gene therapy

GPTKB entity

Statements (101)
Predicate Object
gptkbp:instanceOf gptkb:cancer
gptkb:psychotherapy
immunotherapy
gptkbp:administeredBy intravenous infusion
gptkbp:alsoKnownAs chimeric antigen receptor T-cell therapy
gptkbp:alternativeName CAR-T_cell_therapy
CAR_T-cell_therapy
cell_therapy
gptkbp:approvalYear 2017
gptkbp:approved_therapies gptkb:Luxturna
gptkb:Strimvelis
gptkb:Zynteglo
gptkb:Zolgensma
gptkbp:approvedBy gptkb:FDA
1990
multiple myeloma
diffuse large B-cell lymphoma
B-cell acute lymphoblastic leukemia
gptkbp:canBe infectious diseases
rare diseases
eye diseases
neurological disorders
germline
somatic
monogenic diseases
non-viral vectors
polygenic diseases
viral vectors
gptkbp:clinicalTrialPhase gptkb:Phase_I
Phase II
Phase III
gptkbp:contraindication active infection
uncontrolled CNS disease
gptkbp:cost hundreds of thousands of dollars
gptkbp:deliveredBy ex vivo
in vivo
gptkbp:developedBy gptkb:Carl_June
gptkbp:ethical_issues accessibility
cost
germline modification
gptkbp:field gptkb:medicine
genetics
gptkbp:first_patient gptkb:Ashanti_DeSilva
gptkbp:involves chimeric antigen receptor
gptkbp:marketedAs gptkb:Gilead_Sciences
gptkb:Novartis
gptkb:Bristol_Myers_Squibb
gptkbp:mechanismOfAction redirects T cells to recognize and kill cancer cells
gptkbp:method insertion of genetic material
modification of genes
replacement of faulty genes
gptkbp:monitors oncologist
hematologist
gptkbp:potentialComplication gptkb:tumor_lysis_syndrome
infections
B-cell aplasia
hypogammaglobulinemia
gptkbp:product gptkb:Abecma
gptkb:Tecartus
gptkb:Kymriah
gptkb:Yescarta
gptkb:Breyanzi
gptkbp:purpose treat acquired diseases
treat genetic disorders
gptkbp:regulates gptkb:FDA
gptkb:EMA
gene expression
gptkbp:relatedTo gptkb:CRISPR
genome editing
gene therapy
RNA therapy
gptkbp:requires leukapheresis
lymphodepleting chemotherapy
personalized manufacturing
gptkbp:researched_since 1970s
gptkbp:researchInterest solid tumors
improving persistence
reducing toxicity
gptkbp:riskFactor gptkb:cancer
immune response
off-target effects
insertional mutagenesis
gptkbp:sideEffect neurotoxicity
cytokine release syndrome
gptkbp:storage cryopreservation
gptkbp:studiedBy gptkb:University_of_Pennsylvania
gptkb:National_Cancer_Institute
gptkbp:target cancer cells
gptkbp:targetsAntigen gptkb:CD19
gptkb:BCMA
gptkbp:treatment gptkb:cancer
gptkb:anemia
hemophilia
sickle cell disease
immunodeficiency
cystic fibrosis
muscular dystrophy
gptkbp:uses genetically engineered T cells
gptkbp:bfsParent gptkb:Daniel_G._Anderson
gptkb:biotechnology
gptkbp:bfsLayer 4