viral vector Articles
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Viral Vector Characterization: Why and How
Why need viral vector characterization? Cell and gene therapies for various rare diseases are currently undergoing clinical trials worldwide. The rapid development in this field has led to an increase in regulatory scrutiny and product characterization requirements, as well as a bottleneck in viral vector supply. The manufacturing processes and analytical tools for gene therapy viral vectors need ...
By Protheragen
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DNA nanoparticles for gene delivery to cells and tissue
The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. The major aim of gene therapy is to effectively deliver the genetic materials into cells, genetically modifying and repairing cell functions, which may induce therapeutic healing of disease conditions. This ...
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Development of Gene Therapy Viral Vectors for Rare Diseases
In many cases, gene therapy requires a vector to deliver the gene therapy drug to the target cell. Viral vectors have been one of the most widely studied vectors due to their outstanding transduction efficiency and other significant advantages. Viral vector-based gene therapy has now achieved good clinical results. More than a dozen viral gene therapy products have been approved for the treatment ...
By Protheragen
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Plasmid DNA, mRNA and Viral Vectors – the Building Blocks of Cell and Gene Therapy Processing
The growing pipeline of cell and gene therapy therapies has led to an increase in demand for gene delivery vehicles such as plasmid DNA (pDNA), messenger RNA (mRNA) and viral vectors. They can be used as stand-alone treatments or can be used as starting materials required for further processing of cell and gene therapeutics. When used as starting materials, they have been shown to have a ...
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Introduction to AAV as a Gene Therapy Vector, Part 2
What are AAVs? and what makes them good candidates for gene therapy? Introduction In the first part of our three part series introducing AAV as a gene therapy vector, we talked about basic AAV vector biology. In this post, we’re going to take a step back to answer the question of “Why AAV?” and look at some opportunities in the AAV engineering space. Why AAV? Viral vectors ...
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The novel suspension quail cell line CCX.E10 is an ideal cell substrate for NDV virus and vector production
Newcastle disease is a highly infectious viral disease of the avian species. The causative agent is Newcastle disease virus (NDV), which belongs to the family of Paramyxoviridae. Many commercial vaccines, both live and inactivated, are available on the market and protect from NDV infection and disease. In addition, viral vectors based on Newcastle disease virus have been previously ...
By Nuvonis
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Research And Development of Infectious Disease Vaccines
As of January 1, 2023, global vaccine development includes a total of 966 vaccine candidates, of which 23% (220) are traditional inactivated or attenuated vaccines. Advances in molecular technology have facilitated the development of other platforms, such as recombinant protein vaccines, nucleic acid vaccines, and viral vector vaccines, which have further diversified global vaccine development. ...
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A Closer Look at Reporter Lentiviral Particles: Tool for Biomedical Research
Introduction For years, scientists have leveraged the brilliant potentialities of genetic science to explore how cells function and interact in living organisms. Among the numerous tools employed are "reporter systems," which use a visible marker to track gene expression, protein interactions, and other vital biological processes. One such system employs reporter lentiviral particles, using the ...
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Gene Therapy for Cystic Fibrosis
What is cystic fibrosis? Cystic fibrosis (CF) is a rare genetic disorder that affects the lungs, digestive system, and other organs. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes a protein that regulates chloride ions transport across cell membranes. These mutations cause the CFTR protein to be either absent or not function ...
By Protheragen
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The first CRISPR gene editing of vascular endothelium was made possible thanks to nanoparticles
The vascular endothelium is a single layer of endothelial cells that lines the surface of the vascular lumen, and it plays an important role in maintaining vascular homeostasis and tissue fluid balance. Many disorders, such as coronary artery disease, stroke, bronchopulmonary dysplasia, and pulmonary hypertension, are caused by endothelial dysfunction. Genome editing in endothelial cells can also ...
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Injecting mRNA and generating CAR-T directly in the body to repair the heart in one shot
January 6, 2022—Researchers at the Perelman School of Medicine at the University of Pennsylvania published a research paper in Science titled: CAR-T cells produced in vivo to treat cardiac injury. Heart damage or inflammation can induce fibroblasts to overproduce fibrous material, causing cardiac fibrosis, which hardens the heart muscle and impairs heart function. Heart failure, liver ...
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Introduction to AAV as a Gene Therapy Vector, Part 1
What are AAVs? and what makes them good candidates for gene therapy? In two previous posts, we introduced gene therapy, a method for curing genetic diseases by providing healthy copies of defective genes, and Adeno-associated virus (AAV) capsids, the gene therapy delivery system Dyno focuses on. In those posts, we also discussed how natural variants of AAV did not evolve for the specialized ...
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Chemyx Syringe Pumps in Neuroscience Research
Introduction The classical field of neuroscience entails biological or psychological investigation of the nervous system, including the brain, spinal cord and the systemic network of sensory nerve cells called neurons.1 Traditionally, this field comprised of purely biological studies but has since flourished into an interdisciplinary sphere addressing the developmental, physiological, ...
By Chemyx Inc
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The Key Technology of RNA Therapy
It is certain that RNA therapy is no longer a no-man's land for medical innovation. Not only have several products been launched and received good market response, but there are also many pipelines in the research and development side to advance to the middle and late clinical stage, and behind the boom are the technological breakthroughs of scientists. There are three key points in the ...
By BOC Sciences
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Peptide Vaccine Design
Vaccines are considered one of the most successful medical interventions in the past few centuries, aiming to harness the human immune system and generate lasting protection against specific diseases. Traditional vaccines rely on the use of inactivated pathogens to trigger an immune response. However, many of these formulations carry a high risk of causing allergies or autoimmune reactions. ...
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Overcoming Immunological Challenges Limiting Capsid-Mediated Gene Therapy With Machine Learning
A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors. Recent advances in high-throughput DNA synthesis, multiplexing and sequencing technologies have accelerated engineering of improved capsid properties such as production yield, packaging efficiency, ...
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Project - MentorEye
The aim of the project was to develop a Polish complementary system of molecular surgical navigation for the treatment of ...
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The Limitations and Challenges of Cancer Therapies
Considerable investment into cancer research has led to the development of remarkable new therapies, which have greatly improved therapeutic outcome for the countless individuals diagnosed with cancer. Until recently, almost all therapies that were granted clinical approval were small molecules. However, biotechnological advances in the last several decades have made it possible to develop ...
By Indee Labs
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New Cell Contenders in the CAR Field (Part 2)
Why do we need new cell contenders in the CAR field? Well, different CAR immune cells have different advantages and sometimes unique challenges or limitations. So, we began our discussion of alternative CAR-cell types with CAR-NK cells due to their current standing as the most popular alternative CAR-based therapy. However, the expansion of CARs to other cell types has already begun and ...
By Indee Labs
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