AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated...
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| Published in | Blood Vol. 101; no. 8; pp. 2963 - 2972 |
|---|---|
| Main Authors | , , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Washington, DC
Elsevier Inc
15.04.2003
The Americain Society of Hematology |
| Subjects | |
| Online Access | Get full text |
| ISSN | 0006-4971 1528-0020 |
| DOI | 10.1182/blood-2002-10-3296 |
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| Abstract | Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. In an open-label dose-escalation study, adult men with severe hemophilia B (F.IX < 1%) due to a missense mutation were injected at multiple intramuscular sites with an rAAV vector. At doses ranging from 2 × 1011vector genomes (vg)/kg to 1.8 × 1012vg/kg, there was no evidence of local or systemic toxicity up to 40 months after injection. Muscle biopsies of injection sites performed 2 to 10 months after vector administration confirmed gene transfer as evidenced by Southern blot and transgene expression as evidenced by immunohistochemical staining. Pre-existing high-titer antibodies to AAV did not prevent gene transfer or expression. Despite strong evidence for gene transfer and expression, circulating levels of F.IX were in all cases less than 2% and most were less than 1%. Although more extensive transduction of muscle fibers will be required to develop a therapy that reliably raises circulating levels to more than 1% in all subjects, these results of the first parenteral administration of rAAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans in a manner similar to that seen in animals. |
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| AbstractList | Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. In an open-label dose-escalation study, adult men with severe hemophilia B (F.IX < 1%) due to a missense mutation were injected at multiple intramuscular sites with an rAAV vector. At doses ranging from 2 x 10(11) vector genomes (vg)/kg to 1.8 x 10(12) vg/kg, there was no evidence of local or systemic toxicity up to 40 months after injection. Muscle biopsies of injection sites performed 2 to 10 months after vector administration confirmed gene transfer as evidenced by Southern blot and transgene expression as evidenced by immunohistochemical staining. Pre-existing high-titer antibodies to AAV did not prevent gene transfer or expression. Despite strong evidence for gene transfer and expression, circulating levels of F.IX were in all cases less than 2% and most were less than 1%. Although more extensive transduction of muscle fibers will be required to develop a therapy that reliably raises circulating levels to more than 1% in all subjects, these results of the first parenteral administration of rAAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans in a manner similar to that seen in animals. Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. In an open-label dose-escalation study, adult men with severe hemophilia B (F.IX < 1%) due to a missense mutation were injected at multiple intramuscular sites with an rAAV vector. At doses ranging from 2 × 1011vector genomes (vg)/kg to 1.8 × 1012vg/kg, there was no evidence of local or systemic toxicity up to 40 months after injection. Muscle biopsies of injection sites performed 2 to 10 months after vector administration confirmed gene transfer as evidenced by Southern blot and transgene expression as evidenced by immunohistochemical staining. Pre-existing high-titer antibodies to AAV did not prevent gene transfer or expression. Despite strong evidence for gene transfer and expression, circulating levels of F.IX were in all cases less than 2% and most were less than 1%. Although more extensive transduction of muscle fibers will be required to develop a therapy that reliably raises circulating levels to more than 1% in all subjects, these results of the first parenteral administration of rAAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans in a manner similar to that seen in animals. Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. In an open-label dose-escalation study, adult men with severe hemophilia B (F.IX < 1%) due to a missense mutation were injected at multiple intramuscular sites with an rAAV vector. At doses ranging from 2 x 10(11) vector genomes (vg)/kg to 1.8 x 10(12) vg/kg, there was no evidence of local or systemic toxicity up to 40 months after injection. Muscle biopsies of injection sites performed 2 to 10 months after vector administration confirmed gene transfer as evidenced by Southern blot and transgene expression as evidenced by immunohistochemical staining. Pre-existing high-titer antibodies to AAV did not prevent gene transfer or expression. Despite strong evidence for gene transfer and expression, circulating levels of F.IX were in all cases less than 2% and most were less than 1%. Although more extensive transduction of muscle fibers will be required to develop a therapy that reliably raises circulating levels to more than 1% in all subjects, these results of the first parenteral administration of rAAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans in a manner similar to that seen in animals.Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. In an open-label dose-escalation study, adult men with severe hemophilia B (F.IX < 1%) due to a missense mutation were injected at multiple intramuscular sites with an rAAV vector. At doses ranging from 2 x 10(11) vector genomes (vg)/kg to 1.8 x 10(12) vg/kg, there was no evidence of local or systemic toxicity up to 40 months after injection. Muscle biopsies of injection sites performed 2 to 10 months after vector administration confirmed gene transfer as evidenced by Southern blot and transgene expression as evidenced by immunohistochemical staining. Pre-existing high-titer antibodies to AAV did not prevent gene transfer or expression. Despite strong evidence for gene transfer and expression, circulating levels of F.IX were in all cases less than 2% and most were less than 1%. Although more extensive transduction of muscle fibers will be required to develop a therapy that reliably raises circulating levels to more than 1% in all subjects, these results of the first parenteral administration of rAAV demonstrate that administration of AAV vector by the intramuscular route is safe at the doses tested and effects gene transfer and expression in humans in a manner similar to that seen in animals. |
| Author | Thompson, Arthur Leonard, Debra G.B. Johnson, Frederick A. Skarsgard, Erik Kay, Mark A. Flake, Alan W. Scallan, Ciaran High, Katherine A. Chew, Amy J. Herzog, Roland W. Couto, Linda B. Ragni, Margaret V. Ozelo, Margareth McClelland, Alan Larson, Peter J. Glader, Bertil Arruda, Valder R. Manno, Catherine S. Tai, Shing Jen Hutchison, Sylvia |
| Author_xml | – sequence: 1 givenname: Catherine S. surname: Manno fullname: Manno, Catherine S. email: manno@emailchop.edu – sequence: 2 givenname: Amy J. surname: Chew fullname: Chew, Amy J. – sequence: 3 givenname: Sylvia surname: Hutchison fullname: Hutchison, Sylvia – sequence: 4 givenname: Peter J. surname: Larson fullname: Larson, Peter J. – sequence: 5 givenname: Roland W. surname: Herzog fullname: Herzog, Roland W. – sequence: 6 givenname: Valder R. surname: Arruda fullname: Arruda, Valder R. – sequence: 7 givenname: Shing Jen surname: Tai fullname: Tai, Shing Jen – sequence: 8 givenname: Margaret V. surname: Ragni fullname: Ragni, Margaret V. – sequence: 9 givenname: Arthur surname: Thompson fullname: Thompson, Arthur – sequence: 10 givenname: Margareth surname: Ozelo fullname: Ozelo, Margareth – sequence: 11 givenname: Linda B. surname: Couto fullname: Couto, Linda B. – sequence: 12 givenname: Debra G.B. surname: Leonard fullname: Leonard, Debra G.B. – sequence: 13 givenname: Frederick A. surname: Johnson fullname: Johnson, Frederick A. – sequence: 14 givenname: Alan surname: McClelland fullname: McClelland, Alan – sequence: 15 givenname: Ciaran surname: Scallan fullname: Scallan, Ciaran – sequence: 16 givenname: Erik surname: Skarsgard fullname: Skarsgard, Erik – sequence: 17 givenname: Alan W. surname: Flake fullname: Flake, Alan W. – sequence: 18 givenname: Mark A. surname: Kay fullname: Kay, Mark A. – sequence: 19 givenname: Katherine A. surname: High fullname: High, Katherine A. – sequence: 20 givenname: Bertil surname: Glader fullname: Glader, Bertil |
| BackLink | http://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=14699532$$DView record in Pascal Francis https://www.ncbi.nlm.nih.gov/pubmed/12515715$$D View this record in MEDLINE/PubMed |
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| Publisher | Elsevier Inc The Americain Society of Hematology |
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| Snippet | Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for... |
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| SubjectTerms | Adult Aged Amino Acid Substitution Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Applied cell therapy and gene therapy Biological and medical sciences Biopsy Combined Modality Therapy Dependovirus - genetics Factor IX - analysis Factor IX - genetics Factor IX - therapeutic use Feasibility Studies Genetic Therapy Genetic Vectors - administration & dosage Genetic Vectors - therapeutic use Hematologic and hematopoietic diseases Hemophilia B - complications Hemophilia B - genetics Hemophilia B - therapy Hepatitis, Viral, Human - complications HIV Infections - complications Humans Injections, Intramuscular Male Medical sciences Muscle, Skeletal - metabolism Muscle, Skeletal - pathology Mutation, Missense Platelet diseases and coagulopathies Recombinant Fusion Proteins - analysis Safety Transfusions. Complications. Transfusion reactions. Cell and gene therapy |
| Title | AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B |
| URI | https://dx.doi.org/10.1182/blood-2002-10-3296 https://www.ncbi.nlm.nih.gov/pubmed/12515715 https://www.proquest.com/docview/73155930 |
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