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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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Immunodeficiency Diseases01:25

Immunodeficiency Diseases

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Immunodeficiency disorders are conditions in which the immune system's ability to fight infectious disease and cancer is compromised or entirely absent. The immune system comprises a complex network of cells, tissues, and organs that work together to protect the body from potentially harmful invaders. When this system is deficient or not functioning properly, it leaves the body susceptible to infections, diseases, or other complications.
There are three main causes of immunodeficiency...
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Gene Families01:57

Gene Families

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Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
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Group Therapy01:26

Group Therapy

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Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
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Genome Size and the Evolution of New Genes03:21

Genome Size and the Evolution of New Genes

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While every living organism has a genome of some kind (be it RNA, or DNA), there is considerable variation in the sizes of these blueprints. One major factor that impacts genome size is whether the organism is prokaryotic or eukaryotic. In prokaryotes, the genome contains little to no non-coding sequence, such that genes are tightly clustered in groups or operons sequentially along the chromosome. Conversely, the genes in eukaryotes are punctuated by long stretches of non-coding sequence.
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Gene Flow02:39

Gene Flow

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Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
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Related Experiment Video

Updated: Jan 22, 2026

Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy
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Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy

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Gene therapy for primary immunodeficiency.

Claire Booth1, Rosa Romano2, Maria Grazia Roncarolo2,3

  • 1Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, UK.

Human Molecular Genetics
|July 13, 2019
PubMed
Summary

Gene therapy shows promise for treating genetic disorders like primary immunodeficiencies. Advances in gene editing and lentiviral vectors are paving the way for new, effective treatments.

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Area of Science:

  • * Molecular Biology and Genetics
  • * Immunology
  • * Hematology

Background:

  • * Gene therapy has successfully treated primary immunodeficiencies (PIDs) and metabolic disorders over the past two decades.
  • * Early gene therapy utilized gammaretroviral vectors, with advancements leading to safer and more efficient lentiviral platforms for hematopoietic stem cell gene transfer.

Purpose of the Study:

  • * To review recent developments in gene therapy, including licensed treatments and novel strategies.
  • * To discuss the potential of gene editing technologies for targeted genetic correction.
  • * To highlight the evolving landscape of gene therapy for genetic diseases.

Main Methods:

  • * Review of recent clinical trials and licensed gene therapies.
  • * Analysis of advancements in vector technology (lentiviral platforms).
  • * Evaluation of gene editing platforms for targeted gene correction.

Main Results:

  • * Successful application of gene therapy for severe combined immunodeficiency, Wiskott-Aldrich syndrome, and leukodystrophy.
  • * Development of gene-corrected autologous T cells as a therapeutic strategy for PIDs.
  • * Promising results from recent clinical trials indicating increased efficacy and safety.

Conclusions:

  • * Gene therapy is rapidly advancing as a viable treatment for numerous genetic conditions.
  • * Autologous gene therapies are expected to become standard care for many severe diseases within the next decade.
  • * Continued innovation in gene editing and vector technology will further expand therapeutic applications.