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Gene therapy is a revolutionary medical approach aimed at treating genetic disorders by modifying the patient's genetic material. It involves techniques like gene replacement, silencing, and activation, using vectors such as CRISPR/Cas9. The therapy has shown promise in treating conditions like cystic fibrosis, hemophilia, and certain cancers, with notable examples including Luxturna and Zolgensma. Despite its potential, gene therapy faces challenges like high costs and safety concerns.
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Gene therapy is an advanced medical technique that alters genetic material to treat genetic disorders
Germline Gene Therapy
Germline gene therapy involves changes to the DNA in germ cells that could be inherited by future generations
Somatic Gene Therapy
Somatic gene therapy targets non-reproductive cells and does not affect the patient's offspring
Gene Replacement
Gene replacement involves inserting new genes to treat genetic disorders
Gene Silencing
Gene silencing aims to deactivate harmful genes
Gene Activation
Gene activation involves activating genes to treat genetic disorders
Viral vectors play a crucial role in gene therapy as carriers that transport therapeutic genes into the cells of patients
Adenoviruses
Adenoviruses can infect a broad range of cells but may elicit immune responses
Retroviruses
Retroviruses integrate their DNA into the host genome
Adeno-associated viruses (AAVs)
AAVs have the ability to infect both dividing and non-dividing cells and sustain long-term gene expression
Lentiviruses
Lentiviruses can infect both dividing and non-dividing cells and sustain long-term gene expression
Gene therapy can treat a variety of diseases, including inherited disorders, certain cancers, and viral infections
Luxturna
Luxturna treats vision loss by supplying a functional gene to address a specific genetic condition
Strimvelis
Strimvelis corrects immune cells to treat a specific genetic condition
Zolgensma
Zolgensma provides a functional gene to treat a specific genetic condition in young children
Gene therapy offers the potential to provide durable and possibly curative outcomes for genetic disorders
Gene therapy presents challenges such as substantial costs, technical complexities, and potential adverse effects
The ongoing development and refinement of gene therapy techniques are crucial for its progress and success in treating genetic disorders
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Gene therapy techniques
Involves gene insertion, gene repair, or gene deactivation.
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Gene therapy targets
Aims at monogenic diseases like cystic fibrosis, hemophilia, and complex conditions like cardiovascular diseases.
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Gene therapy outcomes
Potential for durable, curative results for historically difficult-to-treat diseases.
