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 |