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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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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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Gene Therapy00:59

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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...
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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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Updated: Jun 13, 2025

Production of Human CRISPR-Engineered CAR-T Cells
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Advances in CRISPR/Cas systems-based cell and gene therapy.

Arpita Poddar1, Farah Ahmady2, Prashanth Prithviraj2

  • 1Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia; RMIT University, VIC, Australia.

Progress in Molecular Biology and Translational Science
|September 12, 2024
PubMed
Summary

CRISPR gene editing offers precise tools for cell and gene therapy, advancing treatments by targeting genetic diseases. This technology explores various delivery methods and engineered variants for new medical breakthroughs.

Keywords:
CRISPRCasCellGeneNon-viral vectorTherapyViral vector

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Area of Science:

  • Biomedical Science
  • Genetics
  • Molecular Biology

Background:

  • Cell and gene therapy address diseases at their genetic roots.
  • CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology provides precise genome editing capabilities.
  • CRISPR has become a pivotal tool in advancing biomedical research and therapeutic strategies.

Purpose of the Study:

  • To explore the foundational role and historical development of CRISPR in gene editing.
  • To discuss diverse CRISPR biomolecular formats (plasmid, RNA, protein) and delivery systems (viral, non-viral).
  • To examine engineered CRISPR variants and their emerging clinical applications in gene therapy.

Main Methods:

  • Historical review of CRISPR development.
  • Analysis of various CRISPR biomolecular formats.
  • Examination of viral and non-viral CRISPR delivery systems.
  • Review of engineered CRISPR variants and their therapeutic potential.

Main Results:

  • CRISPR technology has evolved significantly, offering versatile genome editing tools.
  • Multiple CRISPR formats and delivery methods enhance its applicability in gene therapy.
  • Engineered CRISPR variants show promise for targeted genetic interventions.

Conclusions:

  • CRISPR systems are revolutionizing cell and gene therapy by enabling precise genetic modifications.
  • Advancements in CRISPR delivery and engineering are paving the way for breakthrough medical treatments.
  • The integration of CRISPR technology holds immense potential for treating genetic diseases.