Table-1.
Characteristics, advantages and limitations of vectors used in corneal gene therapy
Adenovirus | Retrovirus | Lentivirus | AAV | Non-viral | Nanoparticles | |
---|---|---|---|---|---|---|
Family | Adenoviridae | Retrovirdae | Retrovirdae | Parvoviridae | --- | --- |
Virion size | 70-90 nm | 80-130 nm | 80-130 nm | 18-26 nm | --- | --- |
Genome size | 38-39 kb | 3-9 kb | 3-9 kb | 4.7 kb | --- | --- |
Genome type | dsDNA | ssRNA | ssRNA | ssDNA | RNA or DNA | RNA or DNA |
Virion size | 70-90 nm | 80-130 nm | 80-130 nm | 18-26 nm | --- | --- |
Host genome integration | No | Yes | No, Yes (if impaired) | No | No | No |
Efficiency | Low-high | Moderate-High | High | Moderate-High | Poor | Poor-moderate |
Transgene expression | Transient (days-weeks) | Long-term (months-years) | Long-term (months-years) | Potentially long-term (months-years) | Transient (hours-days) | Variable (hours-months) |
Immunogenicity | High | Moderate-high | Moderate-high | Low-moderate | Low | Low-high |
Infection/tropism | Dividing cells | Dividing cells | Dividing and Non-dividing | Dividing and Non-dividing | Dividing and Non-dividing | Dividing and Non-dividing |
Packaging capacity | ≤ 7.5 kb | ≤ 8 kb | ≤ 8 kb | ≤ 1.8 kb | No limit | No limit |
Advantages | High titer, infect nearly all cell types, non-mutagenic | Highly efficient, sustained long gene expression | Highly efficient for all cell types sustained gene expression | Highly efficient for all cell types, stable transduction, site-specific integration | Safe, low immunogenicity, deliver any size gene | Safe, variable immunogenicity deliver any size gene |
Limitations | Immunogenic, common human virus that reduce potency | Requires dividing cells, low titer, oncogenic, random integration | Immunogenic, HIV origin, random integration potential | Small insert size, low immunogenicity, HSV or adenovirus contamination potential | Poor efficiency, low-high immune reaction, short-term transgene expression | Poor efficiency, low-high immune reaction, short-term transgene expression |