Immunology of Retinitis Pigmentosa and Gene Therapy-Associated Uveitis.
Cold Spring Harb Perspect Med
; 14(1)2024 Jan 02.
Article
in En
| MEDLINE
| ID: mdl-37037600
ABSTRACT
The underlying immune state of inherited retinal degenerations (IRDs) and retinitis pigmentosa (RP) has been an emerging area of interest, wherein the consequences have never been greater given the widespread recognition of gene therapy-associated uveitis (GTU) in gene therapy clinical trials. Whereas some evidence suggests that the adaptive immune system may play a role, the majority of studies indicate that the innate immune system is likely the primary driver of neuroinflammation in RP. During retinal degeneration, discrete mechanisms activate resident microglia and promote infiltrating macrophages that can either be protective or detrimental to photoreceptor cell death. This persistent stimulation of innate immunity, overlaid by the introduction of viral antigens as part of gene therapy, has the potential to trigger a complex microglia/macrophage-driven proinflammatory state. A better understanding of the immune pathophysiology in IRD and GTU will be necessary to improve the success of developing novel treatments for IRDs.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Retinal Degeneration
/
Uveitis
/
Retinitis Pigmentosa
Type of study:
Risk_factors_studies
Limits:
Humans
Language:
En
Journal:
Cold Spring Harb Perspect Med
Year:
2024
Document type:
Article