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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
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Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Updated: May 12, 2026

Conditional Reprogramming of Pediatric Human Esophageal Epithelial Cells for Use in Tissue Engineering and Disease Investigation
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Gene therapy for PIDs: progress, pitfalls and prospects.

Sayandip Mukherjee1, Adrian J Thrasher

  • 1Centre for Immunodeficiency, Molecular Immunology Unit, University College London Institute of Child Health, 30 Guilford Street, London WC1N1EH, UK.

Gene
|April 10, 2013
PubMed
Summary

Gene therapy offers promising treatments for primary immunodeficiency disorders (PIDs). Research focuses on improving safety and efficacy by addressing viral vector integration and transgene expression for better outcomes.

Keywords:
ADA-SCIDCGDDSBEF-1αERTGALVGSHGene therapyGvHDHLAHRHSCTHSCsIFNILLCRLTRLVsMDSMLVMNNADPHNK cellsPEGPGKPIDPIDsROSRVsSAESCIDSCID-X1SFFVSINT cell-acute lymphoblastic leukaemiaT-ALLTALENUCOEVSV-GWASWASpWiskott–Aldrich syndromeWiskott–Aldrich syndrome proteinX-linked neutropeniaX-linked severe combined immunodeficiencyX-linked thrombocytopeniaXLNXLTZFNadenosine deaminase deficiency-severe combined immunodeficiencychronic granulomatous disorderdouble strand breakelongation factor 1αenzyme replacement therapygenomic safe harbourgibbon ape leukaemia virusgraft versus host diseasehaematopoietic stem cell transplanthaematopoietic stem cellshomologous recombinationhuman leukocyte antigeninterferoninterleukinlentiviral vectorslocus control regionlong terminal repeatmeganucleasesmiRmicroRNAmurine leukaemia virusmyelodysplastic syndromenatural killer cellsnicotinamide adenine dinucleotide phosphate hydrogenphosphoglycerokinasepolyethylene glycolprimary immunodeficiency disordersreactive oxygen speciesretroviral vectorsself-inactivatingserious adverse eventspleen focus-forming virustranscription activator-like effector nucleasesubiquitous chromatin opening elementvesicular stomatitis virus-G proteinzinc finger nuclease

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Area of Science:

  • Immunology
  • Molecular Biology
  • Biotechnology

Background:

  • Gene therapy has advanced significantly for primary immunodeficiency disorders (PIDs) over the last decade.
  • Current methods involve ex-vivo gene transfer into autologous hematopoietic stem cells (HSCs) using viral vectors.

Purpose of the Study:

  • To review progress in gene therapy trials for ADA-SCID, SCID-X1, CGD, and WAS.
  • To discuss challenges such as insertional mutagenesis and transgene expression.
  • To outline advancements enhancing the safety and efficacy of gene therapy for PIDs.

Main Methods:

  • Review of clinical trial data and pre-clinical investigations.
  • Analysis of viral vector integration tendencies.
  • Evaluation of transgene expression, stability, and efficacy.

Main Results:

  • Gene therapy has shown encouraging outcomes for several PIDs.
  • Malignant events due to insertional mutagenesis are a significant concern.
  • Improvements in vector design and gene expression are crucial.

Conclusions:

  • Gene therapy for PIDs has progressed but requires careful risk-benefit assessment.
  • Ongoing research aims to mitigate adverse events and optimize therapeutic strategies.
  • Future advancements are expected to improve the safety and efficacy profile of PID gene therapies.