Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral
gene delivery systems including lipid-based DNA therapeutics offer the opportunity to …

Polymeric nanoparticles-based therapeutics show great promise in the treatment of a wide
range of diseases, due to the flexibility in which their structures can be modified, with …

Improvements in the gene transfer vectors used in therapeutic trials have led to substantial
clinical successes in patients with serious genetic conditions, such as immunodeficiency …

Background Lung delivery of plasmid DNA encoding the CFTR gene complexed with a
cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to …

Nanoscale objects, whether of biologic origin or synthetically created, are being developed
into devices for a variety of bionanotechnology diagnostic and pharmaceutical applications …

Gene therapy finds its niche | Nature Biotechnology Skip to main content Thank you for visiting
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Tissue engineering scaffolds have emerged as a powerful tool within regenerative medicine.
These materials are being designed to create environments that promote regeneration …

Cystic fibrosis (CF) is a monogenic autosomal recessive disorder that affects about 70 000
people worldwide. The clinical manifestations of the disease are caused by defects in the …

Gene therapy covers a broad spectrum of applications, from gene replacement and
knockdown for genetic or acquired diseases such as cancer, to vaccination, each with …

Hepatocellular carcinoma (HCC) develops predominantly in the inflammatory environment
of a cirrhotic liver caused by hepatitis, toxin exposure, or chronic liver disease. A targeted …