A5058310447- RNA Interference and Gene Delivery
- Advanced biosensing and bioanalysis techniques
- Neonatal Respiratory Health Research
- Nanoparticle-Based Drug Delivery
- Cystic Fibrosis Research Advances
- Dendrimers and Hyperbranched Polymers
- DNA and Nucleic Acid Chemistry
- Neurogenetic and Muscular Disorders Research
- MicroRNA in disease regulation
- Virus-based gene therapy research
- Advanced Drug Delivery Systems
- Immunotherapy and Immune Responses
- Infant Nutrition and Health
- Silk-based biomaterials and applications
- CRISPR and Genetic Engineering
- Skin and Cellular Biology Research
University College Dublin
2021-2024
University of Castilla-La Mancha
2017-2022
Instituto de Salud Carlos III
2017-2022
Centro de Investigación Biomédica en Red
2022
Biomedical Research Networking Center on Neurodegenerative Diseases
2017-2020
Abstract Recent advances in molecular biology have led to the CRISPR revolution, but lack of an efficient and safe delivery system into cells tissues continues hinder clinical translation approaches. Polymeric vectors offer attractive alternative viruses as due their large packaging capacity safety profile. In this paper, we demonstrated potential use a highly branched poly( β -amino ester) polymer, HPAE-EB, enable genomic editing via CRISPRCas9-targeted excision exon 80 COL7A1 gene, through...
Herein, we present the first evaluation of cationic dendrimers based on 2,2-bis(methylol)propionic acid (bis-MPA) as nonviral vectors for transfection short interfering RNA (siRNA) in cell cultures. The study encompassed generation one to four (G1⁻G4), modified bear 6⁻48 amino end-groups, where G2⁻G4 proved be capable siRNA complexation and protection against RNase-mediated degradation. were nontoxic astrocytes, glioma (C6), glioblastoma (U87), while G3 G4 exhibited concentration dependent...
Significance Inflammation is a relevant part of the physiological response immune system to fight infectious diseases. However, excessive inflammation can also participate in pathogenesis several To open new avenues treat negative aspects inflammation, we have used biocompatible nanoparticles, neutrally charged G3-G4 phosphorus dendrimers, which are not toxic and good solubility chemical stability aqueous solutions. These nanoparticles very efficient, vitro vivo, tackle inflammatory produced...
Lung cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, leading to dysfunctional CFTR protein. Gene therapy offers promise for treatment of lung CF. However, development and clinical application gene have long been hampered absence safe highly efficient delivery vectors. In this work, novel polymer-based replacement approach was developed. A series poly (β-amino esters) (PAEs) with various topological structures...
Small interfering ribonucleic acid (siRNA) has the potential to revolutionize therapeutics since it can knockdown very efficiently target protein. It is starting be widely used interfere with cell infection by HIV. However, naked siRNAs are unable get into cell, requiring use of carriers protect them from degradation and transporting across membrane. There no information about which most efficient endocytosis route for high siRNA transfection efficiency. One promising deliver cyclodextrin...
To study the structural requirements that a cyclooligosaccharide-based nanoparticle must fulfill to be an efficient siRNA transfection vector.siRNA protection from degradation by RNAses, efficiency and thermodynamic parameters of nanoparticle/siRNA interactions were studied on pairs amphiphilic molecules using biochemical techniques molecular dynamics.The lower solvent accessible surface area in presence nanoparticle, higher RNAse-mediated corresponding nanocomplex; moderate binding energy...
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare autosomal inherited skin disorder caused by mutations in the COL7A1 gene that encodes type VII collagen (C7). The development of an efficient replacement strategy for RDEB mainly hindered lack vectors able to encapsulate and transfect large cDNA size this gene. To address problem, our group has opted use polymeric-based non-viral delivery systems minicircle DNA. With approach, safety improved avoiding usage viruses, absence...
Synthetic double-stranded small interfering RNAs (siRNAs) mimic interference (RNAi) and can bind target mRNAs with a high degree of specificity, leading to selective knockdown the proteins they encode. However, siRNAs are very labile must be both protected transported by nanoparticles efficiently delivered into cells. In this work, we used Janus-type polycationic amphiphilic β-cyclodextrin derivative transfect targeting encoding mitogen-activated protein kinase (p42-MAPK) or Ras homolog...