Adeno-associated virus (AAV) vector
GPTKB entity
Statements (52)
Predicate | Object |
---|---|
gptkbp:instanceOf |
viral vector
|
gptkbp:built |
gptkb:Adeno-associated_virus
|
gptkbp:canBe |
therapeutic genes
|
gptkbp:characterDevelopment |
multiple serotypes
|
gptkbp:contains |
single-stranded DNA
|
gptkbp:electronicSystems |
non-dividing cells
|
gptkbp:hasProductLine |
recombinant DNA technology
|
gptkbp:hasSibling |
20-25 nm
|
https://www.w3.org/2000/01/rdf-schema#label |
Adeno-associated virus (AAV) vector
|
gptkbp:isAttendedBy |
injection
capsid proteins |
gptkbp:isCharacterizedBy |
high transduction efficiency
low immunogenicity long-term expression stable integration tissue tropism |
gptkbp:isCompatibleWith |
various delivery methods
|
gptkbp:isConsidered |
a promising tool
safe for human use |
gptkbp:isEvaluatedBy |
safety and efficacy
|
gptkbp:isInspiredBy |
heat
UV light detergents |
gptkbp:isNotableFor |
pathogenicity
|
gptkbp:isStudiedIn |
cancer therapy
muscular dystrophy hemophilia neurological disorders cystic fibrosis retinal diseases |
gptkbp:isUsedBy |
genetic disorders
|
gptkbp:isUsedFor |
CRISPR components
|
gptkbp:isUsedIn |
clinical trials
immunotherapy preclinical studies vaccine development gene editing cardiovascular research cell therapy treatment of autoimmune diseases treatment of infectious diseases neurodegenerative disease research treatment of rare diseases treatment of inherited disorders eye gene therapy liver gene therapy muscle gene therapy treatment of metabolic disorders treatment of neurological disorders |
gptkbp:isVisitedBy |
pre-existing immunity
|
gptkbp:targets |
specific tissues
|
gptkbp:usedFor |
gene therapy
|