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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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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.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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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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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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Related Experiment Video

Updated: Oct 18, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

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Innovations in CRISPR-Based Therapies.

Gokul Kesavan1,2

  • 1Vowels Lifesciences Private Limited, 271, 5th Main Rd, 4th Block, Jayanagar, Bengaluru, Karnataka, 560011, India. gokul.kesavan@vowelslifesciences.com.

Molecular Biotechnology
|September 29, 2021
PubMed
Summary
This summary is machine-generated.

CRISPR gene editing shows promise for treating genetic diseases like sickle cell anemia and blindness. Both in vivo and ex vivo approaches are advancing, but challenges in safety, cost, and delivery remain for widespread clinical use.

Keywords:
Base editorsCRISPR therapyGene therapyGenome editingMutagenesis

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Area of Science:

  • Biotechnology
  • Genetics
  • Medical Research

Background:

  • Gene and cell therapies have advanced significantly in recent years.
  • CRISPR-edited stem cells are successfully treating blood disorders and progressing in clinical trials for blindness.
  • CRISPR technology offers precise gene editing capabilities.

Purpose of the Study:

  • To review the current status of CRISPR-based therapies, including in vivo and ex vivo approaches.
  • To assess the challenges and opportunities for clinical translation of CRISPR therapies.
  • To discuss the safety, affordability, and feasibility of CRISPR therapies for broad patient populations.

Main Methods:

  • Review of existing literature on CRISPR-based gene and cell therapies.
  • Analysis of in vivo and ex vivo therapeutic strategies.
  • Discussion of clinical trial outcomes and translational challenges.

Main Results:

  • In vivo CRISPR therapies are practical for eye and liver diseases due to targeted delivery.
  • Ex vivo CRISPR therapy allows for pre-infusion edit characterization and limits off-target effects.
  • Successful applications in treating blood disorders and ongoing trials for vision restoration.

Conclusions:

  • CRISPR therapies hold significant potential for treating a range of genetic disorders.
  • Clinical translation requires addressing challenges in delivery, safety, cost, and scalability.
  • Both in vivo and ex vivo strategies present distinct advantages and hurdles for widespread adoption.