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Image: Two megakaryoctes (see arrows) are visible in this slide of bone marrow (Photo courtesy of Wikimedia Commons).

Gene Therapy Treatment Uses Microparticles to Deliver DNA or RNA

A novel gene therapy approach for correcting hereditary blood disorders is based on the use of megakaryocytic microparticles (MkMPs) to deliver plasmid DNAs and small RNAs to hematopoietic stem and progenitor cells (HSPCs). More...
18 Dec 2018
Image: Toxic amyloid plaques (red) and tau tangles (brown) form on the brain of a mouse modeled to have Alzheimer\'s disease. This study shows that a DNA vaccine reduces both amyloid and tau in the mouse AD model, with no adverse immune responses (Photo courtesy of the University of Texas Southwestern Medical Center).

Novel DNA Vaccine Prevents AD in Mouse Model

A team of Alzheimer's disease (AD) researchers has shown that a DNA vaccine directed at the toxic Abeta42 peptide protected the animals comprising a mouse (AD) model from both amyloid plaques and pathogenic tau tangles. More...
04 Dec 2018
Image: A scanning electron micrograph (SEM) of a human T-cell (Photo courtesy of the U.S. National Institutes of Health).

Technology Evaluated for T-cell Receptor Identification and Cloning

A team of cancer researchers developed a tracking and screening system that accurately identifies T-cell receptors from individual tumors within just a few days. More...
21 Nov 2018
Image: A biodegradable inorganic nanoscaffold consisting of stem cells, proteins, and drugs for advanced stem cell therapy and drug delivery (Photo courtesy of KiBum Lee, Letao Yang and Sy-Tsong Dean Chueng, Rutgers University).

Biodegradable Nanoscaffolds Improve Chances for Stem Cell Therapies

The therapeutic potential of biodegradable nanoscaffolds made from manganese dioxide (MnO2) for advanced stem cell transplantation and neural tissue engineering was discussed favorably in a recent paper. More...
15 Nov 2018
Image: A graphic representation of DNA editing (Photo courtesy of the U.S. National Institutes of Health/Jill George).

In vivo CRISPR/Cas9 Therapy Prevents Arrhythmias in Model

Delivery of the CRISPR/Cas9 gene-editing tool via an adeno-associated viral vector (AAV) directly to heart cells in a mouse model of catecholaminergic polymorphic ventricular tachycardia was found to efficiently and specifically prevent development of arrhythmias. More...
14 Nov 2018
Image: The crystal structure of S. pyogenes Cas9 in complex with sgRNA and its target DNA at 2.5-Angstrom resolution (Photo courtesy of Wikimedia Commons).

Immune Sensitivity Hampers Clinical Applications of CRISPR/Cas9

Findings presented in a recent paper suggest that use of the CRISPR/Cas9 gene-editing tool in clinical settings may be more complicated than previously thought, since most people have a pre-existing immune sensitivity to proteins from the bacterial source of Cas9. More...
12 Nov 2018
Image: A re-examination of adeno-associated virus capsid structural data revealed evidence for alterations at several sites (Photo courtesy of Lakshmanan Govindasamy, University of Pennsylvania).

Researchers Aim to Reduce Variability of Gene Therapy Vectors

A recently published study illuminated a previously unknown aspect of adeno-associated virus capsid heterogeneity and highlighted its importance in the development of these vectors for use in gene therapy applications. More...
30 Oct 2018

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