Secure and effective gene delivery system of plasmid DNA coated by polynucleotide [PDF]
, 2015 Polynucleotides are anionic macromolecules which are expected to transfer into the targeted cells through specific uptake mechanisms. So, we developed polynucleotides coating complexes of plasmid DNA (pDNA) and polyethylenimine (PEI) for a secure and ...
Sasaki, Hitoshi +10 more
core +1 more source
Immune Activities of Polycationic Vectors for Gene Delivery
Frontiers in Pharmacology, 2017 Polycationic vectors are used widely in the field of gene delivery, while currently their immune activities in vivo are poorly understood. In this comprehensive review, we aim to present an overview of existing mechanisms of adverse immune responses ...
Xiaotian Zhao +10 more
doaj +1 more source
Supramolecular Systems for Gene and Drug Delivery [PDF]
Pharmaceutics, 2022 Several biomaterial-based supramolecular systems (cyclodextrins [...]
José A. Lebrón +2 more
openaire +6 more sources
Gene Delivery to the Nervous System
Molecular Therapy, 2008 The National Institute of Neurological Disorders and Stroke (NINDS) sponsored a workshop on gene delivery to the nervous system, which took place on 12–13 November 2007 in Washington, DC. The purpose of the workshop was to convene neuroscientists, molecular virologists/vectorologists, and surgical neurologists to assess the state of the art of gene ...
Schubert, Manfred +4 more
openaire +2 more sources
Novel therapeutic approaches for various cancer types using a modified sleeping beauty-based gene delivery system. [PDF]
PLoS ONE, 2014 Successful gene therapy largely depends on the selective introduction of therapeutic genes into the appropriate target cancer cells. One of the most effective and promising approaches for targeting tumor tissue during gene delivery is the use of viral ...
In-Sun Hong, Hwa-Yong Lee, Hyun-Pyo Kim
doaj +1 more source
Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease
Molecular Therapy: Methods & Clinical Development, 2021 Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD).
Naoya Uchida +11 more
doaj +1 more source
Characterization of Ku70(2)-NLS as bipartite nuclear localization sequence for non-viral gene delivery. [PDF]
, 2012 Several barriers have to be overcome in order to achieve gene expression in target cells, e.g. cellular uptake, endosomal release and translocation to the nucleus.
Maucksch, Christof +17 more
core +2 more sources
Freezing Assisted Gene Delivery Combined with Polyampholyte Nanocarriers [PDF]
, 2017 Physical methodologies such as electroporation and the gene-gun technology have been widely used for transfection; however, their applicability is limited because they lead to cell damage and low cell viability. Therefore, to address these limitations we
Tadashi Nakaji-Hirabayashi +9 more
core +1 more source
Fast-track applications : the potential for direct delivery of proteins and nucleic acids to plant cells for the discovery of the gene function. [PDF]
, 2005 In animal systems, several methods exist for the direct delivery of nucleic acids and proteins into cells for functional analysis. Until recently, these methods have not been applied to plant systems.
Roberts, Michael R.
core +5 more sources
Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology [PDF]
, 2013 Spinal muscular atrophy (SMA) is a genetic disease which is characterized by muscle weakness and atrophy. The disease arises from mutations in the survival motor neuron 1 (SMN1) gene causing degeneration of spinal cord motor neurons.
Little, Daniel
core +6 more sources