Sentences with phrase «models of diseases targeted»

Once this work is completed, a second stage is committed, in which researchers of I - Stem, in collaboration with specialists in animal models of diseases targeted by the therapeutic explore the effects of experimental transplantation of cells obtained laboratory.

Not exact matches

«Now we have two interesting models that are selectively targeting specific parts of the brain: the thalamus in FFI and the hippocampus in CJD,» says Jackson, who is now a Group Leader at the German Center for Neurodegenerative Disease.

Our data demonstrate the feasibility of targeted gene disruption in multiple rat strains within 4 months time, paving the way to a humanized monoclonal antibody platform and additional human disease models.

Complementing the company's in - house Crispr programme, the technology will identify and validate new targets in preclinical models across a range of disease areas.

From accelerating the identification and validation of novel therapeutic targets, to creating better animal models of human diseases in a shorter time frame, to reducing the number of failed products, Crispr looks set to shave millions off R&D costs and boost drug discovery, she says.

«This is the first time a compound targeting Gaucher disease has been tested in a mouse model of Parkinson's disease and was shown to be effective,» said the study's senior author, Marie - Francoise Chesselet, the Charles H. Markham Professor of Neurology at UCLA and director of the UCLA Center for the Study of Parkinson's Ddisease has been tested in a mouse model of Parkinson's disease and was shown to be effective,» said the study's senior author, Marie - Francoise Chesselet, the Charles H. Markham Professor of Neurology at UCLA and director of the UCLA Center for the Study of Parkinson's Ddisease and was shown to be effective,» said the study's senior author, Marie - Francoise Chesselet, the Charles H. Markham Professor of Neurology at UCLA and director of the UCLA Center for the Study of Parkinson's DiseaseDisease.

In the study, exosomes, which are generated by all cells and are naturally present in blood, were modified as «iExosomes,» capable of delivering small RNA to specifically target mutant KRAS, resulting in disease suppression and increased overall survival in mouse models.

This is important because it makes it possible to model almost any autoimmune disease, evaluate new drug therapies, and even identify novel targets in the emerging area of cancer immunotherapies.

Mardinoglu says the team's network modeling approach, which relied on data from the Sweden - based Human Protein Atlas project and The Genotype - Tissue Expression (GTEx) project consortia, can be used in the identification of drug targets and eventually in the development of efficient strategies for treating a number of chronic liver diseases.

Using experimental models and state - of - the - art technology, the scientists found that switching off this protein leads to better control of glucose production from the liver, revealing a potential new target that may be used to treat type 2 diabetes and other metabolic diseases.

Using a recently developed genome - editing technique called CRISPR, a Chinese team has successfully altered two target genes in cynomolgus monkeys, paving the way for the development of monkey models that mimic human diseases.

«The induced pluripotent stem cells we used in this study proved to be extremely useful in disease modelling, and they could offer an excellent platform for drug discovery and testing new therapeutic targets for Alzheimer's disease in the future,» says Early Stage Researcher Minna Oksanen, the lead author of the study.

New research published today in Disease Models & Mechanisms indicates that early intervention should be a key target in the development of new therapeutics for Friedreich's ataxia, as current treatments may be administered too late to target the disease effecDisease Models & Mechanisms indicates that early intervention should be a key target in the development of new therapeutics for Friedreich's ataxia, as current treatments may be administered too late to target the disease effecdisease effectively.

Future work will focus on identifying cellular targets of ampulexins, and potentially generating a useful animal model for the study of Parkinson's disease treatments.

To narrow down the number of chemical compounds that could be potential drug candidates, scientists utilize computer models that can predict how a particular chemical compound might interact with a biological target of interest — for example, a key protein that might be involved with a disease process.

Under the new model, researchers could target disease - specific SNO synthases working downstream of NO.

In particular, a transgenic mouse model of early Alzheimer's disease shows that the more focally targeted an intervention is, the more specific of an effect can be elicited.

The resulting data are being combined in a systems biology approach with high - resolution clinical phenotyping and findings obtained with a large array of established and novel in vitro, ex vivo and in vivo disease models to identify disease - associated genetic variants, disease - defining molecular signatures, and potential targets for therapeutic intervention.

The CCFA Genetics Initiative seeks to deliver validated pathway models of IBD disease mechanisms, new animal models for analysis and development, chemical probes of relevant pathways, new proteins and pathway targets, recruitment of new IBD investigators, and a tested paradigm for rapid analysis and cure for Crohn's disease.

Biomedcode welcomes suggestions and proposals for the development of novel animal disease models as well as for the generation of animals humanized for targets of interest.

Our collaborations with industry, government agencies, and academia focus on developing new disease models, elucidating new therapeutic targets to develop treatments, safety testing on new chemical entities, enabling better and safer design of new consumer products, and new applications in personalized healthcare.

We focus on developing computational methods and tools for (a) analyzing large - scale gene expression data related to human cancer in search for gene markers and disease sub-categories, (b) identifying regulatory elements such as miRNA precursors and their targets in whole genomes of plants and mammals, (c) building theoretical models of gene regulatory networks.

His research interest focuses on 1) development and application of TALEN and CRISPR technologies for targeted genome editing in plant species, and 2) molecular mechanism of plant / microbe interactions and crop disease resistance engineering by using the bacterial blight of rice as a model.

Following a Forward Genetics approach, Fleming researchers identified a novel neurological mouse model caused by a functional mutation in the Slc25a46 gene, a new pathogenic target in a wide spectrum of human neurological diseases, including optic atrophy, Charcot - Marie - Tooth type 2, Leigh syndrome, progressive myoclonic ataxia and lethal congenital pontocerebellar hypoplasia.

Other programs will use cellular models of heart disease created from stem cells to identify potential new drug targets.

Because of the ease with which the CRISPR / Cas9 system can be applied, it has quickly become a robust tool for generating accurate genetic disease models in the laboratory and for identifying novel therapeutic targets in the clinic.

For such study, we have used the McGill - R - Thy1 - APP transgenic rat, which is unique compared to other rodent models in that the AD - like phenotype has been achieved with a single genomic insertion of a mutated human APP transgene; minimizing off - target genetic corruption and therefore being closer to the human disease [32].

The main goal of these technologies is to accelerate the comprehension of our genome and of human diseases, and to promote therapeutic innovation through the validation of molecular targets and their effects in reference model organisms.

They provide a new target for potential therapies, and they furnish a model system for more rapid and less expensive study of prion diseases and potential treatments.

Scientific meeting «Gene targeting, genome editing & transgenesis: research application» We are organizing joint meeting with our colleagues from Masaryk University in Brno dedicated to current progress in transgenic technologies in research application, animal models of diseases and Wnt signalling.

Host: Dr. Matt Farrer Speaker: Dr. Richard Wade - Martins, Univ. of Oxford Title: Translating Parkinson's: modelling disease for target discovery

Targets of research range from basic molecular and cellular mechanisms, the manipulation of these mechanisms in animal models, analyses of the genes and gene products in cardiovascular disease, and clinical research that seeks to improve diagnosis and therapy for patients.

Dr. Falk is also PI of an NIH, pharma, and philanthropic funded translational research laboratory group at CHOP that investigates the causes and global metabolic consequences of mitochondrial disease, as well as targeted therapies, in C. elegans, zebrafish, mouse, and human tissue models of genetic - based respiratory chain dysfunction, and directs multiple clinical treatment trials in mitochondrial disease patients.

In contrast to the current drugs that target one protein or one mechanism, microRNA - based AMD therapy provides a unique «one - molecule, multiple - targets» model for disease treatment, which fits particularly well with the complex nature of AMD.

They will use CRISPR genome - editing technology to correct or insert disease - causing mutations in experimental models, and screen for potential drug targets with the goal of correcting neuronal dysfunction caused by the mutations.

• Disease - driving pathways that involve the human immune system are often targeted by antibodies, and Organ - Chips recreate complex interactions of different human cell types and aspects of the human immune system, overcoming limitations of animal models which do not reflect all human immune cells.

The research lines of the Bakkers group include unraveling the genetics of normal cardiac development and body axis formation during development, investigating the molecular mechanisms of heart regeneration in the zebrafish and how this can be compared to heart injury in the mammals, and modeling of human (cardiac) disease in the zebrafish to unravel biological mechanisms behind the disease and to identify new drug targets.

THE MOUSE MODEL FOR CYSTIC FIBROSIS, as with models for many diseases, owes its existence to a technique called gene targeting, which was developed in the 1980s by Mario Capecchi, a professor of human genetics and biology at the University of Utah who won the 2007 Nobel Prize in Physiology or Medicine for his work.

Two new NSG mouse models allow immunological dissection of graft - versus - host disease (GVHD) responses and in vivo testing of therapeutic agents targeting human CD4 T cells.

Moreover, knock in animal models for syngeneic studies and allowing us to isolate transgenic armed effector cells are developed in order to show proof of concept for the diverse applicability of our modular targeting systems including for tumor diseases, autoimmune diseases, GvHD, transplantation / rejection as well as viral infections.

Functional Medicine provides a model that honors the evidence base and targeted efficacy of the acute care model while also incorporating new medical science and traditional methods that, when taken together, more appropriately address the root causes of chronic disease.