Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.

Succinimide-activated nitroxyl compounds and methods for the synthesis of such compounds are provided. The invention also relates to the use of succinimide-activated nitroxyl compounds to prepare nitroxylated proteins, for example nitroxylated heme proteins (e.g., nitroxylated hemoglobin and nitroxylated myoglobin). The nitroxylated proteins are optionally also conjugated to a polyalkylene oxide (PAO), for example to a polyethylene glycol (PEG). Polynitroxylated heme proteins are useful as oxygen therapeutic agents (OTAs). The invention further relates to pharmaceutical compositions of the nitroxylated proteins and methods for the use of nitroxylated proteins in the treatment of various conditions.

Succinimide-activated nitroxyl compounds and methods for the synthesis of such compounds are provided. The invention also relates to the use of succinimide-activated nitroxyl compounds to prepare nitroxylated proteins, for example nitroxylated heme proteins (e.g., nitroxylated hemoglobin and nitroxylated myoglobin). The nitroxylated proteins are optionally also conjugated to a polyalkylene oxide (PAO), for example to a polyethylene glycol (PEG). Polynitroxylated heme proteins are useful as oxygen therapeutic agents (OTAs). The invention further relates to pharmaceutical compositions of the nitroxylated proteins and methods for the use of nitroxylated proteins in the treatment of various conditions.

The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human β-globin protein or a human γ-globin protein, which is oriented from 5′ to 3′ relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human β-globin protein or a human γ-globin protein, which is oriented from 5′ to 3′ relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

A method for encapsulating a compound of interest in a matrix may include preparing a first aqueous solution including the compound of interest and a gelating agent. An oil or oil mixture and a second aqueous solution having a gelation inducing agent are prepared. The first aqueous solution is emulsified in the oil or oil mixture to form an emulsion of aqueous solution droplets in oil. That emulsion is emulsified in the second aqueous solution. The gelation inducing agent from the second aqueous solution is diffused through the oil towards the interface of the oil and the first aqueous solution. Gelation of the gelating agent at the interface forms the matrix, wherein particles are obtained including the matrix composed of the gelified gelating agent and encapsulating the compound of interest.

A method for encapsulating a compound of interest in a matrix may include preparing a first aqueous solution including the compound of interest and a gelating agent. An oil or oil mixture and a second aqueous solution having a gelation inducing agent are prepared. The first aqueous solution is emulsified in the oil or oil mixture to form an emulsion of aqueous solution droplets in oil. That emulsion is emulsified in the second aqueous solution. The gelation inducing agent from the second aqueous solution is diffused through the oil towards the interface of the oil and the first aqueous solution. Gelation of the gelating agent at the interface forms the matrix, wherein particles are obtained including the matrix composed of the gelified gelating agent and encapsulating the compound of interest.

The present invention relates to an isolated cellular targeted delivery system comprising a CD45.sup.+ leukocyte cell comprising within said cell a complex of one or more iron binding proteins and an active ingredient as well as methods for producing such isolated cellular targeted delivery system and uses of such system for therapy, in particular for therapy of cancer.

The present invention relates to an isolated cellular targeted delivery system comprising a CD45+ leukocyte cell comprising within said cell a complex of one or more iron binding proteins and an active pharmaceutically active substance and/or label as well as methods for producing such isolated cellular targeted delivery system and uses of such system for prophylaxis, therapy, diagnosis or theragnosis, in particular for prophylactic or therapeutic vaccination, therapy of cancer, particularly metastatic cancer or inflammatory diseases.

The present invention refers to an oxygen carrier, preferably hemoglobin or myoglobin, for use in the treatment of an unpleasant feeling of a body surface due to a disease or a disorder.