gene therapy Articles
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Strategies of biotechnology firms towards new, platform technologies
The human therapeutics sector of the biotechnology industry is the subject of a vast body of empirical literature, which attempts to analyse various success and failure factors for participating companies. This paper synthesises the historical trajectory of three current, leading-edge platform biotechnologies, namely the Polymerase Chain Reaction (PCR), combinatorial drug discovery, and gene ...
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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 a Novel Encapsulated Non-Viral Cell- Based, BBB-Penetrant Therapy for MPS I
Introduction MPS I is caused by a deficiency of the lysosomal enzyme a-L-iduronidase (IDUA) leading to GAG accumulation in multiple tissues and organs This accumulation results in a complex array of progressive, multi-systemic pathologies, including CNS manifestations Approved therapies include enzyme replacement therapy (ERT), with chaperone and gene therapies under investigation Treatment ...
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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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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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A Comprehensive Analysis of Cell and Gene Therapy
The scientific realm of biomedicine has witnessed extraordinary evolution in recent years, particularly in the spheres of cell and gene therapies. These therapies have radically revamped disease treatment modalities, transcending traditional approaches to become some of the most groundbreaking advancements in modern healthcare. This article aims to engage in an incisive analysis of cell and gene ...
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RNA-targeted Gene Therapy
In recent years, various nucleic acid-based therapies (NBT) have emerged as effective and specific activators of endogenous gene expression. Unlike gene therapy approaches supplementing gene expression, RNA-targeted therapy enhances protein production by selectively modulating cellular mechanisms mediated by endogenous RNA, such as transcription, splicing, translation, mRNA stability, and ...
By BOC Sciences
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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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Unleashing the Power of AAV Manufacturing for Large Scale Production in Biopharma
Adeno-associated viruses (AAV) have emerged as safe and effective vectors for gene therapy and the advancement of biopharmaceutical research. Today, navigating the complex landscape of AAV manufacturing has become a focal point for professionals in the biopharma industry looking to exploit this technology in the development of breakthrough treatments. Large-scale production of AAV vectors is a ...
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Development of a Novel Encapsulated Non-Viral Cell-Based, BBB-Penetrant Therapy for MPS I
MPS I is caused by deficiency of the lysosomal enzyme a-L-iduronidase (IDUA) leading to GAG accumulation in multiple tissues and organs This accumulation results in a complex array of progressive, multi-systemic pathologies, including CNS manifestations Approved therapies include enzyme replacement therapy (ERT), with chaperone and gene therapies under investigation Treatment with approved ...
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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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Unleashing the Potential of Recombinant Adeno-Associated Virus (rAAV) Particles in Gene Therapy
Recombinase Adeno-Associated Virus (rAAV) particles have taken the center-stage in gene therapy research and development due to their exceptional genetic manipulation capabilities, safety and unprecedented therapeutic success. As biomedical science advances, there is an ever-increasing enthusiasm in the usage of rAAV particles as effective vehicles for gene delivery and the subsequent therapy. ...
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Innovative technology of engineering magnetic DNA nanoparticles for gene therapy
The present research study is a novel innovation materials technology to engineer magnetic DNA nanoparticles for gene therapy and to enhance the localisation and gene expression of a plasmid DNA via magnetic DNA nanoparticles. Spermine (Sm) was chemically introduced to the hydroxyl groups of dextran, a polysaccharide, to obtain cationised dextran (dextran-Sm). When Fe2+ solution was added to the ...
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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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Poly(propyleneimine) dendrimers as potential siRNA delivery nanocarrier: from structure to function
The goal of the present investigation is to reveal the role of the Poly(propyleneimine) (PPI) dendrimer structure on the siRNA nanoparticles formation, facilitation of cell internalisation, and sequence specific knockdown of targeted genes. It was found that the higher generations of PPI dendrimers (G4 and G5) most effectively initiated the complexation of siRNA into discrete nanoparticles when ...
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Applications of nanotechnology in drug delivery systems for the treatment of cancer and diabetes
This paper discusses potential applications of nanotechnology in drug delivery systems and pharmacotherapeutics. There are a few limitations in the use of conventionally available drug delivery systems as pharmacological agents in disease treatment. Lack of target specificity, altered effects and diminished potency due to drug metabolism in the body, cytotoxicity of certain anti-carcinogenic ...
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Dr Annalisa Jenkins in conversation with Adam M Hill. `Extra Time` podcast
Annalisa Jenkins' inspirational career spans over 25 years in the global biopharma industry – she started her career at St Barts, in London, where she trained as a cardiologist, before joining the British Royal Navy as a medical officer during the Gulf Conflict, achieving the rank of surgeon lieutenant commander during her time there. Annalisa spent almost two decades producing a pipeline ...
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Persistent gene editing therapy using LNP-delivered mRNAs
Gene editing technology, represented by CRISPR/Cas9, has greatly accelerated the development of gene therapy, bringing hope to many genetic diseases that otherwise had no cure. In June 2021, Intellia Therapeutics' NTLA-2001, a CRISPR gene editing therapy for the treatment of transthyretin amyloidosis (ATTR) invented by Nobel Laureate Jennifer Doudna, was proven to be safe and effective in a ...
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Indee Lab’s CRISPR Delivery Technology for Affordable Immunotherapies
Ryan Pawell is the founder and CEO of Indee Labs, a Berkeley biotech. The mission of Indee Labs is to use microfluidic vortex shedding as a scalable method to deliver CRISPR reagents into cells for developing affordable cell and gene therapies in the future. In our previous blog post, we summarized the work from their latest publication on CRISPR-editing in T cells using microfluidic devices. In ...
By Indee Labs
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Regenerative medicine: new opportunities for developing countries
There has been a major shift in burden of disease in developing countries. Although traditionally associated with industrialised nations, epidemic levels of non-communicable diseases, such as diabetes, cardiovascular diseases and cancer, now threaten the developing world and are straining health systems that are still struggling with persisting levels of infectious disease. Developing countries ...
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