Patent application number | Description | Published |
20100087511 | SPINAL MUSCULAR ATROPHY (SMA) TREATMENT VIA TARGETING OF SMN2 SPLICE SITE INHIBITORY SEQUENCES - The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for “intronic splicing silencer”), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA). | 04-08-2010 |
20110172311 | Compounds For Treating Human Papillomavirus - Methods and compositions for treating human papillomavirus infections are described. | 07-14-2011 |
20120165394 | SPINAL MUSCULAR ATROPHY (SMA) TREATMENT VIA TARGETING OF SMN2 SPLICE SITE INHIBITORY SEQUENCES - The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for “intronic splicing silencer”), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA). | 06-28-2012 |
20130096160 | ARYLTHIAZOLYL PIPERIDINES AND RELATED COMPOUNDS AS MODULATORS OF SURVIVAL MOTOR NEURON (SMN) PROTEIN PRODUCTION - Aryl substituted thiazol-2-yl-piperidines and related compounds useful as modulators of survival motor neuron (SMN) protein production are provided herein. Without being bound to any particular theory it is believed the aryl substituted thiazol-2-yl-piperidines and related compounds provided herein act to increase production of the SMN2 form of survival motor neuron protein. These compounds are useful for treating spinal muscular atrophy. Pharmaceutical compositions containing a carrier and one or more of the aryl substituted thiazol-2-yl-piperidine or related compounds described herein are also provided. Methods of treating spinal muscular atrophy are also provided by this disclosure. | 04-18-2013 |
20140066492 | SPINAL MUSCULAR ATROPHY (SMA) TREATMENT VIA TARGETING OF SMN2 SPLICE SITE INHIBITORY SEQUENCES - The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for “intronic splicing silencer”), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA). | 03-06-2014 |
Patent application number | Description | Published |
20130098330 | CONNECTING ROD SYSTEM FOR AN ENGINE - An internal combustion engine includes a piston, a crankshaft having a journal, and a connecting rod. The connecting rod includes a first end coupled to the piston and a second end coupled to the journal of the crankshaft. The second end of the connecting rod includes a bearing surface configured to interface with the journal, and further includes an open portion extending through the second end from the bearing surface to the exterior of the second end. | 04-25-2013 |
20130125809 | SYSTEM FOR CHECKING OIL LEVEL - An engine includes a crankcase defining an interior volume for containing lubricant, a fill apparatus including a fill passage and a cap removably coupled to the fill passage, the fill passage defining a main conduit through which the lubricant is provided to the interior volume, a visual interface including an indicator movable in response to an amount of the lubricant in the interior volume to indicate different amounts of the lubricant in the interior volume, a float configured to float on a surface of the lubricant, and a float rod coupling the float to the indicator so that movement of the float in response to changes in the amount of the lubricant in the interior volume causes movement of the indicator. | 05-23-2013 |
20140053794 | CENTRIFUGAL FAN - A centrifugal fan for use with an engine includes a hub defining a rotational axis, a plurality of blades arranged about the rotational axis, and a screen coupled to the hub and blades for co-rotation therewith. The hub, the blades, and the screen are integrally formed as a single piece. | 02-27-2014 |