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Therapeutics

Image: In a recent study, CRISPR gene editing was shown to halt the progression of Duchenne muscular dystrophy (DMD) in dogs. The photomicrographs show dystrophin (in green) in a healthy diaphragm muscle (left), absence of dystrophin in a dog with DMD (center), and restoration of dystrophin in dogs treated with CRISPR (right) (Photo courtesy of the University of Texas Southwestern Medical Center).

Gene Editing Reverses DMD Muscle Damage in Model

The CRISPR/Cas9 genome-editing tool was used in a proof-of-concept study conducted in dogs suggested that the technique could be clinically useful for the treatment of Duchenne muscular dystrophy. More...
10 Sep 2018
Image: The role of miRNAs in inflammatory bowel disease (IBD): miR-29 loaded on a supercarbonate apatite prevented the development of inflammation by suppressing the production of inflammatory cytokines (IL-6, TGF-beta, and IL-23) secreted from dendritic cells and by suppressing the differentiation of naive T-cells to Th17 cells (Photo courtesy of Osaka University).

Nucleic Acid Therapy Effective in Inflammatory Bowel Disease Model

A team of Japanese researchers working with a mouse model recently described a novel method for using microRNAs to treat inflammatory bowel disease (IBD). More...
06 Sep 2018
Image: Gene therapy treated airway stem cells (Photo courtesy of Dr. Nigel Farrow, University of Adelaide).

Preconditioning Enhances Stem Cell Transplantation in CF Model

Disruption of the epithelial cell layer using the agent polidocanol was found to facilitate effective transplantation of cultured stem cells into nasal airways in a mouse model of cystic fibrosis. More...
15 Aug 2018
Image: Red blood cells can safely transport nanoscale drug carriers to chosen organs by targeted placement of intravascular catheters (Photo courtesy of the University of Pennsylvania).

RBC Transporters Used for Targeting Nanoparticle Drug Carriers

A novel, patent pending nanoparticle drug delivery system uses red blood cells to transport drug carriers directly to selected target organs with minimal exposure to other areas of the body. More...
14 Aug 2018
Image: A representation of the molecular structure of PDHX protein (Photo courtesy of Wikimedia Commons).

Cancer Growth Promoted by an Oncogenic MicroRNA

Cancer researchers have discovered a critical molecular mechanism by which a specific microRNA promotes breast cancer growth through suppression of production of the enzyme PDHX (pyruvate dehydrogenase complex component X). More...
02 Aug 2018
Image: A micrograph of a teratoma showing tissue from all three germ layers: mesoderm (immature cartilage - left-upper), endoderm (gastrointestinal glands - center-bottom) and ectoderm (epidermis - right) (Photo courtesy of Wikimedia Commons).

Teratomas Offer Source for Rebuilding Damaged Tissues

Muscle stem cells, which were derived from benign teratoma tumors, were found to demonstrate exceptional potential as starting material for rebuilding and maintaining damaged muscle tissues. More...
19 Jul 2018
Image: This artist\'s concept shows two nanoparticles coming into the brain (at top) while a pair of scissors cuts at bottom right. Use of gene editing to cut DNA at a precise location reduced autism symptoms in mice with fragile X syndrome (Photo courtesy of the University of California, Berkeley).

CRISPR/Cas9 Corrects Symptoms of Fragile X Syndrome in Model

In order to correct fragile X syndrome in a mouse model, a team of neurological disease researchers used gold nanoparticles rather than viruses to deliver the CRISP\Cas9 gene-editing tool to cells in striatum, a brain region associated with formation of habits. More...
06 Jul 2018


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