«Stem
cell disease model clarifies bone cancer trigger.»
In close collaboration with Dr. Yamanaka Dr. Conklin's laboratory was able to establish new iPS
cell disease models, gain new insights into iPS cell biology, and develop new tissue engineering methods.
Not exact matches
He conducted his postdoctoral research at Brigham and Women's Hospital / Harvard Medical School, where he researched the role of the Wnt signaling pathway in mouse
models of kidney
disease, and was part of a team that discovered a stem
cell subtype responsible for solid organ fibrosis.
«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.
In the present study, the researchers have discovered a reason for reduced fertility in people with autoimmune polyendocrine syndrome type 1 (APS1), which increases the risk of developing autoimmune
disease (caused by the immune system attacking and damaging healthy
cells) and which is often used as a
model for autoimmune
disease in general.
«Chronic inflammation of the intestine is thought to be caused by abnormal interactions between gut microbes, intestinal epithelial
cells and the immune system, but so far it has been impossible to determine how each of these factors contribute to the development of intestinal bowel
disease,» said Hyun Jung Kim, Ph.D., former Wyss Technology Development Fellow and first author on the study, speaking about the limitations of conventional in vitro and animal
models of bacterial overgrowth and inflammation of the intestines.
Currently, I work on three directions: (1)
cell motility and the cytoskeleton, (2)
modeling of physiology and
diseases (such as autoimmune diabetes), and (3) swarming and aggregation behaviour in social organisms.
Exposed to both air and fluid, the
cells might also be used to
model the lungs and test drugs for lung
disease.
The researchers are beginning to use their
model to test whether Zika virus, and other pathogens associated with congenital
disease, can infect placental
cells and / or cross the placental barrier.
«The fact that a [
cell] culture expresses most genes present in the brain says nothing about its appropriateness as a
disease model,» says Knoblich.
«This research has broad impact, because by deepening our understanding of
cell reprogramming we have the potential to improve
disease modeling and the generation of better sources of patient - specific specialized
cells suitable for replacement therapy,» said Plath.
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.
With our human gut - on - a-chip, we can not only culture the normal gut microbiome for extended times, but we can also analyze contributions of pathogens, immune
cells, and vascular and lymphatic endothelium, as well as
model specific
diseases to understand complex pathophysiological responses of the intestinal tract.»
Guo and his collaborators continue their studies by establishing additional mouse
models of leukemia that have been transplanted with patient
cells of relapsed and refractory
disease.
This proof - of - principle study shows «for the first time... that human iPS
cells can be used to
model a diverse range of inherited
diseases in adult
cells,» the authors wrote in their paper, published online in The Journal of Clinical Investigation August 25.
A new study has found that stem
cell therapy can reduce lung inflammation in an animal
model of chronic obstructive pulmonary
disease (COPD) and cystic fibrosis.
Qi concluded that the peptide reduced nerve
cell impairment caused by Huntington's
disease in the animal
model.
The research is also the first to demonstrate beneficial effects of UDCA on dopaminergic neurons, the nerve
cells affected in Parkinson's
disease, in a fly
model of Parkinson's
disease which carries the same genetic change as some patients with the condition.
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.
The study involved laboratory
cell lines of human leukemia and mouse
models of the
disease.
Realistic stem
cell therapies to replace
diseased or damaged tissue may still be years away, but researchers have uncovered a promising new use for these undifferentiated
cells: they can be programmed to become patient - specific laboratory
models of inherited liver
disease.
The UT Southwestern group had previously used CRISPR - Cas9, the original gene - editing system, to correct the Duchenne defect in a mouse
model of the
disease and in human
cells.
The study successfully countered harmful effects of mutant huntingtin and protected nerve
cells in several
models of Huntington's
disease.
The scientists then tested three new mTOR inhibitors currently under development (pp242, AZD8055 and INK128) in combination with the chemotherapies AraC, Etoposide and Cisplatin to see how they affected laboratory lines of leukemia
cells and mouse
models of the
disease.
«The compounds identified in this study, when administered orally, both reduced the inflammation that is a hallmark of multiple sclerosis and protected against the nerve
cell damage seen in mouse
models of the
disease,» said Jeffery Haines, PhD, a post-doctoral fellow at Mount Sinai and the study's lead author.
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.
Using animal
models of precancerous polyps in the bowel, Chung and his team determined that certain types of immune
cells within a chronically inflamed intestine can become rewired, causing them — paradoxically — to contribute to
disease development rather than protect against it.
In addition to helping understand
disease by providing more powerful study
models, «what this technology would allow you to do is reprogram a skin
cell, for example, from a Parkinson's patient... into a pluripotent
cell and then in a petri dish redirect that
cell into... a neuron» to treat that patient.
«The study of this type of tumours has been problematic up to now due to the lack of
cell models and the appropriate animal
models,» says CNIC researcher Juan Carlos Ramírez, who adds that the difficulty of generating these chromosomal translocations had limited the availability of
cells with this mark of the
disease.
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.
Researchers at University of California San Diego School of Medicine report that a single infusion of wildtype hematopoietic stem and progenitor
cells (HSPCs) into a mouse
model of Friedreich's ataxia (FA) measurably halted cellular damage caused by the degenerative
disease.
This accomplishment opens the door for
disease modeling and drug screening and brings personalized
cell therapy a step closer for patients with diabetes.
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.
Induced pluripotent stem
cells (iPSCs)-- adult
cells reprogrammed back to an embryonic stem
cell - like state — may better
model the genetic contributions to each patient's particular
disease.
Additionally, work in a mouse
model revealed similar
cells, indicating that the progenitors are conserved from mouse to human, and therefore, they must be «important
cells with promising potential for
cell therapy in treating liver
disease,» explained Dr. Gouon - Evans.
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.
Still, he says, other groups have arrived at opposing results regarding the role of T
cells in the
disease, in part, perhaps, because their animal
models aren't the same.
As a result, they can now test drugs directly on living
cells afflicted with the
disease, and they can accurately
model how it progresses.
More recently, researchers have induced stem
cells from
diseased human somatic
cells, which may serve as new
model systems for various illnesses.
«New gene editing technique turns human pluripotent stem
cells into a
model system for polycystic kidney
disease.»
Now, a team of scientists at the Icahn School of Medicine at Mount Sinai have developed the Just EGFP Death - Inducing T -
cell, or JEDI T -
cells, which enable the visualization of T -
cell antigens, allowing researchers to study T -
cell interactions with different
cell types,
model disease states, and finally determine the functions of otherwise poorly characterized
cell populations.
Stem
cell models, derived from healthy or
diseased cells, are also being developed for use in drug efficacy and toxicity testing.
This is particularly important as it indicates the method can be used in a variety of
cell types to create
disease models and contribute to the discovery of new
disease - specific therapeutics.
This was observed in human ovarian cancer
cells grown in culture, and then in mouse
models of the
disease.
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.
More work is needed to determine whether the findings on rapamycin hold true in animal
models of Leigh syndrome and other neurodegenerative
diseases, and to ascertain how exactly rapamycin is altering the metabolism of the
cells.
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.»
The
disease model, described in a new study by a UC San Francisco - led team, involves taking skin
cells from patients with the bone
disease, reprogramming them in a lab dish to their embryonic state, and deriving stem
cells from them.
The human
cell - based
disease model is expected to lead to a better understanding of these disorders and other illnesses, Hsiao said.