Patent application number | Description | Published |
20110070220 | Immunomodulating gene therapy - The present invention relates, in general, to Pompe disease and, in particular, to a methods of treating Pompe disease and to compounds/constructs suitable for use in such methods. | 03-24-2011 |
20110294193 | VIRAL VECTORS AND METHODS FOR PRODUCING AND USING THE SAME - A recombinant hybrid virus, including: (a) a deleted adenovirus vector genome comprising the adenovirus 5′ and 3′ cis-elements for viral replication and encapsidation, and further comprising a deletion in an adenovirus genomic region selected from the group consisting of: (i) the polymerase region, wherein said deletion essentially prevents the expression of a functional polymerase protein from said deleted region and said hybrid virus does not otherwise express a functional polymerase protein, (ii) the preterminal protein region, wherein said deletion essentially prevents the expression of a functional preterminal protein from said deleted region, and said hybrid virus does not otherwise express a functional preterminal protein, and (iii) both the regions of (i) and (ii); and (b) a recombinant adeno-associated virus (AAV) vector genome flanked by the adenovirus vector genome sequences of (a), said recombinant AAV vector genome comprising (i) AAV 5′ and 3′ inverted terminal repeats, (ii) an AAV packaging sequence, and (iii) a heterologous nucleic acid sequence, wherein said heterologous nucleic acid sequence is flanked by the 5′ and 3′ AAV inverted terminal repeats of (i). Methods of making and using the recombinant hybrid virus are also disclosed. | 12-01-2011 |
20120082653 | METHODS OF LYSOSOMAL STORAGE DISEASE THERAPY - Methods of treating a lysosomal storage disorder and methods of increasing cellular uptake of a lysosomal enzyme using β2 agonists or therapeutic agents that increase expression of receptors for a lysosomal enzyme. | 04-05-2012 |
20120276072 | CONSTRUCTS FOR EXPRESSING LYSOSOMAL POLYPEPTIDES - Provided are isolated nucleic acids for expressing lysosomal polypeptides such as lysosomal acid α-glucosidase (GAA) and vectors comprising the same. The invention provides an isolated nucleic acid encoding a chimeric polypeptide comprising a secretory signal sequence operably linked to a lysosomal polypeptide. The invention also provides an isolated nucleic acid comprising a coding region encoding a GAA and a GAA 3′ untranslated region (UTR), wherein the GAA 3′ UTR comprises a deletion therein. The invention further provides an isolated nucleic acid comprising a coding region encoding a GAA and a 3′ UTR wherein the 3′ UTR is less than about 200 nucleotides in length and comprises a segment that is heterologous to the GAA coding region. Also provided are methods of making and using delivery vectors encoding lysosomal polypeptides to produce the lysosomal polypeptide to treat subjects afflicted with a deficiency in the lysosomal polypeptide. | 11-01-2012 |
20130052189 | METHOD OF TREATING PATIENTS UNDERGOING PROTEIN REPLACEMENT THERAPY, GENE REPLACEMENT THERAPY, OR OTHER THERAPEUTIC MODALITIES - The present invention relates, in general, to a method of treating patients undergoing enzyme replacement therapy (ERT) or other therapy involving the administration of a proteinaceous therapeutic agent as well gene replacement therapy with non-viral or viral vectors, or other therapeutic modality or modalities, used alone or in combination, which involve the administration of exogenous substances for potential therapeutic benefit, including, but not limited to DNA vaccines, siRNA, splice-site switching oligomers (SSOs) as well as RNA-based nanoparticles (RNPs) and nanovaccines. The invention further relates to compounds and compositions suitable for use in such methods. | 02-28-2013 |
20150037329 | METHOD OF REDUCING TITERS OF ANTIBODIES SPECIFIC FOR A THERAPEUTIC AGENT - The present invention relates, in general, to a method of treating patients undergoing enzyme replacement therapy (ERT) or other therapy involving the administration of a proteinaceous therapeutic agent as well gene replacement therapy with non-viral or viral vectors, or other therapeutic modality or modalities, used alone or in combination, which involve the administration of exogenous substances for potential therapeutic benefit, including, but not limited to DNA vaccines, siRNA, splice-site switching oligomers (SSOs) as well as RNA-based nanoparticles (RNPs) and nanovaccines. The invention further relates to compounds and compositions suitable for use in such methods. | 02-05-2015 |
Patent application number | Description | Published |
20140137975 | Plasticized, Conductive Polyoxymethylene for Fuel Applications - Polymer compositions containing a plasticized, impact modified polyoxymethylene composition that is compounded with a conductive filler composition are disclosed that are flexible while also exhibiting electrostatic dissipative (ESD) capabilities. The polymer compositions can be formed into shaped articles such as tubes, hoses, etc. for use in fuel transfer systems. The plasticized, impact modified polyoxymethylene composition includes a functionalized polyoxymethylene, a plasticizer, and an impact modifier. Meanwhile, the conductive filler composition includes a conductive filler and a high flow polyoxymethylene. The compositions can be compounded into a pellet product which can then be formed into a shaped article that complies with the ESD capabilities required by SAE J1645. It is also to be understood that the polymer composition components can be blended and formed into a pellet product in a single step that can be used to produce a shaped article that complies with the ESD capabilities required by SAE J1645. | 05-22-2014 |
20140142228 | Plasticized, Conductive Polyoxymethylene for Fuel Applications - Polymer compositions containing a plasticized, impact modified polyoxymethylene composition that is compounded with a conductive filler composition are disclosed that are flexible while also exhibiting electrostatic dissipative (ESD) capabilities. The polymer compositions can be formed into shaped articles such as tubes, hoses, etc. for use in fuel transfer systems. The plasticized, impact modified polyoxymethylene composition includes a functionalized polyoxymethylene, a plasticizer, and an impact modifier. Meanwhile, the conductive filler composition includes a conductive filler and a high flow polyoxymethylene. The compositions can be compounded into a pellet product which can then be formed into a shaped article that complies with the ESD capabilities required by SAE J1645. It is also to be understood that the polymer composition components can be blended and formed into a pellet product in a single step that can be used to produce a shaped article that complies with the ESD capabilities required by SAE J1645. | 05-22-2014 |
20140166940 | Laser-Weldable Electrostatically Dissipative Polyoxymethylene Based on Stainless Steel Fibers - Polymer compositions containing a polyoxymethylene base polymer that is compounded with a conductive filler and pigment carbon black are disclosed that exhibit electrostatic dissipative (ESD) capabilities while also being laser weldable. The polymer composition can be formed into shaped or molded articles such as filters, flanges, connectors, etc. for use in fuel systems where conductivity, ductility, weldability, and resistance to aggressive fuels are required. The compositions can be compounded into a pellet product which can then be formed into a shaped article that complies with the ESD capabilities required by SAE J1645. At the same time, parts produced from the compositions can absorb laser energy sufficiently to form a high quality welded interface with parts produced from other compositions that are transparent to laser radiation and cannot absorb laser energy sufficiently. | 06-19-2014 |
20140186562 | Conductive Polyoxymethylene Based on Stainless Steel Fibers - Polymer compositions containing a polyoxymethylene base polymer that is compounded with a conductive filler, a polyalkylene glycol, and a metal oxide are disclosed that exhibit electrostatic dissipative (ESD) capabilities while also being resistant to degradation upon exposure to aggressive fuels. For instance, the polymer compositions can have a volume resistivity of from about 1 Ohm-cm to about 200 Ohm-cm and a surface resistivity of about 10 | 07-03-2014 |