Properties (52)
Predicate | Object |
---|---|
gptkbp:instanceOf |
gptkb:Research_Institute
|
gptkbp:canBe |
therapeutic genes
|
gptkbp:hasEconomy |
neurons
|
gptkbp:hasProductLine |
recombinant DNA technology
|
gptkbp:hasRelatedPatent |
cardiovascular diseases
|
gptkbp:hasResearchFocus |
gene editing
|
gptkbp:hasSibling |
4.7 kb
|
https://www.w3.org/2000/01/rdf-schema#label |
AAV serotype 9
|
gptkbp:isAttendedBy |
animal models
intravenous injection |
gptkbp:isCharacterizedBy |
low immunogenicity
|
gptkbp:isCompatibleWith |
CRISPR technology
|
gptkbp:isConsidered |
a promising vector for CNS delivery
clinical applications in humans delivering large genes therapeutic gene delivery strategies treatment of inherited disorders |
gptkbp:isEvaluatedBy |
safety and efficacy
its effectiveness in various tissues |
gptkbp:isFacilitatedBy |
gene expression in target cells
|
gptkbp:isInvolvedIn |
retinal diseases
|
gptkbp:isKnownFor |
a non-pathogenic virus
high transduction efficiency its ability to integrate into host genome. skeletal muscle cells tropism for the central nervous system |
gptkbp:isNotedFor |
long-term expression of transgenes
|
gptkbp:isPartOf |
biomedical research initiatives
gene delivery systems AAV vector system advanced gene therapy techniques gene therapy protocols innovative therapeutic approaches other AAV serotypes therapeutic applications of AAVs vector development strategies |
gptkbp:isStudiedIn |
cancer therapy
neurological disorders clinical applications for rare diseases its ability to cross the blood-brain barrier its potential in treating genetic disorders |
gptkbp:isUsedBy |
muscular dystrophy
|
gptkbp:isUsedFor |
neurodegenerative diseases
|
gptkbp:isUsedIn |
gene therapy
vaccine development |
gptkbp:isUtilizedFor |
gene function
treatment of spinal muscular atrophy its role in gene therapy delivery mechanisms its use in ocular gene therapy |
gptkbp:isUtilizedIn |
clinical trials
preclinical studies |
gptkbp:isValuedFor |
AAV2
|