Biology – Genetic technology applied to medicine | e-Consult
Genetic technology applied to medicine (1 questions)
Several viral vectors are used in gene therapy, each with distinct characteristics:
Vector Type | Advantages | Disadvantages | SCID Treatment? | Inherited Eye Disease? |
Retroviruses (e.g., Murine Retrovirus) | Efficient integration into host genome, long-term expression. | Insertional mutagenesis (disrupts genes), limited packaging capacity. | Commonly used. | Commonly used. |
Adenoviruses | High transduction efficiency, can infect a wide range of cell types. | Transient expression (doesn't integrate into genome), can elicit strong immune response. | Less common. | Less common. |
Adeno-associated viruses (AAVs) | Low immunogenicity, long-term expression, can infect a variety of cell types. | Limited packaging capacity, potential for pre-existing immunity. | Increasingly popular. | Increasingly popular. |
Retroviruses are often used for SCID due to their ability to integrate into HSCs, providing long-term correction. However, the risk of insertional mutagenesis is a concern. AAVs are favored for inherited eye diseases because of their lower immunogenicity and ability to target retinal cells directly. The limited packaging capacity of AAVs can be a constraint, but newer, larger AAV serotypes are being developed.