Abstract: The present invention provides a compound, which is an inhibitor of phosphoinositide 3-kinase delta or a pharmaceutically acceptable salt and/or solvate thereof, for use in the treatment of an immunobullous skin disease mediated by autoantibodies by oral administration.

Abstract: The present invention provides a pharmaceutical composition which comprises (a) a compound which is an inhibitor of phosphoinositide 3-kinase delta or a pharmaceutically acceptable salt and/or solvate thereof, and (b) a corticosteroid.

Abstract: The present invention provides a method for treating a disorder such as hemophilia. A method of treating hemophilia in a mammal by administering recombinant virus virions comprising a nucleotide sequence having an adenoviral inverted terminal repeat fusion sequence, a packaging signal, a transcriptional control region, and a nucleic acid encoding a therapeutic protein such as FVIII. In addition, the DNA molecule does not encode an adenoviral protein. It is preferred that the virions be administered to the mammal under conditions that result in the expression of the therapeutic protein at a level that provides a therapeutic effect in said mammal.

Abstract: This invention is related to adenoviral (Ad) vectors and their applications in the field of genetic medicine, including gene transfer, gene therapy, and gene vaccination. More specifically, this invention is related to the Ad vectors that carry the minimal cis-element of the Ad genome (mini-Ad vector) and are capable of delivering transgenes and/or heterologous DNA up to 36 kb. The generation and propagation of the mini-Ad vectors require trans-complementation of a packaging-attenuated and replication-defective helper Ad (helper) in an Ad helper cell line.