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- Retinitis Pigmentosa and Therapeutic Approaches: A Systematic . . .
Long-term safety and efficacy data, optimized delivery methods, and consideration of RP’s genetic heterogeneity are needed Furthermore, the high cost of gene- and cell-based therapies presents economic barriers to widespread adoption
- Gene Therapy for Inherited Retinal Disease: Long-Term . . .
Herein, we review studies investigating the episomal persistence of different recombinant adeno-associated virus (rAAV) vector genomes and the preclinical and clinical evidence of long-term effects of different RPE65 gene replacement therapies
- Gene Therapy for Retinitis Pigmentosa: Current Challenges and . . .
Promising positive safety and efficacy results from phase 1 2 clinical trials support the progress of RPGR XLRP gene replacement therapy, raising expectations for ongoing and upcoming phase 3 clinical trials that may lead to the long-awaited approval of RPGR gene therapy for treatment
- Retinitis Pigmentosa - StatPearls - NCBI Bookshelf
While there is currently no cure for RP, ongoing research is exploring potential treatments and therapies to slow its progression or restore vision in patients with RP Management often involves low-vision aids, orientation, and mobility training to help individuals adapt to their changing vision
- Full article: Emerging gene therapy products for RPGR . . .
The molecular and biological complexity of RPGR represents the biggest challenge to develop a gene therapy vector and to predict the likelihood of success and long-term effect of a gene therapy treatment
- Gene Therapy for Inherited Retinal Disease: Long-Term . . .
Herein, we review studies investigating the episomal persistence of different recombinant adeno-associated virus (rAAV) vector genomes and the preclinical and clinical evidence of long-term effects of different RPE65 gene replacement therapies
- Gene Therapy for Retinal Degenerative Diseases: Progress . . .
However, despite the immense progress over the last several decades, both old and new challenges remain, such as the long-term effects of treatments, immunogenicity, targeting, and manufacturing
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