Topics

PubMed Journals Articles About "Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative" RSS

16:19 EDT 17th October 2019 | BioPortfolio

Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative articles that have been published worldwide.

More Information about "Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative" on BioPortfolio

We have published hundreds of Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative news stories on BioPortfolio along with dozens of Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative Clinical Trials and PubMed Articles about Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative Companies in our database. You can also find out about relevant Prevail Licenses REGENXBIO AAV9 Gene Delivery Technology Neurodegenerative Drugs and Medications on this site too.

Showing "Prevail licenses REGENXBIO AAV9 gene delivery technology neurodegenerative" PubMed Articles 1–25 of 21,000+

Design, Preparation, and Characterization of Peptide-Based Nanocarrier for Gene Delivery.

The delivery of nucleic acid to eukaryotic cells is challenging due to presence of various intra- and extracellular barriers and need to delivery carriers. However, current gene delivery carriers, including PLL, PEI, and liposome, suffer from nanocarrier associate toxicity, low efficiency and polydispersity, and non-biodegradability. Here we describe our strategy for developing safe, efficacious, and monodisperse peptide-based carrier for gene delivery. We explain the use of genetic engineering technology f...


Chitosan for gene delivery: Methods for improvement and applications.

Gene therapy is a promising strategy for treating challenging diseases. The successful delivery of genes is a critical step for gene therapy. However, concerns about immunogenicity and toxicity are the main obstacles against the widespread use of effective viral systems. Therefore, nonviral vectors are regarded as good alternatives to viral vectors. Chitosan is a natural cationic polysaccharide that could be used to create nonviral gene delivery vectors. Various methods have been developed to improve the pr...

Pre-existing antibodies to candidate gene therapy vectors (adeno-associated vector serotypes) in domestic cats.

Adeno-associated virus (AAV) vectors represent promising candidates for gene therapy; however, pre-existing neutralizing antibodies (NAb) may reduce AAV vector delivery efficiency. In this study, the presence of AAV NAb was investigated in cats, which serve as a larger and outbred animal model for the prediction of gene therapy outcomes in humans but also in cats.Serum/plasma samples from 230 client-owned Swiss cats and 20 specified pathogen-free cats were investigated for NAb to AAV1, AAV2, AAV5, AAV6, AAV...


CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model.

Huntington's disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin (HTT) gene, which results in the production of a mutant protein that forms inclusions and selectively destroys neurons in the striatum and other adjacent structures. The RNA-guided Cas9 endonuclease from CRISPR-Cas9 systems is a versatile technology for inducing DNA double-strand breaks that can stimulate the introduction of frame...

Cas9 Ribonucleoprotein Complex Delivery: Methods and Applications for Neuroinflammation.

The CRISPR/Cas9 system is a revolutionary gene editing technology that combines simplicity of use and efficiency of mutagenesis. As this technology progresses toward human therapies, valid concerns including off-target mutations and immunogenicity must be addressed. One approach to address these issues is to minimize the presence of the CRISPR/Cas9 components by maintaining a tighter temporal control of Cas9 endonuclease and reducing the time period of activity. This has been achieved to some degree by deli...

Gene Delivery Using Lipoplexes and Polyplexes: Principles, Limitations and Solutions.

Gene therapy has attracted considerable attention for the treatment of genetic and acquired diseases. Successful gene therapy occurs when the therapeutic genes penetrate targeted cells and become available to the intracellular active site. Currently, a promising approach in gene delivery is the use of nonviral gene delivery vectors that lack immunogenicity but have low toxicity and potential tissue specificity. The widely used, existing nonviral gene vectors are cationic lipids and polymers that can pass ac...

Intracerebral Delivery in Complex 3-D Arrays: The Intracerebral Microinjection Instrument (IMI).

This video article describes and illustrates the function and application of the Intracerebral Microinjection Instrument (IMI). This newly developed technology allows delivery of therapeutics within the human brain in complex three-dimensional arrays using a single pass or minimal overlying penetrations through brain tissue. The IMI utilizes a delivery microcannula with a reduced diameter that minimizes local trauma and is capable of delivering precise volumes of therapeutics to discrete brain substructures...

Gene delivery by peptide-assisted transport.

The diverse amino acid chemistries and secondary structures in peptides provide 'minimalist' mimics of motifs in proteins and offer many ideal properties for targeted delivery approaches. Several non-viral vectors (polymers and lipids) have been studied for their potential applications in gene delivery. However, non-specific uptake, lack of targeting, inability to escape endosomes, and inefficient nuclear delivery limit their application. Peptide-assisted trafficking of non-viral vectors can potentially ove...

The Importance of Patients' Perceptions of Technology: Reminders for Nursing Care Delivery.

This study investigates hospitalized patients' perceptions of nurses' use of technology in care delivery and provides an overview of the professional debate regarding high-touch versus high-technology nursing practice.

Combination therapy and co-delivery strategies to optimize treatment of posterior segment neurodegenerative diseases.

Neurodegenerative diseases affecting the posterior segment of the eye are one of the major causes of irreversible blindness worldwide. The pathogenesis of these retinal pathologies is characterized by a multifactorial etiology, involving the complex interaction of different apoptotic mechanisms, suggesting that effective treatments will require a multimodal approach. Thus, combination therapy based on the potential synergistic activities of drugs with different mechanisms of action is currently receiving co...

Regulatory roles of the miR-200 family in neurodegenerative diseases.

Neurodegenerative diseases are chronic and progressive disorders which are not effectively treated through adopting conventional therapies. For this unmet medical need, alternative therapeutic methods including gene-based therapies are emphasized. MicroRNAs (miRNAs) are small non-coding RNAs which can regulate gene expression at the post-transcriptional level. In recent years, dysregulated miRNAs have been indicated to be implicated in the occurrence and development of neurodegenerative diseases. They are i...

Organic/inorganic nanohybrids as multifunctional gene delivery systems.

In this review, we summarize the rational design and versatile applications of organic/inorganic hybrid gene carriers as multifunctional delivery systems.

Functionalized Liposomal Nanoparticles for Efficient Gene Delivery System to Neuronal Cell Transfection.

Liposome based delivery systems provide a promising strategy for treatment of neurodegenerative diseases. A rational design of brain-targeted liposomes can support the development of more efficient treatments with drugs and gene materials. Here, we characterized surface modified liposomes with transferrin (Tf) protein and penetratin (Pen), a cell-penetrating peptide, for efficient and targeted gene delivery to brain cells. PenTf-liposomes efficiently encapsulated plasmid DNA, protected them against enzymati...

Reactive oxygen species-responsive drug delivery systems for the treatment of neurodegenerative diseases.

Neurodegenerative diseases and disorders seriously impact memory and cognition and can become life-threatening. Current medical techniques attempt to combat these detrimental effects mainly through the administration of neuromedicine. However, drug efficacy is limited by rapid dispersal of the drugs to off-target sites while the site of administration is prone to overdose. Many neuropathological conditions are accompanied by excessive reactive oxygen species (ROS) due to the inflammatory response. According...

Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.

Gene therapy approaches are being deployed to treat recessive genetic disorders by restoring the expression of mutated genes. However, the feasibility of these approaches for dominantly-inherited diseases-where treatment may require reduction in the expression of a toxic mutant protein resulting from a gain-of-function (GoF) allele-is unclear. Here we show the efficacy of allele-specific RNAi as a potential therapeutic for Charcot-Marie-Tooth type 2D (CMT2D), caused by dominant mutations in glycyl tRNA-synt...

Co-delivery of Plasmid and Curcumin with Mesoporous Silica Nanoparticles for Promoting Neurite Outgrowth.

Reactive oxygen species (ROS)-induced oxidative stress leads to neuron damage is involved in the pathogenesis of chronic inflammation in neurodegenerative diseases (NDs), such as Alzheimer's (AD), Parkinson's (PD) and amyotrophic lateral sclerosis (ALS). Researchers, therefore, are looking for anti-inflammatory drugs and gene therapy approaches to slow down or even prevent neurological disorders. Combining therapeutics have shown the synergistic effect in the treatment of human diseases. Many nanocarriers c...

DNA tetrahedron-based nanogels for siRNA delivery and gene silencing.

DNA tetrahedra are employed as building blocks to construct a novel DNA-based nanogel for intracellular siRNA delivery. With a compact architecture that embeds functional siRNA inside, the self-assembled nanogel can efficiently enter cells and knock down the target gene expression, making them potential noncationic vectors for functional nucleic acid delivery.

An effective and biocompatible polyethylenimine based vaginal suppository for gene delivery.

Gene therapy targeted human papillomavirus (HPV) is a promising treatment for cervical cancer, and the key for clinical application depends on an effective gene delivery method. Our aim was to formulate a new pharmaceutical formula for appropriate gene delivery intravaginally. For the first time, we here developed a new polyethylenimine (PEI) based vaginal suppository. The sectional immunofluorescence results confirmed the delivery efficacy both in vivo and in vitro. The quenching fluorescence and decreased...

Lipid nanoparticles for delivery of therapeutic RNA oligonucleotides.

Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNP). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and s...

Challenges of Gene Delivery to the Central Nervous System and the Growing use of Biomaterial Vectors.

Gene therapy is a promising form of treatment for those suffering from neurological disorders or central nervous system (CNS) injury, however, obstacles remain that limit its translational potential. The CNS is protected by the blood brain barrier, and this barrier blocks genes from traversing into the CNS if administered outside of the CNS. Viral and non-viral gene delivery vehicles, commonly referred to as vectors, are modified to enhance delivery efficiency to target locations in the CNS. Still, there ar...

Layered Double Hydroxide Nanoparticles for Efficient Gene Delivery for Cancer Treatment.

The use of cationic polymer based gene delivery vectors has several limitations such as low transfection efficiency, high toxicity, and inactivation by serum. The present work provides an inorganic based nanocarrier for efficient gene delivery and a method for preparing the same through a facile coprecipitation technique. The vehicle showed high loading capacity of DNA and can release the loaded DNA in controlled pH responsive manner. The developed gene delivery vehicle offers remarkable protection against ...

Amelioration of the nigrostriatal pathway facilitated by ultrasound-mediated neurotrophic delivery in early Parkinson's disease.

The blood-brain barrier (BBB) prevents most drugs from gaining access to the brain parenchyma, which is a recognized impediment to the treatment of neurodegenerative disorders like Parkinson's disease (PD). Focused ultrasound (FUS), in conjunction with systemically administered microbubbles, opens the BBB locally, reversibly and non-invasively. Herein, we show that neither FUS applied over both the striatum and the ventral midbrain, without neurotrophic factors, nor intravenous administration of neurotrophi...

Non-viral gene delivery for cancer immunotherapy.

The past decades have witnessed the revolutionizing development in cancer immunotherapies, which boost cancer-specific immune responses for the long-term tumor regression. However, immunotherapy still has limitations, including off-target side effects, long processing times, and limited patient responses. These disadvantages of current immunotherapy are being addressed by improving our understanding of the immune system, as well as by establishing combinational approaches. Advanced biomaterials and gene del...

Non-viral nucleic acid delivery to central nervous system and brain tumor.

Gene therapy is a rapidly emerging remedial route for many serious incurable diseases, such as central nervous system (CNS) diseases. Currently, nucleic acid medicines including DNAs encoding therapeutic or destructive proteins, siRNAs or miRNAs, have been successfully delivered to the CNS with gene delivery vectors via various routes of administration and subsequently exhibited remarkable therapeutic efficiency. Among these vectors, non-viral vectors are favorable for delivering genes into CNS due to many ...

Versatile and multifaceted CRISPR/Cas gene editing tool for plant research.

The ability to create desirable gene variants through targeted changes offers tremendous opportunities for the advancement of basic and applied plant research. Gene editing technologies have opened new avenues to perform such precise gene modifications in diverse biological systems. These technologies use sequence-specific nucleases, such as homing endonucleases, zinc-finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRI...


Quick Search