and therapeutic design.
  • Examination of innovative targeting conditions and approaches for CRISPR-based therapies.

Main Results:

  • CRISPR technology is rapidly expanding, with ongoing clinical trials exploring its therapeutic potential.
  • Emphasis is placed on novel targeting strategies and functional therapeutic designs beyond traditional delivery methods.
  • Research is actively increasing CRISPR editing efficiency for genetic disorders and cancer management.

Conclusions:

  • CRISPR-based therapies show transformative potential for future medical treatments.
  • Continued research into efficiency, targeting, and overcoming challenges is crucial for expanding clinical applications.
  • This review provides a unique perspective on advancing CRISPR technology for improved patient outcomes.