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Related Concept Videos

Immunodeficiency Diseases01:25

Immunodeficiency Diseases

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Immunodeficiency disorders are conditions in which the immune system's ability to fight infectious disease and cancer is compromised or entirely absent. The immune system comprises a complex network of cells, tissues, and organs that work together to protect the body from potentially harmful invaders. When this system is deficient or not functioning properly, it leaves the body susceptible to infections, diseases, or other complications.
There are three main causes of immunodeficiency...
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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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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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Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects.

Rohan Ameratunga1, Euphemia Leung2, See-Tarn Woon3

  • 1Department of Clinical Immunology, Auckland Hospital, Park Rd, Grafton 1010, Auckland, New Zealand; Department of Virology and Immunology, Auckland Hospital, Park Rd, Grafton 1010, Auckland, New Zealand; Department of Molecular Medicine and Pathology, School of Medicine, Faculty of Medical and Health Sciences, University of Auckland, New Zealand.

Clinical Immunology (Orlando, Fla.)
|November 28, 2023
PubMed
Summary

CRISPR gene editing offers potential for Primary Immunodeficiencies (PIDs). While Common Variable Immunodeficiency Disorders (CVID) lack defined genetic causes, CRISPR may help investigate CVID-like disorders and, long-term, treat them.

Keywords:
CRISPR Cas9CVIDCVID-like disordersGene editingGenotoxicity

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Area of Science:

  • Genetics
  • Immunology
  • Molecular Biology

Background:

  • Primary Immunodeficiencies (PIDs) are severe genetic disorders.
  • Common Variable Immunodeficiency Disorders (CVID) are the most frequent symptomatic PID in adults and children.
  • Unlike many PIDs, CVID lacks a definable genetic defect, classifying patients with a CVID phenotype and mutation as having a CVID-like disorder consequent to an inborn error of immunity (IEI).

Purpose of the Study:

  • To explore the clinical utility, limitations, and risks of gene editing technologies, including CRISPR-Cas9, for Primary Immunodeficiencies.
  • To discuss the potential of gene editing in investigating the epigenetic and polygenic mechanisms underlying CVID and CVID-like disorders.
  • To evaluate the long-term prospects of CRISPR-Cas9 and gene-based therapies for treating CVID-like disorders with known underlying IEI.

Main Methods:

  • Review of current literature on CRISPR-Cas9 gene editing and its applications in genetic disorders.
  • Analysis of the genetic basis and phenotypic variability of CVID and CVID-like disorders.
  • Exploration of the potential benefits and risks, including genotoxicity, of gene editing technologies.

Main Results:

  • CRISPR-Cas9 presents opportunities for correcting severe genetic defects.
  • CVID-like disorders exhibit varied inheritance patterns (autosomal recessive and dominant) with differing penetrance.
  • Immediate applications focus on in vitro investigation of epigenetic and polygenic factors in CVID.

Conclusions:

  • Gene editing technologies like CRISPR-Cas9 hold promise for understanding and potentially treating CVID-like disorders.
  • Current limitations include collateral genotoxicity and the need to identify underlying genetic causes for effective therapy.
  • Future therapeutic applications are anticipated for CVID-like disorders where the specific inborn error of immunity is identified.