The Experts below are selected from a list of 16746 Experts worldwide ranked by ideXlab platform
H De Verneuil - One of the best experts on this subject based on the ideXlab platform.
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen...
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen III synthase (UROIIIS), the fourth Enzyme of the heme biosynthetic pathway. It is the most severe porphyria and the treatment is largely symptomatic: gene therapy would represent a great therapeutic improvement. As a step toward the development of an effective gene therapy, we have constructed two retroviral vectors, LUSN and pMFG-US (with and without the selectable marker Neo), containing a full-length human cDNA for UROIIIS. Recombinant retroviruses were obtained by transfection of the LUSN or pMFG-US plasmid into the amphotropic packaging cell line ΨCRIP. For each construct, three different producing clones were selected for their high titer (LUSN) or for their ability to express the message at a high level (pMFG-US). In vitro amplification of genomic DNA from target tissue demonstrated the presence of vecto...
Francois Moreaugaudry - One of the best experts on this subject based on the ideXlab platform.
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen...
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen III synthase (UROIIIS), the fourth Enzyme of the heme biosynthetic pathway. It is the most severe porphyria and the treatment is largely symptomatic: gene therapy would represent a great therapeutic improvement. As a step toward the development of an effective gene therapy, we have constructed two retroviral vectors, LUSN and pMFG-US (with and without the selectable marker Neo), containing a full-length human cDNA for UROIIIS. Recombinant retroviruses were obtained by transfection of the LUSN or pMFG-US plasmid into the amphotropic packaging cell line ΨCRIP. For each construct, three different producing clones were selected for their high titer (LUSN) or for their ability to express the message at a high level (pMFG-US). In vitro amplification of genomic DNA from target tissue demonstrated the presence of vecto...
Frederic Mazurier - One of the best experts on this subject based on the ideXlab platform.
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen...
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen III synthase (UROIIIS), the fourth Enzyme of the heme biosynthetic pathway. It is the most severe porphyria and the treatment is largely symptomatic: gene therapy would represent a great therapeutic improvement. As a step toward the development of an effective gene therapy, we have constructed two retroviral vectors, LUSN and pMFG-US (with and without the selectable marker Neo), containing a full-length human cDNA for UROIIIS. Recombinant retroviruses were obtained by transfection of the LUSN or pMFG-US plasmid into the amphotropic packaging cell line ΨCRIP. For each construct, three different producing clones were selected for their high titer (LUSN) or for their ability to express the message at a high level (pMFG-US). In vitro amplification of genomic DNA from target tissue demonstrated the presence of vecto...
M Bensidhoum - One of the best experts on this subject based on the ideXlab platform.
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen...
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen III synthase (UROIIIS), the fourth Enzyme of the heme biosynthetic pathway. It is the most severe porphyria and the treatment is largely symptomatic: gene therapy would represent a great therapeutic improvement. As a step toward the development of an effective gene therapy, we have constructed two retroviral vectors, LUSN and pMFG-US (with and without the selectable marker Neo), containing a full-length human cDNA for UROIIIS. Recombinant retroviruses were obtained by transfection of the LUSN or pMFG-US plasmid into the amphotropic packaging cell line ΨCRIP. For each construct, three different producing clones were selected for their high titer (LUSN) or for their ability to express the message at a high level (pMFG-US). In vitro amplification of genomic DNA from target tissue demonstrated the presence of vecto...
J Reiffers - One of the best experts on this subject based on the ideXlab platform.
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen...
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correction of the Enzyme Defect in cultured congenital erythropoietic porphyria disease cells by retrovirus mediated gene transfer
Human Gene Therapy, 1995Co-Authors: Francois Moreaugaudry, Cecile Ged, C Barbot, Frederic Mazurier, Jeanmichel Boiron, M Bensidhoum, J Reiffers, H De VerneuilAbstract:ABSTRACT Congenital erythropoietic porphyria (CEP) is a genetic disease characterized by an overproduction and accumulation of porphyrins in bone marrow. The Enzyme Defect concerns uroporphyrinogen III synthase (UROIIIS), the fourth Enzyme of the heme biosynthetic pathway. It is the most severe porphyria and the treatment is largely symptomatic: gene therapy would represent a great therapeutic improvement. As a step toward the development of an effective gene therapy, we have constructed two retroviral vectors, LUSN and pMFG-US (with and without the selectable marker Neo), containing a full-length human cDNA for UROIIIS. Recombinant retroviruses were obtained by transfection of the LUSN or pMFG-US plasmid into the amphotropic packaging cell line ΨCRIP. For each construct, three different producing clones were selected for their high titer (LUSN) or for their ability to express the message at a high level (pMFG-US). In vitro amplification of genomic DNA from target tissue demonstrated the presence of vecto...