Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

Abstract: The present invention is directed to compositions and methods for the treatment of hemoglobinopathies.

Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

Abstract: The invention discloses suitable gene and polypeptide targets for the development of new therapeutics to treat, prevent or ameliorate conditions associated with mitochondrial dysfunction. The invention also relates to methods to treat, prevent or ameliorate said conditions and pharmaceutical compositions therefor, as well as to a method to identify compounds with therapeutic usefulness to treat conditions associated with mitochondrial dysfunction.

Abstract: Use of a ERR? agonist Or pharmaceutically acceptable salt thereof, for the prevention, delay of progression or the treatment of diabetes, insulin resistance, metabolic disease/metabolic syndrome, dyslipdemia, obesity, overweight, Neurodegenerative diseases such as Parkinson's disease. Alzheimer's disease, Huntington's disease or improvement of exercise endurance capacity.

Abstract: A method of transferring at least one DNA sequence into cells by transducing the cells, in vivo or ex vivo, with a modified adenovirus. The adenovirus, prior to modification, is of a first serotype. In the modified adenovirus, at least a portion of the fiber, and in particular the head portion, is removed from the adenovirus of the first serotype and replaced with a portion, in particular the head portion, of the fiber of an adenovirus of a second serotype. Such method is useful in transducing cells which may be refractory to the adenovirus of the first serotype, yet include a receptor which binds to the head portion of the fiber of the adenovirus of the second serotype.

Abstract: Provided are adenoviral vectors and the production of such vectors. In particular, fiber shaft modifications for efficient targeting of adenoviral vectors are provided. The fiber shaft modifications can be combined with other modifications, such as fiber knob and/or penton modifications, to produce fully ablated (detargeted) adenoviral vectors. A scale-up method for the propagation of detargeted adenoviral vectors is also provided.

Abstract: Provided are adenoviral vectors and the production of such vectors. In particular, fiber shaft modifications for efficient targeting of adenoviral vectors are provided. The fiber shaft modifications can be combined with other modifications, such as fiber knob and/or penton modifications, to produce fully ablated (detargeted) adenoviral vectors. A scale-up method for the propagation of detargeted adenoviral vectors is also provided.

Abstract: A method of transferring at least one DNA sequence into cells by transducing the cells, in vivo or ex vivo, with a modified adenovirus. The adenovirus, prior to modification, is of a first serotype. In the modified adenovirus, at least a portion of the fiber, and in particular the head portion, is removed from the adenovirus of the first serotype and replaced with a portion, in particular the head portion, of the fiber of an adenovirus of a second serotype. Such method is useful in transducing cells which may be refractory to the adenovirus of the first serotype, yet include a receptor which binds to the head portion of the fiber of the adenovirus of the second serotype.

Abstract: This invention relates to mutated adenoviral fiber proteins and adenovirus particles containing such proteins. It further relates to polynucleotides encoding the proteins and vectors containing polynucleotides. It also relates to methods for making and using the adenoviral particles. With the mutated fiber proteins, the adenovirus particles no longer bind to their natural cellular receptor. They can then be “retargeted” to a specific cell type through the addition of a ligand to the virus capsid, which causes the virus to bind to and infect such cell. Specific fiber mutations are listed, which ablate binding to the natural receptor. Adenovirus particles with certain fiber mutations were found to enhance gene transfer to and expression in liver as compared to viral particles with wild-type fiber.

Abstract: The present invention relates to targeting molecules which are useful to specifically target an adenoviral particle to a desired cell type. These targeting molecules comprise a soluble adenoviral receptor domain, a trimerization domain, and a targeting ligand domain. Further provided are polynucleotides encoding such targeting molecule, expression vectors including such polynucleotides, and methods to target an adenoviral particle to a cell, as well as methods to deliver a heterologous gene selectively to a cell.

Abstract: A method of transferring at least one DNA sequence into cells by transducing the cells, in vivo or ex vivo, with a modified adenovirus. The adenovirus, prior to modification, is of a first serotype. In the modified adenovirus, at least a portion of the fiber, and in particular the head portion, is removed from the adenovirus of the first serotype and replaced with a portion, in particular the head portion, of the fiber of an adenovirus of a second serotype. Such method is useful in transducing cells which may be refractory to the adenovirus of the first serotype, yet include a receptor which binds to the head portion of the fiber of the adenovirus of the second serotype.

Abstract: An adenovirus wherein the adenovirus fiber protein includes a ligand which is specific for a receptor located on a desired cell type. The adenovirus may have at least a portion of the adenovirus fiber protein removed and replaced with a ligand which is specific for a receptor located on a desired cell type, or the adenovirus may include a fusion protein of the adenovirus fiber protein and the ligand. Such an adenovirus may also include a gene(s) encoding a therapeutic agent(s) and may be "targeted" in order to deliver such gene(s) to a desired cell type.

Abstract: An adenovirus wherein the adenovirus fiber protein includes a ligand which is specific for a receptor located on a desired cell type. The adenovirus may have at least a portion of the adenovirus fiber protein removed and replaced with a ligand which is specific for a receptor located on a desired cell type, or the adenovirus may include a fusion protein of the adenovirus fiber protein and the ligand. Such an adenovirus may also include a gene(s) encoding a therapeutic agent(s) and may be "targeted" in order to deliver such gene(s) to a desired cell type.