ReviewCellular and molecular life sciences : CMLS2025
Decoding retinitis pigmentosa: molecular targets and therapy with focus on pre-mRNA splicing.
Review in Cellular and molecular life sciences : CMLS, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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0 citing papers in PubMed.
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Authors and funding
2 authors.
Funding
Abstract
Retinitis pigmentosa (RP) is the most common cause of inherited blindness, with mutations in splicing factors playing a significant role in its pathogenesis. Many scientists have been puzzled by the fact that mutations in several key spliceosomal components have such a confined effect on the retina. In this review, we summarize findings gained from studies using cell culture, animal models, and retinal organoids to better understand the molecular mechanisms underlying the tissue specificity of splicing factor dysfunction to retinal degeneration. Although RP currently has no definitive cure, recent advances in gene therapy, antisense oligonucleotides, and cell transplantation are opening new therapeutic approaches to slow disease progression and preserve retinal function. We also discuss the strengths and challenges of current strategies and point to the critical improvements required for their successful clinical application.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.