Reprogramming human somatic cells to pluripotency represents a valuable resource for the development of in vitro
based models for human disease and holds tremendous potential for deriving patient - specific pluripotent stem cells.
Not exact matches
«The study results elucidate the molecular mechanisms underlying
disease progression in multiple sclerosis
models, providing a
basis for future clinical trials to determine safety and efficacy of these chemical agents in
humans with demyelinating disorders,» says Patrizia Casaccia, MD, PhD, Professor of Neuroscience, Genetics and Genomic Sciences at Mount Sinai and senior author of the study.
In recent years, songbirds» similarities to
human vocal learning have piqued researchers» interests in using them as a functional animal
model to study the neurological
basis for Huntington's
disease.
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.
Though mutations that cause
human cancer have traditionally been thought to originate from heredity or environmental sources, these results — grounded in a novel mathematical
model based on data from around the world — support a role
for so - called «R» or random mutations in driving the
disease.
The Center, which collaborates with CiRA, is creating
human, iPS -
based disease models needed to accelerate drug development
for a host of devastating
for cardiovascular, viral and neurological illnesses.
At longer extend,
based on our technical and scientific expertises, this project aims to extend pathological
modelling to others neuro - muscular
diseases, such as Spinal Muscular Atrophy (SMA)
for which,
human pluripotent stem cells carrying the causal mutation have been derived.
Dr. Pfaff has developed a novel embryonic - stem cell -
based model of SMA that phenocopies the pathology of
human SMA, and can be used to study the
basis of the
disease and screen
for compounds that might increase survival of motor neurons.
Each cell -
based treatment approach is presented as a potential therapy
for a specific
disease or a
disease category (e.g.: cardiovascular
diseases, metabolic
diseases, etc.), studied in animal
models or in
humans.
Using genetic and epigenetic analyses coupled with powerful perturbation technologies to test gene functions in
human cells and mouse
models, we hope to identify the critical drivers of this
disease and the
basis for therapeutic responses.
We therefore suggest that the presence of the mutated transgenes (AβPP and PS1), which are per se the
basis for the genetic form of Alzheimer's
disease in
humans, directly interferes with gut function as shown here
for the
disease model mice.
These experiments are innovative because they seek to improve a mouse
model based on current knowledge from
human disease, while also testing novel therapies that could be of benefit
for affected individuals.
Robert D. Schreiber, Ph.D., an associate director of CRI's Scientific Advisory Council
based at Washington University School of Medicine in Saint Louis, Missouri, developed a new
model of breast cancer that more closely resembles the progression of hormone receptor - positive
disease in
humans, overcoming a major obstacle in the study of breast cancer and the development of new immune -
based therapies
for the
disease.
These two properties (self - renewal and pluripotency) confers
human pluripotent stem cells a unique interest
for clinical applications since they could allow the production of infinite quantities of cells
for disease modelling, drug screening and cell
based therapy.