Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success
Abstract Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of...
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| Format: | Article |
| Language: | English |
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Springer Nature
2016-04-01
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| Series: | EMBO Molecular Medicine |
| Online Access: | https://doi.org/10.15252/emmm.201606325 |
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| _version_ | 1849761772390580224 |
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| author | Tuan Huy Nguyen Ignacio Anegon |
| author_facet | Tuan Huy Nguyen Ignacio Anegon |
| author_sort | Tuan Huy Nguyen |
| collection | DOAJ |
| description | Abstract Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of life, while 5–20% suffices to ameliorate the bleeding disorder. A paper by Guan et al (2016) in this issue of EMBO Molecular Medicine reports on the direct CRISPRs/Cas9‐mediated correction in the liver of a hemophilia‐causing point mutation in FIX. |
| format | Article |
| id | doaj-art-82cd7f84374d4624bebcce2420092fa3 |
| institution | DOAJ |
| issn | 1757-4676 1757-4684 |
| language | English |
| publishDate | 2016-04-01 |
| publisher | Springer Nature |
| record_format | Article |
| series | EMBO Molecular Medicine |
| spelling | doaj-art-82cd7f84374d4624bebcce2420092fa32025-08-20T03:05:55ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842016-04-018543944110.15252/emmm.201606325Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to successTuan Huy Nguyen0Ignacio Anegon1INSERM, UMR 1064‐Center for Research in Transplantation and ImmunologyINSERM, UMR 1064‐Center for Research in Transplantation and ImmunologyAbstract Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of life, while 5–20% suffices to ameliorate the bleeding disorder. A paper by Guan et al (2016) in this issue of EMBO Molecular Medicine reports on the direct CRISPRs/Cas9‐mediated correction in the liver of a hemophilia‐causing point mutation in FIX.https://doi.org/10.15252/emmm.201606325 |
| spellingShingle | Tuan Huy Nguyen Ignacio Anegon Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success EMBO Molecular Medicine |
| title | Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success |
| title_full | Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success |
| title_fullStr | Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success |
| title_full_unstemmed | Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success |
| title_short | Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success |
| title_sort | successful correction of hemophilia by crispr cas9 genome editing in vivo delivery vector and immune responses are the key to success |
| url | https://doi.org/10.15252/emmm.201606325 |
| work_keys_str_mv | AT tuanhuynguyen successfulcorrectionofhemophiliabycrisprcas9genomeeditinginvivodeliveryvectorandimmuneresponsesarethekeytosuccess AT ignacioanegon successfulcorrectionofhemophiliabycrisprcas9genomeeditinginvivodeliveryvectorandimmuneresponsesarethekeytosuccess |