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TL;DR: Coronary artery calcification is highly prevalent in the hemodialysis population and is associated with older age, higher BMI, inflammation and reduced trabecular bone volume, and may represent an incomplete self-defensive response to the progression of atherosclerosis in he modialysis patients.
140 citations
TL;DR: Interactions between homeodomain and Olig bHLH proteins evidently regulate neural cell fate acquisition and diversification in the ventral neural tube.
139 citations
TL;DR: For many gene therapy applications, finding ways to counter the immunostimulatory properties of plasmid DNA vectors is an important approach designed to enhance the vector safety profile, thereby increasing its effective therapeutic index.
139 citations
TL;DR: Results indicate that purified rhASM can be activated in vitro by loss of the free thiol on the C-terminal cysteine via chemical modification, dimerization, or deletion of this amino acid residue.
139 citations
TL;DR: This study examines the merits of administering a recombinant adeno-associated viral vector designed to deliver small interfering RNA (siRNA) that targets the degradation of the Htt transcript to lower Htt levels and correct the behavioral, biochemical, and neuropathological deficits shown to be associated with the YAC128 mouse model of HD.
Abstract: Huntington's disease (HD) is a fatal autosomal dominant neurodegenerative disease caused by an increase in the number of polyglutamine residues in the huntingtin (Htt) protein. With the identification of the underlying basis of HD, therapies are being developed that reduce expression of the causative mutant Htt. RNA interference (RNAi) that seeks to selectively reduce the expression of such disease-causing agents is emerging as a potential therapeutic strategy for this and similar disorders. This study examines the merits of administering a recombinant adeno-associated viral (AAV) vector designed to deliver small interfering RNA (siRNA) that targets the degradation of the Htt transcript. The aim was to lower Htt levels and to correct the behavioral, biochemical, and neuropathological deficits shown to be associated with the YAC128 mouse model of HD. Our data demonstrate that AAV-mediated RNAi is effective at transducing greater than 80% of the cells in the striatum and partially reducing the levels (~40%) of both wild-type and mutant Htt in this region. Concomitant with these reductions are significant improvements in behavioral deficits, reduction of striatal Htt aggregates, and partial correction of the aberrant striatal transcriptional profile observed in YAC128 mice. Importantly, a partial reduction of both the mutant and wild-type Htt levels is not associated with any notable overt neurotoxicity. Collectively, these results support the continued development of AAV-mediated RNAi as a therapeutic strategy for HD.
139 citations
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Showing all 3085 results
| Name | H-index | Papers | Citations |
|---|---|---|---|
| George M. Whitesides | 240 | 1739 | 269833 |
| Stephen J. O'Brien | 153 | 1062 | 93025 |
| Robert B. Jackson | 132 | 458 | 91332 |
| Glenn M. Chertow | 128 | 764 | 82401 |
| Jon Clardy | 116 | 983 | 56617 |
| John J. Fung | 115 | 1011 | 52924 |
| Robert B. Colvin | 111 | 556 | 52034 |
| Sergio Giralt | 109 | 1024 | 48513 |
| Paul Saftig | 107 | 356 | 49929 |
| Robert J. Desnick | 102 | 694 | 39698 |
| Robert A. Soslow | 87 | 427 | 29014 |
| Richard J. Roman | 84 | 461 | 23760 |
| Diana W. Bianchi | 81 | 405 | 24554 |
| Paolo Raggi | 80 | 439 | 33332 |
| Helmut G. Rennke | 77 | 256 | 33959 |