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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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CRISPR01:59

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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 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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Group Therapy01:26

Group Therapy

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Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
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Gene Flow02:39

Gene Flow

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Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
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Gene Families01:57

Gene Families

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Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
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Related Experiment Video

Updated: Jan 27, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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Toward In Vivo Gene Therapy Using CRISPR.

Kristian Alsbjerg Skipper1, Jacob Giehm Mikkelsen2

  • 1Department of Biomedicine, Aarhus University, Aarhus, Denmark.

Methods in Molecular Biology (Clifton, N.J.)
|March 27, 2019
PubMed
Summary

In vivo CRISPR gene editing shows promise for treating genetic diseases directly in patients. This review explores current methods and challenges for effective and safe in vivo genome editing in mice.

Keywords:
AAVAlpha-1 antitrypsin deficiencyCas9 immune responsesEyeGenome editingHypercholesterolemiaKnockoutMuscular dystrophy

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • CRISPR technology enables precise genome editing, advancing towards clinical applications for genetic disorders.
  • While ex vivo gene editing is progressing, in vivo CRISPR applications directly in patients present significant challenges.
  • Existing in vivo CRISPR strategies often utilize viral vectors for delivering Cas9 endonuclease and single guide RNA.

Purpose of the Study:

  • To review the current state of in vivo CRISPR genome editing.
  • To highlight proof-of-concept studies demonstrating efficacious in vivo editing.
  • To discuss the potential need for transient CRISPR component expression and immune evasion strategies.

Main Methods:

  • Review of prominent studies involving CRISPR-Cas9 gene targeting in animal models.
  • Focus on delivery methods using classical vector systems.
  • Examination of gene editing in mouse liver, muscles, and eyes.

Main Results:

  • Accumulating evidence supports the efficacy of in vivo CRISPR editing.
  • Viral vectors are commonly employed for delivering CRISPR components.
  • The review highlights successful gene targeting in various mouse tissues.

Conclusions:

  • In vivo CRISPR gene editing is a rapidly advancing field with therapeutic potential.
  • Further research is needed to optimize delivery, ensure safety, and manage immune responses.
  • Transient expression of CRISPR components may be sufficient and beneficial for safety.