Novel aminoglycosides, represented by Formulae Ia and Ib, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.

The present invention relates to a process for producing a low endotoxin alkali chitosan, and also to a process for producing low endotoxin neutral chitosan, chitosan salt and chitosan derivatives, and to the products of such processes. The process comprises contacting chitosan with an alkali solution to form a mixture and leaving the mixture for at least about 12 hours. The low endotoxin alkali chitosan may be used in the manufacture of other useful chitosan based products.

The present invention provides a pharmaceutical composition for subcutaneous administration comprising a blood factor and a colloidal particle comprising about 0.5 to 20 mole percent of an amphipathic lipid derivatized with a biocompatible hydrophilic polymer, wherein the blood factor is not encapsulated in said colloidal particle.

Methods for prophylactic treatment of hemophilia B are provided in which modified FIX polypeptides, particularly FIXa, are subcutaneously administered daily or less frequently, such as every other day, or every 2, 3 or 4 days. The treatment results in normal coagulation pharmacokinetics and normal levels of FIX, or mild hemophilia B.

Methods for prophylactic treatment of hemophilia B are provided in which modified FIX polypeptides, particularly FIXa, are subcutaneously administered daily or less frequently, such as every other day, or every 2, 3 or 4 days. The treatment results in normal coagulation pharmacokinetics and normal levels of FIX, or mild hemophilia B.

The present invention provides, among other things, methods of delivering mRNA in vivo, including administering to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome such that the administering of the composition results in the expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c:

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or a pharmaceutically acceptable salt thereof.

The present invention provides methods of treating one or more complications of premature birth suffered by premature infants, comprising administering to the premature infant umbilical cord blood stem cells and, optionally, placental stem cells. The present invention also provides methods of combining and administering, and compositions comprising, umbilical cord blood stem cells, particularly autologous cord blood cells, and placental stem cells for the treatment of premature infants.

The present invention relates to the field of chromatography. More closely, the invention relates to a chromatographic method for purification of proteins, such as Factor VIII, von Willebrand factor and Factor IX. The chromatographic method is performed on a matrix comprising an inner porous core and outer porous lid surrounding said core.

The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to optimized polynucleotide sequences encoding Factor IX proteins, vector comprising the same, and methods of using these compositions to treat a bleeding disorder.

The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to optimized polynucleotide sequences encoding Factor IX proteins, vector comprising the same, and methods of using these compositions to treat a bleeding disorder.