Statements (58)
| Predicate | Object |
|---|---|
| gptkbp:instance_of |
gptkb:immunotherapy
|
| gptkbp:advocacy |
important for awareness
|
| gptkbp:affects |
immune system
|
| gptkbp:associated_with |
gptkb:adenosine_deaminase_deficiency
|
| gptkbp:caused_by |
ADA gene mutation
|
| gptkbp:clinical_trial |
gptkb:pneumonia
failure to thrive recurrent infections ongoing for new therapies chronic diarrhea oral thrush critical for understanding disease important for future treatments |
| gptkbp:collaborations |
between institutions
|
| gptkbp:condition |
metabolic disorders
autoimmune disorders |
| gptkbp:diagnosis |
newborn screening
|
| gptkbp:educational_resources |
available for patients and families
|
| gptkbp:emergency_services |
critical for infections
|
| gptkbp:financial_support |
available through organizations
|
| gptkbp:first_described_by |
1970s
|
| gptkbp:first_successful_treatment |
1990s
|
| gptkbp:funding |
increased in recent years
|
| gptkbp:gene_therapy_success_rate |
high in clinical trials
|
| gptkbp:genetic_studies |
available for diagnosis
recommended for families ongoing to find new therapies |
| gptkbp:global_health_issue |
recognized by health organizations
|
| gptkbp:historical_significance |
first gene therapy success in humans
|
| https://www.w3.org/2000/01/rdf-schema#label |
ADA-SCID
|
| gptkbp:immunological_feature |
absence of B cells
absence of T cells absence of NK cells |
| gptkbp:inherits_from |
autosomal recessive
|
| gptkbp:is_a_basis_for |
well understood
|
| gptkbp:is_a_resource_for |
available for families
|
| gptkbp:is_common_in |
certain populations
|
| gptkbp:lifespan |
varies with treatment
|
| gptkbp:long-term_monitoring |
necessary for treated patients
|
| gptkbp:patient_population |
varies by region
|
| gptkbp:prevalence |
rare disorder
|
| gptkbp:provides_guidance_on |
developed for management
|
| gptkbp:public_awareness |
increased through advocacy groups
|
| gptkbp:research |
ongoing clinical trials
|
| gptkbp:research_focus |
long-term outcomes of treatments
improving gene therapy techniques understanding immune system development |
| gptkbp:screenings |
PCR-based tests
|
| gptkbp:side_effect |
improved with treatment
significant without treatment |
| gptkbp:symptoms |
increased susceptibility to infections
|
| gptkbp:treatment |
gptkb:gene_therapy
gptkb:Biology bone marrow transplant evolving rapidly improving with advancements |
| gptkbp:bfsParent |
gptkb:immunotherapy
|
| gptkbp:bfsLayer |
4
|