The success story of hemophilia care first began in the 1970s, when the availability of
plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided …

Pullulan is a water soluble exo-polysaccharide produced by yeast like fungus
Aureobasidium pullulans. Pullulan structure have a unique characteristic ie co-existence of …

The concept behind gene therapy is simple—by delivering corrective genetic material to the
cells of a patient the symptoms of disease can be alleviated. But seven years after the first …

The art and science of gene therapy has received much attention of late. The tragic death of
18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the …

Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation
factor IX (F. IX). Previously, we established an experimental basis for gene transfer as a …

The efficient delivery of therapeutic genes and appropriate gene expression are the crucial
issues for clinically relevant gene therapy. Viruses are naturally evolved vehicles which …

Branched polyethylenimine (PEI) chains with an average molecular mass of 2 kDa (PEI2)
have been covalently attached to gold nanoparticles (GNPs), and the potency of the …

Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in
experimental animals. Although the vector genomes are found both as extrachromosomes …

Gene replacement therapy is an attractive approach for treatment of genetic disease, but
may be complicated by the risk of a neutralizing immune response to the therapeutic gene …

Epithelial tissues function to protect the organism from physical, chemical, and microbial
damage and are essential for survival. To perform this role, epithelial keratinocytes undergo …