Interpreting genetic variants in kidney and genitourinary disorders will likely require systematic curation of...

1. This secondary analysis of genetic data emphasized the high number of potentially “pathogenic” variants that are revealed through exome sequencing when analyzing a...

Gene therapy targets Duchenne muscular dystrophy in new trial [PreClinical]

1. Mutations in the gene encoding dystrophin, a large scaffolding protein that supports muscle cell structure, result in Duchenne muscular dystrophy (DMD).2. A gene...

Replacement of recipient hematopoietic cells with donor cells may be a major factor in...

1. Allo-HSCT treatment resulted in significant prolonged reduction in HIV-1 viral reservoirs.2. Mouse models showed adoptive transfer of peripheral CD4+ T cells to immunosuppressed...

Designer opioid receptor agonist provides pain relief without opioid side effects in nonhuman primates...

1. Mu opioid peptide (MOP) receptor agonists provide effective pain relief, but are associated with severe side effects such as respiratory depression, opioid-induced hyperalgesia,...

Microtubules linked to cardiomyocyte function in heart failure [PreClinical]

1. Post-translational detyrosination of microtubules results in resistance to cardiomyocyte sarcomere shortening and stretch.2. Pharmacologic and genetic strategies to inhibit the detyrosination of...

Comparative analysis of wounds from oral mucosa versus skin reveals novel therapeutic targets for...

1. Comparative profiling of wounds induced in the oral mucosa versus skin revealed expression of genes that prime the oral mucosa for wound healing....

Viral vector based gene therapies show promise for treating focal epilepsy [PreClinical]

1. Seizures are often associated with accumulation of extracellular glutamate due to the synchronous discharges of excitatory neurons.2. Treatment of rats with a lentiviral...

Human neural stem cell grafts in rhesus monkeys improve motor function after spinal cord...

1. Following cervical spinal cord injury, rhesus monkeys were grafted with human neural progenitor cells.2. Over the course of 9 months, grafted human neural...

Early diagnosis of HIV infection through analysis of oral fluid [Pre-Clinical]

1. A novel method – Antibody Detection by Agglutination-PCR (ADAP) – was developed to diagnose HIV in oral fluid samples.2. ADAP is both highly...

Improved islet encapsulation device for the treatment of type 1 diabetes [PreClinical]

1. A device for islet transplantation for type 1 diabetes was designed and demonstrated to be safe and retrievable in animal models.2. When tested...

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