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Therapeutics

Image: The lack of dystrophin, shown in red, in an unedited human heart muscle cell with DMD (left) and the restoration of dystrophin in a CRISPR-edited human heart muscle cell (right) (Photo courtesy of the University of Texas Southwestern Medical Center).

MD Mouse Model Used to Tweak Gene-Editing Protocols

A new mouse model of Duchenne muscular dystrophy (DMD) that carries the exon 44 deletion mutation was used to develop strategies for correction of this mutation employing CRISPR/Cas9 to edit surrounding exons, which restores the dystrophin open reading frame. More...
25 Mar 2019
Image: Bone regenerates when two human cell populations (pericytes and adventitial cells) are mixed and implanted in a skull bone defect in immunocompromised mice (Photo courtesy of Yiyun Wang, Johns Hopkins University).

Mixture of Adipose-derived Cells Prove Better at Inducing Bone Repair

Researchers have defined the differential but overlapping roles of two cell subsets involved in the paracrine induction of bone repair. More...
06 Mar 2019
Image: Platelet decoys (pink), unlike normal platelets, are not activated by collagen fibers (gray), making them an attractive option for antithrombotic and cancer treatment (Photo courtesy of Harvard University).

Modified Platelets Reduce Risk of Blood Clots and Prevent Metastasis

A promising method for reducing the risk of blood clots and preventing tumor metastasis is based on treatment with blood platelets that have been modified to be incapable of activation and aggregation while retaining their functional binding properties. More...
27 Feb 2019
Image: A scanning electron micrograph (SEM) of engineered T-cells lacking coronin 1 (Photo courtesy of the University of Basel).

Novel Method Prevents Rejection of Transplanted Organs

A team of Swiss researchers reported finding a method that prevents rejection of transplanted organs without lessening the immune system's ability to fight off infection by pathogenic microorganisms. More...
28 Jan 2019
Image: A diagram of how regulatory T-cells suppress effector T-cells and dendritic cells (Photo courtesy of Wikimedia Commons).

Gene Therapy Cures Severe Autoimmune Disorder in Model

An often fatal autoimmune disorder was cured in mice by using a lentiviral vector to insert a working copy of the defective FOXP3 (Forkhead box P3, also known as scurfin) gene into blood stem cells. More...
24 Jan 2019
Image: A representation of the movement of the flower-like particle as it makes its way to deliver therapeutic genes (Photo courtesy of Washington State University).

Fluorinated Nanoparticles Facilitate Drug Transfer to Cellular Cytosol

In a proof-of-principle study, novel "nanoflower" drug transporters were used to successfully deliver model drug analogues to cells growing in culture. More...
22 Jan 2019
Image: A molecular model of the Melanocortin 4 receptor (Mc4r) protein (Photo courtesy of Wikimedia Commons).

Gene Editing Prevents Mice from Developing Hereditary Obesity

A modified version of the CRISPR/Cas9 genome-editing tool was used to correct a mutated gene in mice, which prevented the animals from becoming obese. More...
27 Dec 2018


BioResearch Therapeutics channel deals with all aspects of therapies beyond the five-year horizon.
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