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Related Experiment Videos

Evolving Gene Therapy in Primary Immunodeficiency.

Adrian J Thrasher1, David A Williams2

  • 1Great Ormond Street Hospital for Children NHS Foundation Trust, London WC1N 3JH, UK; University College London Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|April 4, 2017
PubMed
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Severe combined immunodeficiency (SCID) was fatal before 1968. Advances in newborn screening and gene therapy now offer cures for most SCID patients.

Area of Science:

  • Immunology
  • Genetics
  • Pediatrics

Background:

  • Severe combined immunodeficiency (SCID) historically led to fatal infections in infants.
  • The first successful bone marrow transplant occurred in 1968, offering a potential treatment.
  • Primary immunodeficiencies (PIDs) represent a group of rare genetic disorders affecting the immune system.

Observation:

  • Newborn screening for SCID has become more widely available.
  • Allogeneic transplantation procedures have significantly improved.
  • Hematopoietic stem and progenitor cell (HSC/P) gene therapy has emerged as a successful treatment.

Findings:

  • The majority of children with SCID can now be identified and cured.
  • Clinical gene therapy for SCID has progressed significantly since its inception.
Keywords:
gene therapyimmunodeficienciesretrovirus

Related Experiment Videos

  • New targets and technologies are being developed for PID treatment.

    • Implications:

    • Gene therapy offers a promising curative approach for SCID and other PIDs.
    • Early identification through newborn screening improves treatment outcomes.
    • Continued research into gene therapy holds potential for treating a wider range of genetic disorders.