After creating a stem cell
model of the disease from patients» skin cells, the Scripps Research scientists found strong evidence for the involvement of a DNA repair enzyme in the FXN triplet repeat expansion.
The advent of human - induced pluripotent stem cell (hiPSC) technology has provided a unique opportunity to establish cellular
models of disease from individual patients, and to study the effects of the underlying genetic aberrations upon multiple different cell types, many of which would not normally be accessible.
Not exact matches
But aside
from raising awareness and more than $ 20 million for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's
disease, over the past month, the challenge also may be unleashing a new
model for the $ 300 billion - a-year business
of charitable giving.
A NATION - WIDE outbreak
of foot and mouth
disease (FMD) could strip more than $ 50 billion
from Australia's livestock industry over 10 years, according to a new costing
model from the Australian Bureau
of Agricultural and Resource Economics and Sciences (ABARES).
More broadly, updated longitudinal studies
of the natural history
of hypertension, diabetes, and cardiovascular
disease are needed, because the data
from the National Health and Nutrition Examination Survey that we used in our
model are somewhat dated.
«In theory, we could
model progression
of the
disease by reprogramming skin cells
from patients at a range
of ages, including before symptoms begin.
To develop their «
disease in a dish»
model, the team took skin cells
from patients with Allan - Herndon - Dudley syndrome and reprogrammed them into induced pluripotent stem cells, which then can be developed into any type
of tissue in the body.
Researchers
from Instituto de Medicina Molecular (iMM) Lisboa have created a chimera virus that allows the study
of molecules to treat cancers caused by human herpes virus infection in mice
models of disease.
The behavioral tests used here
modeled one dimension
of the
disease — an inability to experience pleasure
from normal activities — but not others, such as stress and anxiety, and probably tap into different brain mechanisms in mice than in humans, he says.
Reductions in biodiversity
from illegal wildlife trade can have other substantial negative human health impacts, including the loss
of potential sources
of pharmaceuticals, experimental
models for studying
disease, crop pollination and micronutrients for humans lacking alternative sources
of protein.
In preclinical studies using cell
models that mimicked liver cells
of patients with the rare
disease Friedreich's ataxia (FA), a widely used cholesterol - lowering drug increased a precursor
of HDL (high - density lipoprotein), the «good cholesterol,» according to new research published in PLOS ONE
from the Perelman School
of Medicine at the University
of Pennsylvania.
Satellite imagery is used for all sorts
of climate study,
from identifying conditions that allow infectious
diseases like West Nile virus and cholera to emerge, to creating
models for predicting hurricanes, to distinguishing natural resources such as wind, water and sunlight.
Specifically, the Mount Sinai study was designed to test whether pharmacological compounds designed to block the function
of XPO1 / CRM1 could stop
disease progression in mouse
models that exhibit some
of the characteristics
of MS. Researchers found that two chemical agents (called KPT - 276 and KPT - 350) prevented XPO1 / CRM1
from shuttling cargo out
of the nucleus
of nerve cells, which protected them
from free radicals and structural damage.
This weather - based
model was then used to predict the impact on severity
of the
disease of future weather scenarios for the period
from 2020 to 2050.
Their mathematical
model also predicted how the Disneyland outbreak helped push California back
from the tipping point by making parents more afraid
of the
disease than the vaccine.
These techniques include: human tissue created by reprogramming cells
from people with the relevant
disease (dubbed «patient in a dish»); «body on a chip» devices, where human tissue samples on a silicon chip are linked by a circulating blood substitute; many computer
modelling approaches, such as virtual organs, virtual patients and virtual clinical trials; and microdosing studies, where tiny doses
of drugs given to volunteers allow scientists to study their metabolism in humans, safely and with unsurpassed accuracy.
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.
The journal provides cutting - edge research including results
from animal
models that are likely to apply to patients, studies in human tissue that provide new information about therapies or
disease, and innovative reports
of drug discovery and development.
He co-authored papers with his doctor, wrote a case study about himself, proposed a new
model of the
disease, and currently coordinates a dozen Castleman studies
from his small office at UPenn, where he is an assistant professor.
Most importantly, these cells protected mice
from developing diabetes in a
model of disease, having the critical ability to produce insulin in response to changes in glucose levels.
So until recently, much
of what scientists learned was gleaned by inference or
from studying mouse
models of the
disease.
This research depicts the findings
of Sampson et al., who show that signals
from gut microbes are required for the neuroinflammatory responses as well as hallmark gastrointestinal and a-synuclein-dependent motor deficits in a
model of Parkinson's
disease.
Next, the research team will examine specifically whether these liver cells obtained
from human embryonic stem cells in a dish help repair injured livers in preclinical animal
models of liver
disease.
Antibiotic treatment reduced motor deficits and molecular hallmarks
of Parkinson's
disease in a mouse
model, whereas transplantation
of gut microbes
from patients with Parkinson's
disease exacerbated symptoms in these mice.
Researchers
from the University's Institute
of Translational Medicine have found that the anti-epileptic drug ethosuximide has protective effects in certain neurodegenerative
disease models.
She and her colleagues will be exploring the role
of YY1 further, using clinical samples as well as mouse
models, to look at the protein in
diseases like lupus to deepen their understanding
of how autoimmunity could result
from the «escape»
of immune genes
from X chromosome inactivation.
This anxious behavior mirrored that
of CdLS patients, while autopsied brain tissue
from individuals with CdLS showed symptoms
of disease that matched those
of the experimental mice suggesting that they were a good animal
model.
Desgrosellier said the team will follow up with mouse
models containing tumor fragments
from patients to better reflect the diversity
of cell types present in human
disease.
Dr Leonardo Guasti added: «It represents an entirely new concept for the study
of the adrenal gland as the ability to generate donor - specific and functional adrenal - like cells will facilitate the next generation
of cell - based treatments for adrenal insufficiency, the
modelling of adrenal specific
diseases, and the testing
of personalised interventions on cells derived
from patients.»
Cultural stigma against
diseases that some falsely believe to be weaknesses
of character prevent people
from coming forward for trials — a problem when there are no good animal
models for
diseases like depression and schizophrenia.
In order to test their hypothesis in a
model that more closely mimicked human
disease, the researchers also tested the two drugs side - by - side on slices
of tumors removed
from patients during radical prostatectomy.
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.
The
model, described in a study published this week in Biomicrofluidics,
from AIP Publishing, will enable researchers to study potential
disease causes and test new drugs to treat IBD, said study author Amy Dawson, a postdoctoral researcher in the lab
of John Greenman at the University
of Hull, U.K..
Using a yeast
model of Parkinson's
disease, Lee and his team discovered two
of the compounds prevented the AS protein
from clumping, effectively allowing the cells to grow normally.
«Alzheimer's in a dish: Stem cells
from patients offer
model and drug - discovery platform for early onset form
of disease.»
«Traditional methods
of infectious
disease research using animal
models have provided limited information about
disease progression until the study's endpoint, when investigators could analyze tissues
from those animals,» he said.
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.
Reported today in
Disease Models & Mechanisms, Karim Si - Tayeb and colleagues in the research group of Bertrand Cariou from l'Institut du Thorax, Nantes used an innovative approach to develop unique patient cell - based models of PCSK9 - driven hypercholestero
Models & Mechanisms, Karim Si - Tayeb and colleagues in the research group
of Bertrand Cariou
from l'Institut du Thorax, Nantes used an innovative approach to develop unique patient cell - based
models of PCSK9 - driven hypercholestero
models of PCSK9 - driven hypercholesterolemia.
Using data
from the outbreak
of cholera that started in 2010 in the region, they developed a mathematical
model of the
disease's epidemiology.
Application
of sun protection factor 30 (SPF30) sunscreen prior to exposure to ultraviolet - B (UVB) light delayed melanoma onset in a mouse
model of the
disease, according to data
from a team at The Ohio State University Comprehensive Cancer Center Comprehensive Cancer Center — Arthur G. James Cancer Hospital and Richard J. Solove Research Institute.
«Use
of induced pluripotent stem cell (iPSC) technology» — which involves taking skin cells
from patients and reprogramming them into embryonic - like stem cells capable
of turning into other specific cell types relevant for studying a particular
disease — «makes it possible to
model dementias that affect people later in life,» says senior study author Catherine Verfaillie
of KU Leuven.
Those tests don't give us the details we need to understand the nature
of the pathology at the cellular level, so we
modeled the
disease to perform a systematic analysis
of the optic nerve,
from its origin in the eyes to termination in the brain.»
Later, he found that the inhibition
of this signaling route also protected Alzheimer's -
model mice
from behavioral impairments and pathological phenomena typical to the
disease.
Muotri noted that the research represents one
of the first efforts to use iPSCs and brain in - a-dish technology to generate novel insights about a
disease process and not simply replicate data
from other
models.
He is also developing a robust and comprehensive panel
of 3 - D cell culture
models from patient - derived primary cells that can be used to characterize different
disease phenotypes and investigate the chemo - response
of cells to novel or known drugs.
But this remarkable therapeutic benefit isn't limited to MS. Previous research in animal
models that mimic other
diseases suggest that certain forms
of amyloids can ease damage
from strokes, traumatic brain injuries and even heart attacks.
Previous studies
from this group have shown that lowering tau levels reduces abnormal brain activity in
models of Alzheimer's
disease, but this is the first demonstration that tau reduction may also be beneficial in intractable genetic epilepsy.
Most
of those findings came
from studies in cell line
models of diseases, however.
Now, in an article published in
Disease Models & Mechanisms, a team
from the University
of Alabama at Birmingham and Stanford University School
of Medicine describe the use
of a BDNF loop domain mimetic to improve motor function and object location memory in a mouse
model of Rett syndrome.
In a development that could lead to a new generation
of drugs to precisely treat a range
of diseases, scientists
from the Florida campus
of The Scripps Research Institute (TSRI) have for the first time designed a drug candidate that decreases the growth
of tumor cells in animal
models in one
of the hardest to treat cancers — triple negative breast cancer.