|
gptkbp:instanceOf
|
Medical Treatment
|
|
gptkbp:approved_therapies
|
gptkb:Luxturna
gptkb:Strimvelis
gptkb:Zolgensma
|
|
gptkbp:canBe
|
gptkb:Somatic
Germline
Non-viral methods
Viral vectors
|
|
gptkbp:controversy
|
Ethical concerns
Germline modification
|
|
gptkbp:deliveredBy
|
Ex vivo
In vivo
|
|
gptkbp:field
|
Medicine
Genetics
|
|
gptkbp:first_clinical_trial
|
1990
|
|
gptkbp:first_disease_treated
|
gptkb:Adenosine_deaminase_deficiency
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Gene Therapy
|
|
gptkbp:method
|
Introduce genetic material into cells
|
|
gptkbp:notablePerson
|
gptkb:Katherine_High
James M. Wilson
W. French Anderson
|
|
gptkbp:purpose
|
Treat acquired diseases
Treat genetic disorders
|
|
gptkbp:regulates
|
gptkb:FDA
gptkb:EMA
|
|
gptkbp:researchArea
|
gptkb:cancer
gptkb:Cystic_fibrosis
gptkb:HIV/AIDS
gptkb:Sickle_cell_disease
Hemophilia
Muscular dystrophy
|
|
gptkbp:riskFactor
|
Immune response
Insertional mutagenesis
Off-target effects
|
|
gptkbp:technology
|
gptkb:CRISPR
gptkb:TALENs
gptkb:Zinc_finger_nucleases
|
|
gptkbp:treatment
|
gptkb:Spinal_muscular_atrophy
gptkb:cancer
gptkb:Cystic_fibrosis
gptkb:Hemophilia_B
gptkb:Duchenne_muscular_dystrophy
gptkb:ADA-SCID
gptkb:Hemophilia_A
Lymphoma
Leukemia
Neurological disorders
Retinal diseases
Metabolic disorders
Beta-thalassemia
Sickle cell anemia
Inherited blindness
Monogenic diseases
Polygenic diseases
|
|
gptkbp:bfsParent
|
gptkb:Cell_Therapy
|
|
gptkbp:bfsLayer
|
6
|