A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
A longstanding goal for the treatment of hemophilia B is the development of a gene transfer strategy that can maintain sustained production of clotting factor IX (F.IX) in the absence of an immune response. To this end, we have sought to use lentiviral vectors (LVs) as a means for systemic gene tran...
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| Published in | Blood Vol. 110; no. 13; pp. 4144 - 4152 |
|---|---|
| Main Authors | , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Inc
15.12.2007
|
| Subjects | |
| Online Access | Get full text |
| ISSN | 0006-4971 1528-0020 |
| DOI | 10.1182/blood-2007-03-078493 |
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| Abstract | A longstanding goal for the treatment of hemophilia B is the development of a gene transfer strategy that can maintain sustained production of clotting factor IX (F.IX) in the absence of an immune response. To this end, we have sought to use lentiviral vectors (LVs) as a means for systemic gene transfer. Unfortunately, initial evaluation of LVs expressing F.IX from hepatocyte-specific promoters failed to achieve sustained F.IX expression in hemophilia B mice due to the induction of an anti-F.IX cellular immune response. Further analysis suggested that this may be a result of off-target transgene expression in hematopoietic-lineage cells of the spleen. In order to overcome this problem, we modified our vector to contain a target sequence for the hematopoietic-specific microRNA, miR-142-3p. This eliminated off-target expression in hematopoietic cells, and enabled sustained gene transfer in hemophilia B mice for more than 280 days after injection. Treated mice had more than 10% normal F.IX activity, no detectable anti-F.IX antibodies, and were unresponsive to F.IX immunization. Importantly, the mice survived tail-clip challenge, thus demonstrating phenotypic correction of their bleeding diathesis. This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B. |
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| AbstractList | A longstanding goal for the treatment of hemophilia B is the development of a gene transfer strategy that can maintain sustained production of clotting factor IX (F.IX) in the absence of an immune response. To this end, we have sought to use lentiviral vectors (LVs) as a means for systemic gene transfer. Unfortunately, initial evaluation of LVs expressing F.IX from hepatocyte-specific promoters failed to achieve sustained F.IX expression in hemophilia B mice due to the induction of an anti-F.IX cellular immune response. Further analysis suggested that this may be a result of off-target transgene expression in hematopoietic-lineage cells of the spleen. In order to overcome this problem, we modified our vector to contain a target sequence for the hematopoietic-specific microRNA, miR-142-3p. This eliminated off-target expression in hematopoietic cells, and enabled sustained gene transfer in hemophilia B mice for more than 280 days after injection. Treated mice had more than 10% normal F.IX activity, no detectable anti-F.IX antibodies, and were unresponsive to F.IX immunization. Importantly, the mice survived tail-clip challenge, thus demonstrating phenotypic correction of their bleeding diathesis. This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B. A longstanding goal for the treatment of hemophilia B is the development of a gene transfer strategy that can maintain sustained production of clotting factor IX (F.IX) in the absence of an immune response. To this end, we have sought to use lentiviral vectors (LVs) as a means for systemic gene transfer. Unfortunately, initial evaluation of LVs expressing F.IX from hepatocyte-specific promoters failed to achieve sustained F.IX expression in hemophilia B mice due to the induction of an anti-F.IX cellular immune response. Further analysis suggested that this may be a result of off-target transgene expression in hematopoietic-lineage cells of the spleen. In order to overcome this problem, we modified our vector to contain a target sequence for the hematopoietic-specific microRNA, miR-142-3p. This eliminated off-target expression in hematopoietic cells, and enabled sustained gene transfer in hemophilia B mice for more than 280 days after injection. Treated mice had more than 10% normal F.IX activity, no detectable anti-F.IX antibodies, and were unresponsive to F.IX immunization. Importantly, the mice survived tail-clip challenge, thus demonstrating phenotypic correction of their bleeding diathesis. This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B.A longstanding goal for the treatment of hemophilia B is the development of a gene transfer strategy that can maintain sustained production of clotting factor IX (F.IX) in the absence of an immune response. To this end, we have sought to use lentiviral vectors (LVs) as a means for systemic gene transfer. Unfortunately, initial evaluation of LVs expressing F.IX from hepatocyte-specific promoters failed to achieve sustained F.IX expression in hemophilia B mice due to the induction of an anti-F.IX cellular immune response. Further analysis suggested that this may be a result of off-target transgene expression in hematopoietic-lineage cells of the spleen. In order to overcome this problem, we modified our vector to contain a target sequence for the hematopoietic-specific microRNA, miR-142-3p. This eliminated off-target expression in hematopoietic cells, and enabled sustained gene transfer in hemophilia B mice for more than 280 days after injection. Treated mice had more than 10% normal F.IX activity, no detectable anti-F.IX antibodies, and were unresponsive to F.IX immunization. Importantly, the mice survived tail-clip challenge, thus demonstrating phenotypic correction of their bleeding diathesis. This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B. |
| Author | D'Angelo, Armando Sergi, Lucia Sergi Della Valle, Patrizia Naldini, Luigi Lombardo, Angelo Brown, Brian D. Annoni, Andrea Cantore, Alessio |
| Author_xml | – sequence: 1 givenname: Brian D. surname: Brown fullname: Brown, Brian D. organization: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan – sequence: 2 givenname: Alessio surname: Cantore fullname: Cantore, Alessio organization: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan – sequence: 3 givenname: Andrea surname: Annoni fullname: Annoni, Andrea organization: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan – sequence: 4 givenname: Lucia Sergi surname: Sergi fullname: Sergi, Lucia Sergi organization: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan – sequence: 5 givenname: Angelo surname: Lombardo fullname: Lombardo, Angelo organization: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan – sequence: 6 givenname: Patrizia surname: Della Valle fullname: Della Valle, Patrizia organization: Coagulation Service and Thrombosis Research Unit, Fondazione Centro San Raffaele Del Monte Tabor Istituto dì Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Hospital, Milan – sequence: 7 givenname: Armando surname: D'Angelo fullname: D'Angelo, Armando organization: Coagulation Service and Thrombosis Research Unit, Fondazione Centro San Raffaele Del Monte Tabor Istituto dì Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Hospital, Milan – sequence: 8 givenname: Luigi surname: Naldini fullname: Naldini, Luigi email: naldini.luigi@hsr.it organization: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/17726165$$D View this record in MEDLINE/PubMed |
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| SubjectTerms | Animals Antibodies Blood Cells - metabolism Factor IX - administration & dosage Factor IX - immunology Gene Transfer Techniques Genetic Therapy - methods Genetic Vectors - therapeutic use Hemophilia B - therapy Lentivirus - genetics Mice MicroRNAs - genetics MicroRNAs - pharmacology |
| Title | A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice |
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