in vivo gene Articles
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High-throughput identification and validation of in situ-expressed genes of lactococcus lactis
Understanding the functional response of bacteria to their natural environment is one of the current challenges in microbiology. Over the past decades several techniques have been developed to study gene expression in complex natural habitats. Most of these methods, however, are laborious, and validation of results under in situ conditions is cumbersome. Here we report the improvement of the ...
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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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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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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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Perfluorooctane sulfonate (PFOS) affects hormone receptor activity, steroidogenesis, and expression of endocrine‐related genes in vitro and in vivo
Perfluorooctane sulfonate (PFOS) is a widespread and persistent chemical in the environment. We investigated the endocrine‐disrupting effects of PFOS using a combination of in vitro and in vivo assays. Reporter gene assays were used to detect receptor‐mediated (anti‐)estrogenic, (anti‐)androgenic, and (anti‐)thyroid hormone activities. The effect of PFOS on steroidogenesis was assessed both at ...
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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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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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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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Clinical CRISPR Success Demonstrates Need for Quality Control
The Clinical CRISPR Successes Are Stacking Up. But Where’s the Emphasis on Quality Control? In mid-September, Intellia released some promising data on two of their CRISPR-based therapeutics. These results are a significant milestone for genome editing and validation of its effectiveness in the clinic. However, the safety of CRISPR-based therapeutics has been a major concern and part of a ...
By CRISPR QC
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CD19: One of The Most Important Targets for Car-T Cell Therapy
What is CD19 Understanding CD19: Definition and Role in the Immune System CD19, also known as Cluster of Differentiation 19, is a cell surface protein belonging to the immunoglobulin superfamily. It serves as a crucial marker for B cells, playing a pivotal role in the human immune system. While predominantly found on the surface of B cells, CD19 is rarely expressed on other immune cells. Its ...
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