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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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In most mammalian species, females have two X sex chromosomes and males have an X and Y. As a result, mutations on the X chromosome in females may be masked by the presence of a normal allele on the second X. In contrast, a mutation on the X chromosome in males more often causes observable biological defects, as there is no normal X to compensate. Trait variations arising from mutations on the X chromosome are called “X-linked”.
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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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The central dogma explains the flow of genetic information from DNA nucleotides to the amino acid sequence of proteins.
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[Affordable gene therapies?]

Bertrand Jordan1

  • 1Biologiste, généticien et immunologiste, Président d'Aprogène (Association pour la promotion de la Génomique), 13007 Marseille, France.

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|March 3, 2025
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Summary
This summary is machine-generated.

The high cost of gene therapies limits patient access. A study suggests manufacturing improvements could reduce gene therapy costs tenfold, making these life-saving treatments more affordable.

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Area of Science:

  • Biotechnology
  • Genetics
  • Health Economics

Background:

  • The U.S. Food and Drug Administration (FDA) has approved twenty gene therapies.
  • Current gene therapies exceed one million USD per patient, posing significant affordability challenges.

Purpose of the Study:

  • To investigate the cost drivers in gene therapy development and manufacturing.
  • To identify potential strategies for reducing the cost of gene therapies.

Main Methods:

  • Systematic review of gene therapy development and manufacturing processes.
  • Analysis of cost-reduction opportunities within the supply chain.

Main Results:

  • The study indicates a potential tenfold cost reduction for gene therapies.
  • Cost decreases are achievable without substantial legislative reform.
  • The Innovative Genomics Institute contributed to this analysis.

Conclusions:

  • Significant cost reductions in gene therapy manufacturing are feasible.
  • These advancements offer a pathway toward more accessible and affordable genetic treatments.