Skip to main content

Advertisement

Table 1 The properties required for development of an ideal vector system and those that need to be avoided.

From: LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives

Properties needed Properties to be avoided
Stable high titre vectors Vector degradation
Simple and reproducible production Replication competent virus
Unlimited packaging capacity Expression of undesirable viral proteins
Efficient gene transfer to the target cells Germline gene transmission
Controlled genomic integration Insertional mutagenesis
Regulated normal levels of expression Inappropriate toxic expression
Ability to repeat delivery if needed Severe immune response against vector system
Long-term expression Immune response against transgene products