The Experts below are selected from a list of 16743 Experts worldwide ranked by ideXlab platform
Isabelle Richard - One of the best experts on this subject based on the ideXlab platform.
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phenotypic correction of α sarcoglycan deficiency by intra arterial injection of a muscle specific serotype 1 raav vector
Molecular Therapy, 2007Co-Authors: Francoise Fougerousse, Marc Bartoli, Jerome Poupiot, Ludovic Arandel, Muriel Durand, Nicolas Guerchet, Evelyne Gicquel, Olivier Danos, Isabelle RichardAbstract:α-Sarcoglycanopathy (limb-girdle muscular dystrophy type 2D, LGMD2D) is a recessive muscular disorder caused by deficiency in α-sarcoglycan, a transMembrane protein part of the dystrophin-associated complex. To date, no treatment exists for this disease. We constructed recombinant pseudotype-1 adeno-associated virus (rAAV) vectors expressing the human α-sarcoglycan cDNA from a ubiquitous or a muscle-specific promoter. Evidence of specific immune response leading to disappearance of the vector was observed with the ubiquitous promoter. In contrast, efficient and sustained transgene expression with correct sarcolemmal localization and without evident toxicity was obtained with the muscle-specific promoter after intra-arterial injection into the limbs of an LGMD2D murine model. Transgene expression resulted in restoration of the sarcoglycan complex, histological improvement, Membrane Stabilization, and correction of pseudohypertrophy. More importantly, α-sarcoglycan transfer produced full rescue of the contractile force deficits and stretch sensibility and led to an increase of the global activity of the animals when both posterior limbs are injected. Our results establish the feasibility for AAV-mediated α-sarcoglycan gene transfer as a therapeutic approach.
Francoise Fougerousse - One of the best experts on this subject based on the ideXlab platform.
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phenotypic correction of α sarcoglycan deficiency by intra arterial injection of a muscle specific serotype 1 raav vector
Molecular Therapy, 2007Co-Authors: Francoise Fougerousse, Marc Bartoli, Jerome Poupiot, Ludovic Arandel, Muriel Durand, Nicolas Guerchet, Evelyne Gicquel, Olivier Danos, Isabelle RichardAbstract:α-Sarcoglycanopathy (limb-girdle muscular dystrophy type 2D, LGMD2D) is a recessive muscular disorder caused by deficiency in α-sarcoglycan, a transMembrane protein part of the dystrophin-associated complex. To date, no treatment exists for this disease. We constructed recombinant pseudotype-1 adeno-associated virus (rAAV) vectors expressing the human α-sarcoglycan cDNA from a ubiquitous or a muscle-specific promoter. Evidence of specific immune response leading to disappearance of the vector was observed with the ubiquitous promoter. In contrast, efficient and sustained transgene expression with correct sarcolemmal localization and without evident toxicity was obtained with the muscle-specific promoter after intra-arterial injection into the limbs of an LGMD2D murine model. Transgene expression resulted in restoration of the sarcoglycan complex, histological improvement, Membrane Stabilization, and correction of pseudohypertrophy. More importantly, α-sarcoglycan transfer produced full rescue of the contractile force deficits and stretch sensibility and led to an increase of the global activity of the animals when both posterior limbs are injected. Our results establish the feasibility for AAV-mediated α-sarcoglycan gene transfer as a therapeutic approach.
M Rajani - One of the best experts on this subject based on the ideXlab platform.
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antioxidant activity of cinchona officinalis stem bark extracts
Oriental Pharmacy and Experimental Medicine, 2003Co-Authors: M N Ravishankara, Padh Harish, M RajaniAbstract:Stem bark of Cinchona sp. (Rubiaceae) is one of the well known drugs for its therapeutic values in traditional as well as modern medicine. Even though a lot of work has been carried out on quinoline alkaloids of Cinchona, its phenolic constituents received very little attention. In the present study, we evaluated antioxidant properties of C. officinalis stem bark methanolic extract and water extract containing phenolic compounds (total phenolics 21.37, 5.18% w/w respectively in the two extracts) in different in vitro and ex vivo models viz., antiradical activity by DPPH reduction, superoxide radical scavenging activity in riboflavin/light/NBT system, nitric oxide radical scavenging activity in sodium nitroprusside/Greiss reagent system and inhibition of lipid peroxidation induced by iron-ADP-ascorbate in liver homogenate and haemolysis of erythrocytes induced by phenylhydrazine in erythrocyte Membrane Stabilization study. Both the extracts exhibited very good antioxidant activity in all the models tested. The phenolic compounds including tannins present in the stem bark seem to offer protection from the oxidative damage.
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evaluation of antioxidant properties of root bark of hemidesmus indicus r br anantmul
Phytomedicine, 2002Co-Authors: M N Ravishankara, Neeta Shrivastava, Harish Padh, M RajaniAbstract:Hemidesmus indicus R. Br. (Asclepiadaceae) is a well known drug in Ayurveda system of medicine. In the present study, antioxidant activity of methanolic extract of H. indicus root bark was evaluated in several in vitro and ex vivo models. Further, preliminary phytochemical analysis and TLC fingerprint profile of the extract was established to characterize the extract which showed antioxidant properties. The in vitro and ex vivo antioxidant potential of root bark of H. indicus was evaluated in different systems viz. radical scavenging activity by DPPH reduction, superoxide radical scavenging activity in riboflavin/light/NBT system, nitric oxide (NO) radical scavenging activity in sodium nitroprusside/Greiss reagent system and inhibition of lipid peroxidation induced by iron-ADP-ascorbate in liver homogenate and phenylhydrazine induced haemolysis in erythrocyte Membrane Stabilization study. The extract was found to have different levels of antioxidant properties in the models tested. In scavenging DPPH and superoxide radicals, its activity was intense (EC50 = 18.87 and 19.9 microg/ml respectively) while in scavenging NO radical, it was moderate. It also inhibited lipid peroxidation of liver homogenate (EC50 = 43.8 microg/ml) and the haemolysis induced by phenylhydrazine (EC50 = 9.74 microg/ml) confirming the Membrane Stabilization activity. The free radical scavenging property may be one of the mechanisms by which this drug is effective in several free radical mediated disease conditions.
Nicolas Guerchet - One of the best experts on this subject based on the ideXlab platform.
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phenotypic correction of α sarcoglycan deficiency by intra arterial injection of a muscle specific serotype 1 raav vector
Molecular Therapy, 2007Co-Authors: Francoise Fougerousse, Marc Bartoli, Jerome Poupiot, Ludovic Arandel, Muriel Durand, Nicolas Guerchet, Evelyne Gicquel, Olivier Danos, Isabelle RichardAbstract:α-Sarcoglycanopathy (limb-girdle muscular dystrophy type 2D, LGMD2D) is a recessive muscular disorder caused by deficiency in α-sarcoglycan, a transMembrane protein part of the dystrophin-associated complex. To date, no treatment exists for this disease. We constructed recombinant pseudotype-1 adeno-associated virus (rAAV) vectors expressing the human α-sarcoglycan cDNA from a ubiquitous or a muscle-specific promoter. Evidence of specific immune response leading to disappearance of the vector was observed with the ubiquitous promoter. In contrast, efficient and sustained transgene expression with correct sarcolemmal localization and without evident toxicity was obtained with the muscle-specific promoter after intra-arterial injection into the limbs of an LGMD2D murine model. Transgene expression resulted in restoration of the sarcoglycan complex, histological improvement, Membrane Stabilization, and correction of pseudohypertrophy. More importantly, α-sarcoglycan transfer produced full rescue of the contractile force deficits and stretch sensibility and led to an increase of the global activity of the animals when both posterior limbs are injected. Our results establish the feasibility for AAV-mediated α-sarcoglycan gene transfer as a therapeutic approach.
Jerome Poupiot - One of the best experts on this subject based on the ideXlab platform.
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phenotypic correction of α sarcoglycan deficiency by intra arterial injection of a muscle specific serotype 1 raav vector
Molecular Therapy, 2007Co-Authors: Francoise Fougerousse, Marc Bartoli, Jerome Poupiot, Ludovic Arandel, Muriel Durand, Nicolas Guerchet, Evelyne Gicquel, Olivier Danos, Isabelle RichardAbstract:α-Sarcoglycanopathy (limb-girdle muscular dystrophy type 2D, LGMD2D) is a recessive muscular disorder caused by deficiency in α-sarcoglycan, a transMembrane protein part of the dystrophin-associated complex. To date, no treatment exists for this disease. We constructed recombinant pseudotype-1 adeno-associated virus (rAAV) vectors expressing the human α-sarcoglycan cDNA from a ubiquitous or a muscle-specific promoter. Evidence of specific immune response leading to disappearance of the vector was observed with the ubiquitous promoter. In contrast, efficient and sustained transgene expression with correct sarcolemmal localization and without evident toxicity was obtained with the muscle-specific promoter after intra-arterial injection into the limbs of an LGMD2D murine model. Transgene expression resulted in restoration of the sarcoglycan complex, histological improvement, Membrane Stabilization, and correction of pseudohypertrophy. More importantly, α-sarcoglycan transfer produced full rescue of the contractile force deficits and stretch sensibility and led to an increase of the global activity of the animals when both posterior limbs are injected. Our results establish the feasibility for AAV-mediated α-sarcoglycan gene transfer as a therapeutic approach.