Generation and characterization of FMR1 knockout zebrafish.
Fragile X syndrome (FXS) is one of the most common known causes of inherited mental retardation. The gene mutated in FXS is named FMR1, and is well conserved from human to Drosophila. In order to generate a genetic tool to study FMR1 function during vertebrate development, we generated two mutant al...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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Public Library of Science (PLoS)
2009-11-01
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| Series: | PLoS ONE |
| Online Access: | http://europepmc.org/articles/PMC2774943?pdf=render |
| _version_ | 1830460664030691328 |
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| author | Marjo J den Broeder Herma van der Linde Judith R Brouwer Ben A Oostra Rob Willemsen René F Ketting |
| author_facet | Marjo J den Broeder Herma van der Linde Judith R Brouwer Ben A Oostra Rob Willemsen René F Ketting |
| author_sort | Marjo J den Broeder |
| collection | DOAJ |
| description | Fragile X syndrome (FXS) is one of the most common known causes of inherited mental retardation. The gene mutated in FXS is named FMR1, and is well conserved from human to Drosophila. In order to generate a genetic tool to study FMR1 function during vertebrate development, we generated two mutant alleles of the fmr1 gene in zebrafish. Both alleles produce no detectable Fmr protein, and produce viable and fertile progeny with lack of obvious phenotypic features. This is in sharp contrast to published results based on morpholino mediated knock-down of fmr1, reporting defects in craniofacial development and neuronal branching in embryos. These phenotypes we specifically addressed in our knock-out animals, revealing no significant deviations from wild-type animals, suggesting that the published morpholino based fmr1 phenotypes are potential experimental artifacts. Therefore, their relation to fmr1 biology is questionable and morpholino induced fmr1 phenotypes should be avoided in screens for potential drugs suitable for the treatment of FXS. Importantly, a true genetic zebrafish model is now available which can be used to study FXS and to derive potential drugs for FXS treatment. |
| first_indexed | 2024-12-21T11:15:43Z |
| format | Article |
| id | doaj.art-6815080d89ce4b748a6126ecb66c5afb |
| institution | Directory Open Access Journal |
| issn | 1932-6203 |
| language | English |
| last_indexed | 2024-12-21T11:15:43Z |
| publishDate | 2009-11-01 |
| publisher | Public Library of Science (PLoS) |
| record_format | Article |
| series | PLoS ONE |
| spelling | doaj.art-6815080d89ce4b748a6126ecb66c5afb2022-12-21T19:05:56ZengPublic Library of Science (PLoS)PLoS ONE1932-62032009-11-01411e791010.1371/journal.pone.0007910Generation and characterization of FMR1 knockout zebrafish.Marjo J den BroederHerma van der LindeJudith R BrouwerBen A OostraRob WillemsenRené F KettingFragile X syndrome (FXS) is one of the most common known causes of inherited mental retardation. The gene mutated in FXS is named FMR1, and is well conserved from human to Drosophila. In order to generate a genetic tool to study FMR1 function during vertebrate development, we generated two mutant alleles of the fmr1 gene in zebrafish. Both alleles produce no detectable Fmr protein, and produce viable and fertile progeny with lack of obvious phenotypic features. This is in sharp contrast to published results based on morpholino mediated knock-down of fmr1, reporting defects in craniofacial development and neuronal branching in embryos. These phenotypes we specifically addressed in our knock-out animals, revealing no significant deviations from wild-type animals, suggesting that the published morpholino based fmr1 phenotypes are potential experimental artifacts. Therefore, their relation to fmr1 biology is questionable and morpholino induced fmr1 phenotypes should be avoided in screens for potential drugs suitable for the treatment of FXS. Importantly, a true genetic zebrafish model is now available which can be used to study FXS and to derive potential drugs for FXS treatment.http://europepmc.org/articles/PMC2774943?pdf=render |
| spellingShingle | Marjo J den Broeder Herma van der Linde Judith R Brouwer Ben A Oostra Rob Willemsen René F Ketting Generation and characterization of FMR1 knockout zebrafish. PLoS ONE |
| title | Generation and characterization of FMR1 knockout zebrafish. |
| title_full | Generation and characterization of FMR1 knockout zebrafish. |
| title_fullStr | Generation and characterization of FMR1 knockout zebrafish. |
| title_full_unstemmed | Generation and characterization of FMR1 knockout zebrafish. |
| title_short | Generation and characterization of FMR1 knockout zebrafish. |
| title_sort | generation and characterization of fmr1 knockout zebrafish |
| url | http://europepmc.org/articles/PMC2774943?pdf=render |
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