Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

CRISPR01:59

CRISPR

52.6K
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...
52.6K
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

127
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...
127
CRISPR and crRNAs02:53

CRISPR and crRNAs

17.2K
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...
17.2K
Homologous Recombination02:31

Homologous Recombination

50.8K
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...
50.8K
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

6.1K
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...
6.1K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Propensity-matched Comparison of VATS Left Upper Trisegmentectomy and Lobectomy.

The Annals of thoracic surgery·2021
Same author

Interrogation of the dynamic properties of higher-order heterochromatin using CRISPR-dCas9.

Molecular cell·2021
Same author

MicroRNA-20a Targeting LASS2 Promotes the Proliferation, Invasiveness and Migration of Bladder Cancer.

Clinical laboratory·2021
Same author

Quantitative MR relaxation using MR fingerprinting with fractional-order signal evolution.

Journal of magnetic resonance (San Diego, Calif. : 1997)·2021
Same author

Application of a decision tree model in the early identification of severe patients with severe fever with thrombocytopenia syndrome.

PloS one·2021
Same author

Haplotype-resolved genome assembly provides insights into evolutionary history of the tea plant Camellia sinensis.

Nature genetics·2021
Same journal

Correction: Yalçın et al. Impact of SGLT2 Inhibitors on Cardiovascular Risk Scores, Metabolic Parameters, and Laboratory Profiles in Type 2 Diabetes. <i>Life</i> 2025, <i>15</i>, 722.

Life (Basel, Switzerland)·2026
Same journal

Correction: Schubert et al. Minimally Invasive Ablation Strategies for Renal Cell Carcinoma Patients Ineligible for Surgery. <i>Life</i> 2026, <i>16</i>, 73.

Life (Basel, Switzerland)·2026
Same journal

Blood Group Antigen Combinations and COVID-19: Complexity, Associations and Possible Clinical Relevance.

Life (Basel, Switzerland)·2026
Same journal

Beyond HPV in Eastern Europe: Genotype Distribution, Molecular Biomarkers, Vaginal Microbiome, and Implications for Cervical Cancer Prevention.

Life (Basel, Switzerland)·2026
Same journal

Therapeutic Effects of <i>Scutellaria baicalensis</i> Georgi Extract and Baicalein on Olfactory Dysfunction and Neurobehavioral Alterations in a Methimazole-Induced Injury Model.

Life (Basel, Switzerland)·2026
Same journal

The Effects of Unstable Strength Training on Lower Limb Stability in Adolescent Volleyball Players in China.

Life (Basel, Switzerland)·2026
See all related articles

Related Experiment Video

Updated: Aug 16, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
08:20

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

Published on: September 2, 2021

4.2K

CRISPR-Based Tools for Fighting Rare Diseases.

Qingyang Li1, Yanmin Gao1, Haifeng Wang1,2

  • 1Tsinghua-Peking Center for Life Sciences, School of Life Sciences, Tsinghua University, Beijing 100084, China.

Life (Basel, Switzerland)
|December 23, 2022
PubMed
Summary
This summary is machine-generated.

CRISPR-Cas gene editing offers new hope for rare disease treatments. This review explores CRISPR tools and delivery methods, highlighting their potential to combat rare genetic disorders.

Keywords:
CRISPR-Casdelivery systemgene therapyrare disease

More Related Videos

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
09:00

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

8.2K
Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
09:04

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

8.3K

Related Experiment Videos

Last Updated: Aug 16, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
08:20

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

Published on: September 2, 2021

4.2K
Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
09:00

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

8.2K
Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
09:04

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

8.3K

Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Molecular Biology

Background:

  • Rare diseases impact a significant global population.
  • The CRISPR-Cas system is a revolutionary genome engineering technology.
  • CRISPR facilitates understanding rare disease mechanisms and developing therapies.

Purpose of the Study:

  • To review current CRISPR-based tools for rare disease therapies.
  • To compare the advantages and disadvantages of various CRISPR tools and delivery systems.
  • To discuss the therapeutic applications of CRISPR technology in rare diseases.

Main Methods:

  • Literature review of CRISPR-Cas system applications in rare diseases.
  • Comparative analysis of different CRISPR tools (e.g., Cas9, Cas12).
  • Evaluation of various delivery methods (e.g., viral, non-viral).

Main Results:

  • CRISPR-Cas technology shows significant promise for rare disease treatment.
  • Different CRISPR tools offer unique advantages for specific genetic defects.
  • Delivery method choice is critical for therapeutic efficacy and safety.

Conclusions:

  • CRISPR-based tools represent a powerful and evolving therapeutic strategy for rare diseases.
  • Ongoing research aims to optimize CRISPR systems and delivery for clinical translation.
  • The potential of CRISPR to address unmet needs in rare disease therapy is substantial.