Organ targeted prenatal gene therapy—how far are we?
Prenatal gene therapy aims to deliver genes to cells and tissues early in prenatal life, allowing correction of a genetic defect, before long-term tissue damage has occurred. In contrast to postnatal gene therapy, prenatal application can target genes to a large population of dividing stem cells, an...
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2011
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| Online Access: | http://hdl.handle.net/10725/3973 http://dx.doi.org/10.1002/pd.2787 http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.php |
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| _version_ | 1864513462551445504 |
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| author | Abi Nader, Khalil |
| author2 | Mehta, Vedanta Waddington, Simon David, Anna L. |
| author2_role | author author author |
| author_facet | Abi Nader, Khalil Mehta, Vedanta Waddington, Simon David, Anna L. |
| author_role | author |
| dc.creator.none.fl_str_mv | Abi Nader, Khalil Mehta, Vedanta Waddington, Simon David, Anna L. |
| dc.date.none.fl_str_mv | 2011 2016-06-08T05:57:27Z 2016-06-08T05:57:27Z 2016-06-08 |
| dc.identifier.none.fl_str_mv | 0197-3851 http://hdl.handle.net/10725/3973 http://dx.doi.org/10.1002/pd.2787 Mehta, V., Abi Nader, K., Waddington, S., & David, A. L. (2011). Organ targeted prenatal gene therapy—how far are we?. Prenatal diagnosis, 31(7), 720-734. http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.php file:///C:/Users/lib.arch/Downloads/Mehta_et_al-2011-Prenatal_Diagnosis.pdf |
| dc.language.none.fl_str_mv | en |
| dc.relation.none.fl_str_mv | Prenatal Diagnosis |
| dc.rights.*.fl_str_mv | info:eu-repo/semantics/openAccess |
| dc.title.none.fl_str_mv | Organ targeted prenatal gene therapy—how far are we? |
| dc.type.none.fl_str_mv | Article info:eu-repo/semantics/publishedVersion info:eu-repo/semantics/article |
| description | Prenatal gene therapy aims to deliver genes to cells and tissues early in prenatal life, allowing correction of a genetic defect, before long-term tissue damage has occurred. In contrast to postnatal gene therapy, prenatal application can target genes to a large population of dividing stem cells, and the smaller fetal size allows a higher vector-to-target cell ratio to be achieved. Early-gestation delivery may allow the development of immune tolerance to the transgenic protein which would facilitate postnatal repeat vector administration if needed. Targeting particular organs will depend on manipulating the vector to achieve selective tropism and on choosing the most appropriate gestational age and injection method for fetal delivery. Intra-amniotic injection reaches the skin, and other organs that are bathed in the fluid however since gene transfer to the lung and gut is usually poor more direct injection methods will be needed. Delivery to the liver and blood can be achieved by systemic delivery via the umbilical vein or peritoneal cavity. Gene transfer to the central nervous system in the fetus is difficult but newer vectors are available that transduce neuronal tissue even after systemic delivery. Copyright 2011 John Wiley & Sons, Ltd |
| eu_rights_str_mv | openAccess |
| format | article |
| id | LAURepo_38d36c19eb2eb038f64adadbc8c1a5ca |
| identifier_str_mv | 0197-3851 Mehta, V., Abi Nader, K., Waddington, S., & David, A. L. (2011). Organ targeted prenatal gene therapy—how far are we?. Prenatal diagnosis, 31(7), 720-734. file:///C:/Users/lib.arch/Downloads/Mehta_et_al-2011-Prenatal_Diagnosis.pdf |
| language_invalid_str_mv | en |
| network_acronym_str | LAURepo |
| network_name_str | Lebanese American University repository |
| oai_identifier_str | oai:laur.lau.edu.lb:10725/3973 |
| publishDate | 2011 |
| repository.mail.fl_str_mv | |
| repository.name.fl_str_mv | |
| repository_id_str | |
| spelling | Organ targeted prenatal gene therapy—how far are we?Abi Nader, KhalilMehta, VedantaWaddington, SimonDavid, Anna L.Prenatal gene therapy aims to deliver genes to cells and tissues early in prenatal life, allowing correction of a genetic defect, before long-term tissue damage has occurred. In contrast to postnatal gene therapy, prenatal application can target genes to a large population of dividing stem cells, and the smaller fetal size allows a higher vector-to-target cell ratio to be achieved. Early-gestation delivery may allow the development of immune tolerance to the transgenic protein which would facilitate postnatal repeat vector administration if needed. Targeting particular organs will depend on manipulating the vector to achieve selective tropism and on choosing the most appropriate gestational age and injection method for fetal delivery. Intra-amniotic injection reaches the skin, and other organs that are bathed in the fluid however since gene transfer to the lung and gut is usually poor more direct injection methods will be needed. Delivery to the liver and blood can be achieved by systemic delivery via the umbilical vein or peritoneal cavity. Gene transfer to the central nervous system in the fetus is difficult but newer vectors are available that transduce neuronal tissue even after systemic delivery. Copyright 2011 John Wiley & Sons, LtdPublishedN/A2016-06-08T05:57:27Z2016-06-08T05:57:27Z20112016-06-08Articleinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/article0197-3851http://hdl.handle.net/10725/3973http://dx.doi.org/10.1002/pd.2787Mehta, V., Abi Nader, K., Waddington, S., & David, A. L. (2011). Organ targeted prenatal gene therapy—how far are we?. Prenatal diagnosis, 31(7), 720-734.http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.phpfile:///C:/Users/lib.arch/Downloads/Mehta_et_al-2011-Prenatal_Diagnosis.pdfenPrenatal Diagnosisinfo:eu-repo/semantics/openAccessoai:laur.lau.edu.lb:10725/39732021-03-19T10:03:20Z |
| spellingShingle | Organ targeted prenatal gene therapy—how far are we? Abi Nader, Khalil |
| status_str | publishedVersion |
| title | Organ targeted prenatal gene therapy—how far are we? |
| title_full | Organ targeted prenatal gene therapy—how far are we? |
| title_fullStr | Organ targeted prenatal gene therapy—how far are we? |
| title_full_unstemmed | Organ targeted prenatal gene therapy—how far are we? |
| title_short | Organ targeted prenatal gene therapy—how far are we? |
| title_sort | Organ targeted prenatal gene therapy—how far are we? |
| url | http://hdl.handle.net/10725/3973 http://dx.doi.org/10.1002/pd.2787 http://libraries.lau.edu.lb/research/laur/terms-of-use/articles.php |