Statements (59)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:age |
1 year and older
|
| gptkbp:approves |
gene therapy for inherited retinal disease
|
| gptkbp:associated_with |
improved quality of life
|
| gptkbp:basedOn |
AAV technology
|
| gptkbp:clinicalTrials |
gptkb:United_States
Europe Phase 3 improved vision |
| gptkbp:delivers |
retinal cells
RPE65 gene |
| gptkbp:enrollment |
patients with RPE65 mutation
|
| gptkbp:frequency |
once per eye
|
| gptkbp:has_a_focus_on |
clinical studies
|
| https://www.w3.org/2000/01/rdf-schema#label |
Luxturna
|
| gptkbp:impact |
inflammation
rare genetic disorders targets genetic mutations |
| gptkbp:improves |
visual acuity
light sensitivity |
| gptkbp:involves |
viral vector
|
| gptkbp:is_a |
one-time treatment
ocular gene therapy |
| gptkbp:is_a_document_that |
restores vision
has been widely discussed in media has implications for future treatments. has received positive reviews has shown promise in trials has transformed patient outcomes |
| gptkbp:is_a_model_for |
future gene therapies
|
| gptkbp:is_a_place_for |
certain types of blindness
|
| gptkbp:is_a_subject_of |
ongoing research
|
| gptkbp:is_a_time_for |
genetic vision loss
|
| gptkbp:is_essential_for |
ophthalmology
|
| gptkbp:is_part_of |
regenerative medicine
precision medicine orphan_drug_program |
| gptkbp:is_recognized_for |
gptkb:FDA
medical community |
| gptkbp:is_studied_in |
gene therapy efficacy
|
| gptkbp:is_supported_by |
patient advocacy groups
|
| gptkbp:is_used_in |
clinical settings
|
| gptkbp:landmark |
gene therapy history
|
| gptkbp:mandates |
vision loss
hereditary retinal disease |
| gptkbp:marketedAs |
gptkb:Luxturna
|
| gptkbp:offers |
approximately $850,000
|
| gptkbp:operates_in |
ophthalmologist
|
| gptkbp:produces |
gptkb:Spark_Therapeutics
|
| gptkbp:products |
biotechnology
requires careful patient selection |
| gptkbp:requires |
genetic testing
specialized eye care |
| gptkbp:serves |
Leber congenital amaurosis
retinal dystrophy |
| gptkbp:triggerType |
gene replacement therapy
|
| gptkbp:utilizes |
subretinal injection
|
| gptkbp:was_a_catalyst_for |
inherited retinal diseases
|
| gptkbp:year |
2017
|