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Related Concept Videos

Cystic Fibrosis: Pathogenesis01:23

Cystic Fibrosis: Pathogenesis

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Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
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DNA replication is a well-evolved process that copies millions of base pairs with high fidelity during each cell division. Occasionally a wrong base or a long stretch of wrong bases may get added to the daughter strands. If the errors are left unchecked, cells might accumulate several mutations that might endanger their  survival. Therefore, the copying errors are checked and repaired at three levels.
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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

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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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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Genome Editing for Cystic Fibrosis.

Guoshun Wang1

  • 1Department of Microbiology, Immunology and Parasitology, Louisiana State University Health Sciences Center, CSRB 607, 533 Bolivar Street, New Orleans, LA 70112, USA.

Cells
|June 28, 2023
PubMed
Summary
This summary is machine-generated.

Cystic fibrosis (CF) gene therapy using CRISPR/Cas editing offers a potential permanent cure for all patients. This approach aims to repair genetic defects, addressing limitations of current CFTR modulator therapies.

Keywords:
CFTRCRISPR/Cascystic fibrosisgene editinggenome editing

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Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • Cystic fibrosis (CF) is a genetic disorder caused by CFTR gene mutations.
  • Current CFTR modulators treat ~90% of patients but are not curative.
  • A durable, curative therapy for all CF patients is still needed.

Purpose of the Study:

  • To review the progress of gene editing technologies for CF therapy.
  • To discuss the potential of CRISPR/Cas gene editing for CF.

Main Methods:

  • Review of recent advancements in CRISPR/Cas gene editing technology.
  • Analysis of the application of gene editing for CFTR gene repair.

Main Results:

  • CRISPR/Cas gene editing presents a promising platform for genetic therapy.
  • This technology offers a potential route for in-situ repair of CF-causing mutations.

Conclusions:

  • Gene editing technologies are rapidly evolving for CF treatment.
  • CRISPR/Cas gene editing holds potential for a definitive cure for all CF patients.