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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

CRISPR/Cas9 Genome Editing

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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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What is Genetic Engineering?00:49

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Homologous Recombination02:31

Homologous Recombination

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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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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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Related Experiment Video

Updated: Jul 30, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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CRISPR/Cas9-based gene-editing technology for sickle cell disease.

Liangliang Ma1, Shanglun Yang1, Qianya Peng1

  • 1Department of Hematology, Meishan City People's Hospital, Meishan City, Sichuan Province 620000, China.

Gene
|May 14, 2023
PubMed
Summary

Sickle cell disease (SCD) treatment is limited, but gene therapy using CRISPR/Cas9 offers a promising cure. This review explores CRISPR/Cas9

Keywords:
CRISPRCas9Drug deliveryGene therapySickle cell disease

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Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Sickle cell disease (SCD) is a prevalent monogenic blood disorder caused by a specific β-globin gene mutation.
  • Existing treatments for SCD remain limited, with hematopoietic stem cell transplantation (HSCT) having restricted donor availability and utility.
  • Recent advancements in gene therapy, particularly CRISPR/Cas9 gene editing, present a revolutionary approach for treating SCD.

Purpose of the Study:

  • To review the pathogenesis and current therapeutic strategies for sickle cell disease.
  • To summarize the delivery methods for CRISPR/Cas9 gene editing systems.
  • To discuss the current status, challenges, and potential solutions for applying CRISPR/Cas9 in SCD treatment.

Main Methods:

  • Literature review of SCD pathogenesis and treatment options.
  • Summary of CRISPR/Cas9 delivery strategies.
  • In-depth analysis of CRISPR/Cas9 applications, barriers, and future directions for SCD.

Main Results:

  • CRISPR/Cas9 technology enables precise gene targeting, offering a potential cure for SCD.
  • Various delivery strategies for CRISPR/Cas9 are being explored for therapeutic applications.
  • Significant barriers exist in the clinical application of CRISPR/Cas9 for SCD, requiring further research and development.

Conclusions:

  • CRISPR/Cas9 gene editing holds significant promise as a curative therapy for sickle cell disease.
  • Overcoming delivery challenges and addressing application barriers are crucial for the successful clinical translation of CRISPR/Cas9 in SCD.
  • This review provides a foundation for future research and development in gene therapy for SCD.