His uses genome engineering methods to test the role of specific genetic changes in induced pluripotent cell (iPSC)-
derived models of disease.
Not exact matches
«The ongoing line
of work with this drug is an excellent example
of the bench getting even closer to the bedside — our lab work with the drug in patient -
derived xenograft
models of disease makes possible the clinical trials taking place at the University
of Colorado Hospital next door,» Jimeno says.
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.»
In this latest research, Mohan and his colleagues present new findings that detail the use
of a synthetic, plant -
derived compound — abbreviated CDDO — that was shown to effectively suppress the multiple steps
of lupus development in murine
models, including the onset
of kidney
disease.
«This work in an experimental
model is the first published report
of exploiting an uropathogen -
derived host modulatory molecule in a clinically relevant
model of bladder
disease, and it points to the potential utility
of this as an alternate treatment approach.»
But if homologous recombination could be worked out in human (embryonic) stem cells, then cardiomyocytes with mutations in ion channels could be
derived, as well as a large number
of other very useful
disease models of other tissues.
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.
Given the rapid succession
of generations in yeast, we can use it as a
model organism — and study the mechanisms
of aneuploidy in much greater detail to find out whether we can
derive from it new approaches for diagnosing and treating human
diseases.»
Metabolites
derived from the microbiome block protein misfolding in test tubes and prevent neurodegeneration in a fly
model of a
disease related to Parkinson's, hinting that gut -
derived metabolites may hold therapeutic promise (Lap Ho, abstract 573.23, see attached summary).
This Purkinje neuron was
derived from patients with tuberous sclerosis and
model properties
of the
disease at the cellular and molecular level.
Our conversion process should
model late - onset aspects
of the
disease using neurons
derived from patients with the condition.»
«Lab - grown human colons change study
of GI
disease: Stem cell
derived organoids fill gap in
modeling common ailments.»
Human embryonic stem cells
derived from affected embryos during a pre-implantation diagnostic (PGD), as well as the conversion
of somatic cells, such as skin fibroblasts, into induced pluripotent stem cells by genetic manipulation, offer the unique opportunity to have access to a large spectrum
of disease - specific cell
models.
Susceptibility loci to coronary arteritis in animal
model of Kawasaki
disease induced with Candida albicans -
derived substances
However, despite its marked promise for
disease modeling, development
of novel therapies, and regenerative medicine, stem cell
derived organoid technology faces many remaining challenges.
Here, Anna presents how studies
of neural stem cells and neurons
derived from iPS cells
of patients show faithful mimicking
of known
disease phenotypes in our cellular
models of disease, like Alzheimer's
disease, autism, and Down syndrome.
For these purpose, we explore and validate the use
of cell progenies
derived from pluripotent stem cells to produce
disease models in a dish, to screen for compounds (small molecules, biologicals, etc).
In parallel to this cell therapy application, keratynocytes and melanocytes
derived from pluripotent stem cells will be used for pathological
modelling of genodermatoses allowing identification
of new
disease - specific pharmacological treatments
This approach is now being used to
derive embryonic stem cells from a variety
of strains including
disease models from which in vitro tools are in demand, including mouse
models of Alzheimer's
disease, cytogenetic disorders (Turner's syndrome and Down syndrome), and tool strains for systems genetics.
Disease - specific human pluripotent stem cells, from embryonic origin or derived from reprogramming somatic cells, offer the unique opportunity to have access to a large spectrum of disease - specific cell
Disease - specific human pluripotent stem cells, from embryonic origin or
derived from reprogramming somatic cells, offer the unique opportunity to have access to a large spectrum
of disease - specific cell
disease - specific cell
models.
Dopamine neurons
derived from human ES cells efficiently engraft in animal
models of Parkinson's
disease
The experiments, performed in rat
models of Parkinson's
disease, reveal that the latest version
of stem cell -
derived dopamine cells fully mimic the characteristics and function
of the dopamine neurons that are lost in Parkinson's
disease.
He has developed novel cellular
models of neurological
diseases (schizophrenia, MS, autism, Alzheimer's, Parkinson's) using human induced pluripotent stem cells (iPSCs)
derived from donor patients and differentiated to specific neuronal cell types.
This discovery, which earned Dr. Yamanaka the 2012 Nobel Prize in Physiology or Medicine, has revolutionized the fields
of stem cell biology and regenerative medicine and has allowed scientists to
model disease from patient -
derived cells in a dish, thus working towards a new era
of personalized medicine.
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.
In sum, human NSCs represent an invaluable source
of cells to investigate human iPS induction, and also represent a potential source
of cells for
deriving patient - specific pluripotent stem cells for
modeling human
disease.
A recent article in Chemical & Engineering News describes the promising properties
of curcumin, a compound
derived from turmeric, in
models of Alzheimerâ $ ™ s
disease.
«The development
of a functional human kidney glomerulus chip opens up an entirely new experimental path to investigate kidney biology, carry out highly personalized
modeling of kidney
diseases and drug toxicities, and the stem cell -
derived kidney podocytes we developed could even offer a new injectable cell therapy approach for regenerative medicine in patients with life - threatening glomerulopathies in the future,» said Ingber.
Our lab uses both cardiomyoctes
derived from human stem cells (iPS cell -
derived cardiomyocytes) and mouse
models harboring the human mutation to study which exact changes occur during the onset and development
of the
disease.
Taken together, these results demonstrated that
disease - specific human iPS cell —
derived hepatocytes are capable
of modeling key pathological feature
of A1ATD in vitro and may also prove useful for future drug screening assays.
Treatment
of perianal fistulas with human embryonic stem cell -
derived mesenchymal cells: a canine
model of human fistualizing Crohn's
disease.