Search results for "viral vectors"

Citrus biotechnology

2020

Agricultural crops that can better withstand the changing climatic and pathogen landscape have been produced through natural selection throughout the millennia and, in recent years, through the process of human-assisted plant breeding and selection. However, a lack of genetic diversity in many commercially cultivated crops (due to monoculture) has made them more vulnerable to biotic and abiotic stresses (Esquinas-Alcazar, 2005; Keneni et al., 2012). Citrus, belonging to the Rutaceae family, is one of the most important commercial woody fruit crops in the tropical and subtropical areas of the world. The origin of citrus is traced back to parts of tropical and subtropical Southeast Asia (Wu e…

BIOCOMPATIBLE POLYAMINOACID-BASED POLYCATIONS AS NON-VIRAL VECTORS FOR GENE THERAPY OF CYSTIC FIBROSIS.

2009

Non viral colloidal vectors based on polyaminoacids for gene therapy

2008

Treatment of Anderson-Fabry Disease

2020

Fabry disease is an X-linked disorder of glycosphingolipid metabolism that results in progressive accumulation of neutral glycosphingolipids, predominantly globotriaosylsphingosine (Gb3) in lysosomes, as well as other cellular compartments of several tissues, causing multi-organ manifestations (acroparesthesias, hypohidrosis, angiokeratomas, signs and symptoms of cardiac, renal, cerebrovascular involvement). Pathogenic mutations lead to a deficiency of the lysosomal enzyme alpha-galactosidase A (GLA). In the presence of high clinical suspicion, a careful physical examination and specific laboratory tests are required. Finally, the diagnosis of Fabry’s disease is confirmed by the demonstrat…

Well-defined polypeptide-based systems as non-viral vectors for cytosolic delivery

2017

A convenient cytosolic drug delivery constitutes a very powerful tool for the treatment and/or prevention of several relevant human diseases. Along with recent advances in therapeutic technologies based on biomacromolecules (e.g. oligonucleotides or proteins), we also require the development of technologies which improve the transport of therapeutic molecules to the cell of choice. This has led to the emergence of a variety of promising methods over the last 20 years. Despite significant progress, these methods still suffer from several shortcomings including low/variable delivery efficiency, high cytotoxicity, and perhaps most importantly, ineffective endosomal/lysosomal escape. In this co…

Polymeric nanoparticles for siRNA delivery: Production and applications

2017

Gene therapy through the use of siRNA and a polymeric carrier are becoming an efficient therapeutic option to conventional pharmaceutical formulations for the treatment of deadly diseases, such as cancer, pulmonary, ocular and neurodegenerative diseases. However, several considerations regarding the stability, formulation, and efficacy have to be faced up until these systems could be considered to be a marketable pharmaceutical products for to extend siRNA application to clinical practice. This review is focused on the key challenges of siRNA therapeutics, with special attention on the faced obstacles and on the formulation-related difficulties, providing a list of requirements needed for o…