Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
22
pubmed:dateCreated
1999-12-10
pubmed:abstractText
We are interested in using recombinant adeno-associated viral vectors in the treatment of hemophilia A. Because of the size constraints of recombinant adeno-associated viral vectors, we delivered the heavy and light chains of the human factor 8 (hFVIII) cDNA independently by using two separate vectors. Recombinant AAV vectors were constructed that utilized the human elongation factor 1alpha promoter, a human growth factor polyadenylation signal, and the cDNA sequences encoding either the heavy or light chain of hFVIII. Portal vein injections of each vector alone, a combination of both vectors, or a hFIX control vector were performed in C57BL/6 mice. An ELISA specific for the light chain of hFVIII demonstrated very high levels (2-10 microgram/ml) of protein expression in animals injected with the light chain vector alone or with both vectors. We utilized a chromogenic assay in combination with an antibody specific to hFVIII to determine the amount of biologically active hFVIII in mouse plasma. In animals injected with both the heavy and light chain vectors, greater than physiological levels (200-400 ng/ml) of biologically active hFVIII were produced. This suggests that coexpression of the heavy and light chains of hFVIII may be a feasible approach for treatment of hemophilia A.
pubmed:commentsCorrections
http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-1902833, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-1939217, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-2210382, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-2564392, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-3017981, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-3082357, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-3123224, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-3129422, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-3129993, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-6251468, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-6438527, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-7537539, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-7647782, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-7862626, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-8018925, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-81838, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-8234237, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-8639836, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-8916949, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-8934224, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9159155, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9183307, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9207793, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9558383, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9616156, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9813665, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9883840, http://linkedlifedata.com/resource/pubmed/commentcorrection/10535990-9883841
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Oct
pubmed:issn
0027-8424
pubmed:author
pubmed:issnType
Print
pubmed:day
26
pubmed:volume
96
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
12725-30
pubmed:dateRevised
2009-11-18
pubmed:meshHeading
pubmed:year
1999
pubmed:articleTitle
Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein.
pubmed:affiliation
Avigen Inc., 1201 Harbor Bay Parkway, Alameda, CA 94502, USA. lcouto@avigen.com
pubmed:publicationType
Journal Article