Decoding retinitis pigmentosa: molecular targets and therapy with focus on pre-mRNA splicing
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F68378050%3A_____%2F25%3A00644364" target="_blank" >RIV/68378050:_____/25:00644364 - isvavai.cz</a>
Result on the web
<a href="https://link.springer.com/article/10.1007/s00018-025-05987-0" target="_blank" >https://link.springer.com/article/10.1007/s00018-025-05987-0</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1007/s00018-025-05987-0" target="_blank" >10.1007/s00018-025-05987-0</a>
Alternative languages
Result language
angličtina
Original language name
Decoding retinitis pigmentosa: molecular targets and therapy with focus on pre-mRNA splicing
Original language description
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.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
10601 - Cell biology
Result continuities
Project
<a href="/en/project/EH22_008%2F0004575" target="_blank" >EH22_008/0004575: RNA for therapy</a><br>
Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2025
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Cellular and Molecular Life Sciences
ISSN
1420-682X
e-ISSN
1420-9071
Volume of the periodical
83
Issue of the periodical within the volume
1
Country of publishing house
CH - SWITZERLAND
Number of pages
14
Pages from-to
20
UT code for WoS article
001651222200001
EID of the result in the Scopus database
2-s2.0-105026344723