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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.
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Medical management of tuberculosis (TB) patients involves a comprehensive approach that includes diagnosis, treatment, and monitoring. The specific strategies can vary depending on the type of tuberculosis (latent or active), the patient's overall health status, and other considerations.
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Crohn's disease is an inflammatory bowel disorder marked by chronic inflammation of the GI tract. Various treatment strategies for Crohn's disease are employed, such as immunomodulatory agents, glucocorticoids, and biologics or anti-TNF therapy. Azathioprine (Imuran), a commonly used immunomodulatory drug for Crohn's disease, is converted in the body to mercaptopurine, which inhibits purine biosynthesis and cell proliferation. Both are utilized in severe cases of Inflammatory Bowel...
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Tuberculosis, or TB, is a bacterial infectious disease caused by Mycobacterium tuberculosis. While its primary impact is on the lungs, leading to pulmonary tuberculosis, it can also affect various other organs, a condition referred to as extrapulmonary tuberculosis.
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Updated: Dec 15, 2025

Passive Administration of Monoclonal Antibodies Against H. capsulatum and Others Fungal Pathogens
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Progress in treating chronic granulomatous disease.

Andrew R Gennery1,2

  • 1Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

British Journal of Haematology
|July 10, 2020
PubMed
Summary
This summary is machine-generated.

Chronic granulomatous disease (CGD) is an immunodeficiency treated by stem cell transplant or gene therapy. Lentiviral vector gene therapy shows promising results for treating CGD.

Keywords:
chronic granulomatous diseasegene therapyhaematopoietic stem cell transplantationinfectioninflammation

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Area of Science:

  • Immunology
  • Genetics
  • Hematology

Background:

  • Chronic granulomatous disease (CGD) is a primary immunodeficiency.
  • It results from defects in the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase complex subunits.
  • Infections and inflammatory complications are common in CGD patients.

Purpose of the Study:

  • To review current treatment options for CGD.
  • To highlight the potential of gene therapy as an alternative curative approach.

Main Methods:

  • Review of existing literature on CGD treatments.
  • Analysis of outcomes from hematopoietic stem cell transplantation (HSCT).
  • Evaluation of gene therapy approaches, including gammaretroviral and lentiviral vectors.

Main Results:

  • Hematopoietic stem cell transplantation (HSCT) offers a cure for CGD with approximately 85% survival rate.
  • Gammaretroviral gene therapy yielded disappointing results due to genotoxicity and loss of efficacy.
  • Preliminary data suggest lentiviral vector-based gene therapy is highly encouraging for CGD treatment.

Conclusions:

  • HSCT is an established curative therapy for CGD.
  • Lentiviral gene therapy represents a promising emerging alternative for CGD treatment, with potential for improved safety and efficacy.