Adenoviral Vectors for Gene Therapy of Hereditary Diseases
Author Information
Author(s): Muravyeva Anna, Smirnikhina Svetlana, Sanchez-Martin Manuel
Primary Institution: Laboratory of Genome Editing, Research Centre for Medical Genetics, Moscow, Russia
Conclusion
Adenoviral vectors show promise for gene therapy but face significant challenges, particularly in treating hereditary diseases due to high immunogenicity and transient gene expression.
Supporting Evidence
- Adenoviral vectors have high transduction efficiency but are limited by strong immune responses.
- Clinical trials for cystic fibrosis and ornithine transcarbamylase deficiency using adenoviral vectors showed low transduction rates.
- Modifications to adenoviral vectors aim to improve targeting and reduce immune responses.
Takeaway
Adenoviral vectors are like delivery trucks for genes, but they can cause a lot of traffic jams in the body, making it hard for them to deliver their packages effectively.
Methodology
This review analyzes clinical trials and research on adenoviral vectors for gene therapy, focusing on their advantages, limitations, and modifications to improve efficacy.
Limitations
The high immunogenicity of adenoviral vectors limits their effectiveness and safety for repeated use in gene therapy.
Digital Object Identifier (DOI)
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