Statements (51)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:virus
|
| gptkbp:administeredBy |
intramuscular injection
intravenous injection intrathecal injection intraperitoneal injection |
| gptkbp:advantage |
low immunogenicity
high transduction efficiency long-term gene expression pre-existing immunity in some populations |
| gptkbp:derivedFrom |
adeno-associated virus serotype 8
|
| gptkbp:genomeType |
single-stranded DNA
|
| https://www.w3.org/2000/01/rdf-schema#label |
AAV8 vector
|
| gptkbp:limitation |
episomal persistence
limited packaging size potential for immune response |
| gptkbp:packagingCapacity |
~4.7 kb
|
| gptkbp:producedBy |
HEK293 cells
triple transfection method |
| gptkbp:purity |
affinity chromatography
iodixanol gradient ultracentrifugation |
| gptkbp:relatedTo |
AAV2 vector
AAV9 vector |
| gptkbp:safetyProfile |
generally safe
|
| gptkbp:tropism |
gptkb:skeletal_muscle
heart central nervous system liver |
| gptkbp:usedFor |
protein expression
CRISPR/Cas9 delivery RNAi delivery delivery of therapeutic genes reporter gene expression |
| gptkbp:usedIn |
clinical trials
preclinical research gene therapy animal models spinal muscular atrophy gene therapy CNS gene therapy Duchenne muscular dystrophy gene therapy cardiac gene therapy hemophilia gene therapy human gene therapy trials in vivo gene delivery liver-targeted gene therapy metabolic disorder gene therapy muscle-targeted gene therapy preclinical studies in mice preclinical studies in non-human primates retinal gene therapy |
| gptkbp:bfsParent |
gptkb:DTX402
|
| gptkbp:bfsLayer |
7
|