The Experts below are selected from a list of 69 Experts worldwide ranked by ideXlab platform
Jordi Gómez - One of the best experts on this subject based on the ideXlab platform.
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Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting.
Journal of General Virology, 2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 5'UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
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Short Communication Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting
2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 59UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
Anna Nadal - One of the best experts on this subject based on the ideXlab platform.
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Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting.
Journal of General Virology, 2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 5'UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
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Short Communication Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting
2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 59UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
Alfred S. Lewin - One of the best experts on this subject based on the ideXlab platform.
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Retinal Dystrophies: Functional Genomics to Gene Therapy: Novartis Foundation Symposium 255 - Range of retinal diseases potentially treatable by AAV-vectored gene Therapy.
Novartis Foundation symposium, 2008Co-Authors: William W. Hauswirth, Matthew M. Lavail, John G. Flannery, B.j. Raisler, Adrian M. Timmers, Kenneth I. Berns, Alfred S. LewinAbstract:Viable strategies for retinal gene Therapy must be designed to cope with the genetic nature of the disease and/or the primary pathologic process responsible for retinal malfunction. For dominant gene defects the aim must be to destroy the presumably toxic gene product, for recessive gene defects the direct approach aims to provide a wild-type copy of the gene to the affected retinal cell type, and for diseases of either complex or unknown genetic origin, more general cell survival strategies that deal with preserving affected retinal cells are often the best and only option. Hence examples of each type of Therapy will be briefly discussed in several animal models, including Ribozyme Therapy for autosomal dominant retinitis pigmentosa in the transgenic P23H opsin rat, beta-PDE gene augmentation Therapy for autosomal recessive retinitis pigmentosa in the rd mouse, glial cell-derived neurotrophic factor (GDNF) gene Therapy for autosomal dominant RP in the transgenic S334ter opsin rat and pigment epithelial cell-derived neurotrophic factor (PEDF) gene Therapy for neovascular retinal disease in rodents. Each employs a recombinant AAV vectored passenger gene controlled by one of several promoters supporting either photoreceptor-specific expression or more general retinal cell expression depending on the therapeutic requirements.
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Ribozyme gene Therapy for autosomal dominant retinal disease.
Clinical chemistry and laboratory medicine, 2000Co-Authors: William W. Hauswirth, Matthew M. Lavail, John G. Flannery, Alfred S. LewinAbstract:Gene delivery to cells of the retina, particularly to photoreceptor cells, has broad potential both for answering basic questions of retinal biology and for more applied therapeutic purposes. The use of Ribozymes as Therapy for autosomal dominant retinal diseases is a promising technique, and the theoretical and practical basis for their use is discussed. The process involves designing and testing Ribozymes first in vitro and then in animal models of retinal disease. Viral vectors based on the nonpathogenic human adeno-associated virus, when coupled with the strong, rod photoreceptor specific opsin promoter, offer an efficient and nontoxic way to deliver and express Ribozymes in photoreceptor cells for long time periods of time. Effective Ribozyme-mediated Therapy also demands careful in vitro analysis of a Ribozyme's ability to efficiently and specifically distinguish between mutant and wild type RNAs. Finally, effective demonstration of Therapy in an animal model requires careful analysis of any rescue effect in the retina using multiple criteria, including biochemical, structural and physiological assays. For this purpose, Ribozyme Therapy in a transgenic rat model of retinitis pigmentosa containing a dominant rod opsin mutation (proline-to-histidine change at position 23) is discussed in detail.
Jaime Guardia - One of the best experts on this subject based on the ideXlab platform.
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Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting.
Journal of General Virology, 2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 5'UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
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Short Communication Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting
2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 59UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
Hugh D. Robertson - One of the best experts on this subject based on the ideXlab platform.
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Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting.
Journal of General Virology, 2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 5'UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
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Short Communication Characterization of the structure and variability of an internal region of hepatitis C virus RNA for M1 RNA guide sequence Ribozyme targeting
2003Co-Authors: Anna Nadal, Hugh D. Robertson, Jaime Guardia, Jordi GómezAbstract:Accessibility to folded RNA and low potential of variation in the target RNA are crucial requirements for Ribozyme Therapy against virus infections. In hepatitis C virus (HCV), the sequence of the 59UTR is conserved but the highly folded RNA structure severely limits the number of accessible sites. To expand investigation of targeting in the HCV genome, we have considered an internal genomic region whose sequence variation has been widely investigated and which has a particularly conserved RNA structure, which makes it accessible to the human RNase P in vitro. We have first mapped the accessibility of the genomic RNA to complementary DNAs within this internal genomic region. We performed a kinetic and thermodynamic study. Accordingly, we have designed and assayed four RNase P M1 RNA guide sequence Ribozymes targeted to the selected sites. Considerations of RNA structural accessibility and sequence variation indicate that several target sites should be defined for simultaneous attack.
