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Related Concept Videos

Cystic Fibrosis: Management01:24

Cystic Fibrosis: Management

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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic...
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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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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.
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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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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 targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Related Experiment Video

Updated: Sep 7, 2025

Purification of the Cystic Fibrosis Transmembrane Conductance Regulator Protein Expressed in Saccharomyces cerevisiae
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Purification of the Cystic Fibrosis Transmembrane Conductance Regulator Protein Expressed in Saccharomyces cerevisiae

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CFTR RNA- and DNA-based therapies.

Patrick T Harrison1

  • 1Department of Physiology, BioSciences Institute, University College Cork, Ireland.

Current Opinion in Pharmacology
|June 16, 2022
PubMed
Summary

This review explores RNA- and DNA-based therapies for cystic fibrosis (CF) lung disease, highlighting the need for effective delivery systems to treat all CF patients and affected organs.

Area of Science:

  • Molecular Biology
  • Genetic Medicine
  • Pulmonology

Background:

  • Cystic Fibrosis (CF) lung disease is a progressive condition requiring novel therapeutic strategies.
  • Current treatments manage symptoms, but disease-modifying therapies are crucial for long-term outcomes.
  • Nucleic acid-based therapies offer potential for addressing the genetic basis of CF.

Purpose of the Study:

  • To review recent advancements in RNA- and DNA-based methodologies for treating cystic fibrosis.
  • To discuss intracellular targets relevant to CF lung disease.
  • To explore the critical role of delivery systems in achieving clinical success for nucleic acid therapies.

Main Methods:

  • Review of current literature on RNA- and DNA-based therapeutic strategies for CF.

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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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  • Analysis of intracellular targets and delivery systems for nucleic acid therapies.
  • Discussion of the potential for tailored approaches based on CF mutations.

    • Main Results:

    • Significant progress in developing RNA- and DNA-based approaches for CF.
    • Identification of key intracellular targets amenable to nucleic acid-based interventions.
    • Emphasis on the necessity of efficient and safe delivery systems for therapeutic efficacy.

    Conclusions:

    • Nucleic acid-based therapies hold promise for treating cystic fibrosis lung disease.
    • Delivery system innovation is paramount for translating these strategies into clinical practice.
    • Future efforts may involve personalized approaches and expansion to treat all CF-affected organs.