An RNA aptamer restores defective bone growth in FGFR3-related skeletal dysplasia in mice

Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216224%3A14110%2F21%3A00119123" target="_blank" >RIV/00216224:14110/21:00119123 - isvavai.cz</a>
Alternative codes found
RIV/67985904:_____/21:00543675 RIV/00159816:_____/21:00075196 RIV/00216305:26620/21:PU141849
Result on the web
<a href="https://stm.sciencemag.org/content/13/592/eaba4226" target="_blank" >https://stm.sciencemag.org/content/13/592/eaba4226</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1126/scitranslmed.aba4226" target="_blank" >10.1126/scitranslmed.aba4226</a>

Result language
angličtina
Original language name
An RNA aptamer restores defective bone growth in FGFR3-related skeletal dysplasia in mice
Original language description
Achondroplasia is the most prevalent genetic form of dwarfism in humans and is caused by activating mutations in FGFR3 tyrosine kinase. The clinical need for a safe and effective inhibitor of FGFR3 is unmet, leaving achondroplasia currently incurable. Here, we evaluated RBM-007, an RNA aptamer previously developed to neutralize the FGFR3 ligand FGF2, for its activity against FGFR3. In cultured rat chondrocytes or mouse embryonal tibia organ culture, RBM-007 rescued the proliferation arrest, degradation of cartilaginous extracellular matrix, premature senescence, and impaired hypertrophic differentiation induced by FGFR3 signaling. In cartilage xenografts derived from induced pluripotent stem cells from individuals with achondroplasia, RBM-007 rescued impaired chondrocyte differentiation and maturation. When delivered by subcutaneous injection, RBM-007 restored defective skeletal growth in a mouse model of achondroplasia. We thus demonstrate a ligand-trap concept of targeting the cartilage FGFR3 and delineate a potential therapeutic approach for achondroplasia and other FGFR3-related skeletal dysplasias.
Czech name
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Czech description
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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

Project
Result was created during the realization of more than one project. More information in the Projects tab.
Continuities
P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)<br>S - Specificky vyzkum na vysokych skolach

Publication year
2021
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

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