Search results for "Targeting"
showing 10 items of 186 documents
Disruption of the retinitis pigmentosa 28 gene Fam161a in mice affects photoreceptor ciliary structure and leads to progressive retinal degeneration.
2014
Mutations in the FAM161A gene were previously identified as the cause for autosomal-recessive retinitis pigmentosa 28. To study the effects of Fam161a dysfunction in vivo, we generated gene-trapped Fam161a(GT/GT) mice with a disruption of its C-terminal domain essential for protein-protein interactions. We confirmed the absence of the full-length Fam161a protein in the retina of Fam161a(GT/GT) mice using western blots and showed weak expression of a truncated Fam161a protein by immunohistochemistry. Histological analyses demonstrated that photoreceptor segments were disorganized in young Fam161a(GT/GT) mice and that the outer retina was completely lost at 6 months of age. Reactive microglia…
Rapid, reproducible transduction of select forebrain regions by targeted recombinant virus injection into the neonatal mouse brain
2009
Viral vectors can mediate long-term gene expression in different regions of the brain. Recombinant adeno-associated virus (rAAV) and Lenti virus (LV) have both gained prominence due to their ability to achieve specific transduction of various neuronal populations. Whilst widespread gene delivery has been obtained by targeted injection of rAAV in various brain structures, LV has also been utilized for infection of stem cell populations for cell lineage tracing. Both viral vector systems are most commonly used for gene delivery in mature brains, but the great potential of somatic gene delivery into the neonate brain has not been systematically exploited. Here we provide a systematic guideline…
NANOSTRUCTURED LIPID CARRIERS CONTAINING RILUZOLE AND PHARMACEUTICAL FORMULATIONS CONTAINING SAID PARTICLES
2008
The present invention concerns the preparation, the physical-chemical, technological and pharmaceutical characterization of Riluzole-loaded nanoparticle vectors. The chosen production technology permitted to obtain these systems in a reproducible way, with high yields and low costs. In particular, a novel formulation of Riluzole loaded into lipid nanoparticles was developed which is able to carry to the Central Nervous System a higher amount of drug in comparison with the free drug. In vivo results demonstrated that the administration on rats of these formulation permits to obtain a lower deposit of Riluzole in organs such as liver, spleen, heart and kidneys; the consequence is a reduction …
Metallic Core Nanocarriers for Multiple Cancer Targeting
2014
INNOVATIVE CATIONIC SUPRAMOLECULAR VESICULAR AGGREGATES (SVAs) FOR THE PULMONARY TISSUE SELECTIVE TARGETING
2011
A transmembrane serine residue in the Rot1 protein is essential for yeast cell viability
2014
Polar residues are present in TM (transmembrane) helices and may influence the folding or association of membrane proteins. In the present study, we use an in vivo approach to analyse the functional and structural roles for amino acids in membrane-spanning motifs using the Rot1 (reversal of Tor2 lethality 1) protein as a model. Rot1 is an essential membrane protein in Saccharomyces cerevisiae and it contains a single TM domain. An alanine insertion scanning analysis of this TM helix revealed that the integrity of the central domain is essential for protein function. We identified a critical serine residue inside the helix that plays an essential role in maintaining cell viability in S. cere…
Targeting and membrane insertion into the endoplasmic reticulum membrane of Saccharomyces cerevisiae essential protein Rot1
2010
Rot1 is an essential yeast protein that has been related to cell wall biosynthesis, actin cytoskeleton dynamics and protein folding. Rot1 is an N -glycosylated protein anchored to the nuclear envelope–endoplasmic reticulum (ER) membrane by a transmembrane domain at its C-terminal end. Rot1 is translocated to the ER by a post-translational mechanism. Here, we investigate the protein domain required to target and translocate Rot1 to the ER membrane. We found that several deletions of the N-terminal region of Rot1 prevented neither membrane targeting nor the insertion of this protein. Interestingly, we obtained the same results when different truncated forms in the C-terminal transmembrane dom…
GALACTOSE-DECORATED POLYMERIC CARRIERS FOR HEPATOCYTE-SELECTIVE DRUG TARGETING
2015
In this paper, the current available strategies to realize galactose-decorated nanostructured polymeric systems are summarized. These carriers are designed in order to obtain targeted drug delivery to hepatocytes via galactose (GAL) moieties, i.e., for the treatment of viral hepatitis or liver cancer that are the greater causes of global disability and mortality. Usually, the main followed strategy to obtain galactosylated polymeric carriers is to use galactosylated copolymers. The chemical modifications of preformed polymers with sugar-containing reagents is followed for obtaining lactosaminated human albumin, galactosylated phospholipid-polyaminoacid and polylactide (PLA)- polyaminoacid c…