Preclinical News and Research

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Study assesses upper, lower extremity muscle function in Osteogenesis Imperfecta Type IV

Osteogenesis Imperfecta (OI) is a heritable disorder characterized by increased bone fragility. In OI Type IV muscle weakness has important functional consequences. Children and adolescents with OI type IV often have restricted mobility despite multidisciplinary treatment with bisphosphonates, intramedullary rodding surgery and rehabilitation.

26 Jul 2017

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy.

25 Jul 2017

Researchers present novel gene-editing technique to prevent retinal angiogenesis

A research team from the Schepens Eye Research Institute of Massachusetts Eye and Ear has successfully prevented mice from developing angiogenesis of the retina--the sensory tissue at the back of the eye--using gene-editing techniques with CRISPR-Cas9.

25 Jul 2017

CUMC researchers capture first snapshot of key brain receptor in action

Columbia University Medical Center researchers have captured the first three-dimensional snapshots of the AMPA-subtype glutamate receptor in action. The receptor, which regulates most electrical signaling in the brain, is involved in several important brain activities, including memory and learning.

24 Jul 2017

University of Birmingham joins €19 million project to develop better treatments for heart disease

The University of Birmingham is part of a major new €19 million project aimed at breaking new ground for the development of treatments for millions of patients with heart disease in Europe.

19 Jul 2017

Study seeks to understand genetic synergy in cleft palate

Like all of the individual elements of fetal development, palate growth is a marvel of nature. In part of this process, ledges of tissue on the sides of the face grow downwards on each side of the tongue, then upward, fusing at the midline at the top of the mouth.

19 Jul 2017

Research shows how dietary essential fatty acids can help treat Alzheimer’s, other diseases

Understanding how dietary essential fatty acids work may lead to effective treatments for diseases and conditions such as stroke, Alzheimer's disease, age-related macular degeneration, Parkinson's disease and other retinal and neurodegenerative diseases.

18 Jul 2017

Screening individuals at-risk for psychosis may help predict future violent behavior

A new study of young persons at clinical high-risk of developing psychosis has identified measures of violence potential that may be useful in predicting both the increased risk of future violent behavior and the actual development of psychosis.

17 Jul 2017

Cord-blood-derived natural killer cells can be modified to destroy B-cell cancers

Immune cells with a general knack for recognizing and killing many types of infected or abnormal cells also can be engineered to hunt down cells with specific targets on them to treat cancer, researchers at The University of Texas MD Anderson Cancer Center report in the journal Leukemia.

13 Jul 2017

Bone metastasis-targeting drug shows potential as new therapeutic strategy for osteosarcoma

The treatment of osteosarcoma, the most common tumor of bone, is challenging. A study led by The University of Texas MD Anderson Cancer Center found a drug known as bone metastasis-targeting peptidomimetic (BMTP-11) has potential as a new therapeutic strategy for this devastating illness.

13 Jul 2017

New partnership to advance novel airway-delivered gene therapy for treating pulmonary hypertension

Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure.

13 Jul 2017

Targeting acid metabolite reduces size and spread of breast cancer

It's a metabolite found in essentially all our cells that, like so many things, cancer overexpresses. Now scientists have shown that when they inhibit 20-HETE, it reduces both the size of a breast cancer tumor and its ability to spread to the lungs.

12 Jul 2017

Gelesis reports completion of treatment for last patient in pivotal GLOW study

Gelesis, Inc., a biotechnology company developing a novel category of therapies to safely induce weight loss, improve glycemic control, and treat other chronic diseases related to the gastrointestinal (GI) pathway, is pleased to report today that the last patient has completed treatment in the pivotal GLOW (Gelesis Loss Of Weight) Study.

12 Jul 2017

ERYTECH partners with Queen’s University to advance its product candidate for treating arginase-1 deficiency

ERYTECH Pharma, a French clinical-stage biopharmaceutical company developing innovative therapies for rare forms of cancer and orphan diseases, today announced that it has entered into a research collaboration with Queen's University in Canada to advance the preclinical development of ERYTECH's eryminase program specifically for the treatment of arginase-1 deficiency, a rare and severe metabolic disorder related to arginine metabolism.

12 Jul 2017

Tulane University receives $12 million NIH grant to develop drug and vaccine against Lassa fever

The National Institutes of Health has awarded Tulane University more than $12 million to test a promising drug treatment against Lassa fever and develop a vaccine against the deadly disease endemic in parts of West Africa.

11 Jul 2017

Scientists develop potential new therapy for targeting skin cancer metastases in the brain

Investigators from Brigham and Women's Hospital (BWH) and the Harvard Stem Cell Institute have a potential solution for how to kill tumor cells that have metastasized to the brain.

10 Jul 2017

NYU researcher to study biological differences between men and women in oral cancer pain

Nicole Scheff, PhD, a postdoctoral fellow in the laboratory of Dr. Brian L. Schmidt, director of the NYU Bluestone Center for Clinical Research and the NYU Oral Cancer Center, both located at New York University College of Dentistry, has received a two-year, $120,000-plus F32 grant from the National Institute of Dental and Craniofacial Research.

10 Jul 2017

Researchers receive $8.6 million NIH grant for efforts to improve lung transplantation

Researchers at the University of Virginia School of Medicine have received more than $8.6 million in federal grants to support efforts that could dramatically increase the number of lungs available for transplant – and then save the lives of the people who receive them.

6 Jul 2017

Novel small molecule tracer for PET imaging spots blood clots

Blood clots in veins and arteries can lead to heart attack, stroke, and pulmonary embolism, which are major causes of mortality.

6 Jul 2017

Scientists develop novel DNA-based strategy to protect against broad array of influenza viruses

A novel, synthetic, DNA-based strategy to provide protection against a broad array of influenza viruses has been developed in preclinical models by scientists at The Wistar Institute, MedImmune (the global biologics research and development arm of AstraZeneca) and Inovio Pharmaceuticals, Inc.

6 Jul 2017

Preclinical News and Research

Study assesses upper, lower extremity muscle function in Osteogenesis Imperfecta Type IV

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Researchers present novel gene-editing technique to prevent retinal angiogenesis

CUMC researchers capture first snapshot of key brain receptor in action

University of Birmingham joins €19 million project to develop better treatments for heart disease

Study seeks to understand genetic synergy in cleft palate

Research shows how dietary essential fatty acids can help treat Alzheimer’s, other diseases

Screening individuals at-risk for psychosis may help predict future violent behavior

Cord-blood-derived natural killer cells can be modified to destroy B-cell cancers

Bone metastasis-targeting drug shows potential as new therapeutic strategy for osteosarcoma

New partnership to advance novel airway-delivered gene therapy for treating pulmonary hypertension

Targeting acid metabolite reduces size and spread of breast cancer

Gelesis reports completion of treatment for last patient in pivotal GLOW study

ERYTECH partners with Queen’s University to advance its product candidate for treating arginase-1 deficiency

Tulane University receives $12 million NIH grant to develop drug and vaccine against Lassa fever

Scientists develop potential new therapy for targeting skin cancer metastases in the brain

NYU researcher to study biological differences between men and women in oral cancer pain

Researchers receive $8.6 million NIH grant for efforts to improve lung transplantation

Novel small molecule tracer for PET imaging spots blood clots

Scientists develop novel DNA-based strategy to protect against broad array of influenza viruses

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