New insight into the molecular basis of hemophilia A

Since publication of the sequence of the factor VIII gene (F8) in 1984, a large number of mutations that cause hemophilia A (HA) have been identified.With the technical advances associated with mutation screenings, it is now possible to identify a putative F8 sequence alteration in the great majorit...

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التفاصيل البيبلوغرافية
المؤلف الرئيسي: El Maarri, Osman (author)
مؤلفون آخرون: Oldenburg, Johannes (author)
التنسيق: article
منشور في: 2006
الوصول للمادة أونلاين:http://hdl.handle.net/10725/6203
http://dx.doi.org/10.1532/IJH97.06012
http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.php
https://link.springer.com/content/pdf/10.1532%2FIJH97.06012.pdf
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author El Maarri, Osman
author2 Oldenburg, Johannes
author2_role author
author_facet El Maarri, Osman
Oldenburg, Johannes
author_role author
dc.creator.none.fl_str_mv El Maarri, Osman
Oldenburg, Johannes
dc.date.none.fl_str_mv 2006
2017-09-15T09:47:05Z
2017-09-15T09:47:05Z
2017-09-15
dc.identifier.none.fl_str_mv 1865-3774
http://hdl.handle.net/10725/6203
http://dx.doi.org/10.1532/IJH97.06012
Oldenburg, J., & El-Maarri, O. (2006). New insight into the molecular basis of hemophilia A. International journal of hematology, 83(2), 96-102.
http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.php
https://link.springer.com/content/pdf/10.1532%2FIJH97.06012.pdf
dc.language.none.fl_str_mv en
dc.relation.none.fl_str_mv International Journal of Hematology
dc.rights.*.fl_str_mv info:eu-repo/semantics/openAccess
dc.title.none.fl_str_mv New insight into the molecular basis of hemophilia A
dc.type.none.fl_str_mv Article
info:eu-repo/semantics/publishedVersion
info:eu-repo/semantics/article
description Since publication of the sequence of the factor VIII gene (F8) in 1984, a large number of mutations that cause hemophilia A (HA) have been identified.With the technical advances associated with mutation screenings, it is now possible to identify a putative F8 sequence alteration in the great majority of HA patients. The mutation spectrum includes 2 inversion hot spots (intron 1 and intron 22 inversions) mediated by intrachromosomal recombination between 2 copies of long inverted repeats, one of which lies within the F8 gene whereas the other is extragenic. Point mutations are distributed over all of the exons, and deletions or insertions of different sizes and mutations affecting splice sites account for the rest of the known mutations. In a small number of cases, however, we are unable to find any disease-determining DNA changes in the coding regions of the F8 gene. This fact points to possibilities of unknown gene rearrangements that disrupt the F8 gene or mutations in other genes that play a role in the processing/secretion of the factor VIII protein. Moreover, the proof of an absence of F8 messenger RNA (mRNA) in one patient points to either a defect in the expression of F8 mRNA or its rapid degradation, which may represent a novel mechanism leading to HA.
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Oldenburg, J., & El-Maarri, O. (2006). New insight into the molecular basis of hemophilia A. International journal of hematology, 83(2), 96-102.
language_invalid_str_mv en
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spelling New insight into the molecular basis of hemophilia AEl Maarri, OsmanOldenburg, JohannesSince publication of the sequence of the factor VIII gene (F8) in 1984, a large number of mutations that cause hemophilia A (HA) have been identified.With the technical advances associated with mutation screenings, it is now possible to identify a putative F8 sequence alteration in the great majority of HA patients. The mutation spectrum includes 2 inversion hot spots (intron 1 and intron 22 inversions) mediated by intrachromosomal recombination between 2 copies of long inverted repeats, one of which lies within the F8 gene whereas the other is extragenic. Point mutations are distributed over all of the exons, and deletions or insertions of different sizes and mutations affecting splice sites account for the rest of the known mutations. In a small number of cases, however, we are unable to find any disease-determining DNA changes in the coding regions of the F8 gene. This fact points to possibilities of unknown gene rearrangements that disrupt the F8 gene or mutations in other genes that play a role in the processing/secretion of the factor VIII protein. Moreover, the proof of an absence of F8 messenger RNA (mRNA) in one patient points to either a defect in the expression of F8 mRNA or its rapid degradation, which may represent a novel mechanism leading to HA.PublishedN/A2017-09-15T09:47:05Z2017-09-15T09:47:05Z20062017-09-15Articleinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/article1865-3774http://hdl.handle.net/10725/6203http://dx.doi.org/10.1532/IJH97.06012Oldenburg, J., & El-Maarri, O. (2006). New insight into the molecular basis of hemophilia A. International journal of hematology, 83(2), 96-102.http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.phphttps://link.springer.com/content/pdf/10.1532%2FIJH97.06012.pdfenInternational Journal of Hematologyinfo:eu-repo/semantics/openAccessoai:laur.lau.edu.lb:10725/62032021-03-19T10:43:10Z
spellingShingle New insight into the molecular basis of hemophilia A
El Maarri, Osman
status_str publishedVersion
title New insight into the molecular basis of hemophilia A
title_full New insight into the molecular basis of hemophilia A
title_fullStr New insight into the molecular basis of hemophilia A
title_full_unstemmed New insight into the molecular basis of hemophilia A
title_short New insight into the molecular basis of hemophilia A
title_sort New insight into the molecular basis of hemophilia A
url http://hdl.handle.net/10725/6203
http://dx.doi.org/10.1532/IJH97.06012
http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.php
https://link.springer.com/content/pdf/10.1532%2FIJH97.06012.pdf