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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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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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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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Gene Conversion02:08

Gene Conversion

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Other than maintaining genome stability via DNA repair, homologous recombination plays an important role in diversifying the genome. In fact, the recombination of sequences forms the molecular basis of genomic evolution. Random and non-random permutations of genomic sequences create a library of new amalgamated sequences. These newly formed genomes can determine the fitness and survival of cells. In bacteria, homologous and non-homologous types of recombination lead to the evolution of new...
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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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What is Gene Expression?01:42

What is Gene Expression?

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Overview
Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
Genetic Information Flows from DNA to RNA to Protein
A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is made up of nucleotides and proteins consist of amino...
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Related Experiment Video

Updated: Feb 5, 2026

Production and Purification of Baculovirus for Gene Therapy Application
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Gene Therapy for Hemoglobinopathies.

Marina Cavazzana1, Fulvio Mavilio1,2

  • 11 University of Paris Descartes-Sorbonne Paris Cité , IMAGINE Institute, Paris, France; and University of Modena and Reggio Emilia , Modena, Italy .

Human Gene Therapy
|September 12, 2018
PubMed
Summary

Gene therapy offers hope for blood disorders like beta-thalassemia and sickle-cell disease by correcting stem cells. Current methods face challenges, but gene editing may provide a more accessible future treatment.

Keywords:
gene editingglobin geneslentiviral vectorssickle-cell diseasethalassemia

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Area of Science:

  • Hematology
  • Gene Therapy
  • Molecular Biology

Background:

  • Gene therapy using lentiviral vectors shows promise for treating beta-thalassemia and sickle-cell disease by correcting hematopoietic stem cells.
  • Current approaches involve transplanting autologous stem cells modified with a functional beta-globin gene.

Purpose of the Study:

  • To review the current state of gene therapy for hemoglobinopathies.
  • To identify limitations of existing gene therapy strategies.
  • To explore gene editing as a potential alternative.

Main Methods:

  • Review of preclinical and clinical studies on lentiviral vector-based gene therapy.
  • Analysis of factors limiting current gene therapy efficacy.
  • Discussion of gene editing technologies for hemoglobinopathies.

Main Results:

  • Gene therapy has demonstrated safety and efficacy in clinical studies.
  • Limitations include stem cell dose/quality, gene transfer efficiency, and myeloablative regimen toxicity.
  • High cost and complexity restrict accessibility, particularly in low-income countries.

Conclusions:

  • Lentiviral vector-based gene therapy is a viable approach for beta-thalassemia and sickle-cell disease.
  • Overcoming current limitations is crucial for broader application.
  • Gene editing presents a promising alternative that requires further clinical validation.