Not exact matches
Gene therapy delivered to a
specific part
of the brain reverses symptoms
of depression in a mouse
model of the
disease — potentially laying the groundwork for a new approach to treating severe cases
of human depression in which drugs are ineffective.
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.»
We demonstrated that DENV serotype 2 (DENV2)--
specific human monoclonal antibody (HMAb) 2D22 is therapeutic in a mouse
model of antibody - enhanced severe dengue
disease.
The researchers hope their new cell lines will be a useful resource for studying the cellular and molecular intricacies
of Huntington's further, and suggest they may provide a
model for examining other
diseases of the brain that are
specific to
humans.
The researchers» strategy — generating
disease -
specific nerve cells, identifying a causative gene for developmental defects, validating the gene -
specific defect in animal
models, and then investigating interactions with other genes both in animal
models and in
humans — represents a promising new approach for understanding the mechanisms underlying some
of the most intractable psychiatric illnesses.
However, Takebe's liver bud has the advantage
of being grown from iPS cells, rather than, for example, the primary
human hepatocytes used in Bhatia's graft, which could make it useful in
modelling rare
diseases or examining the
specific genetic backgrounds
of the iPS cell donors.
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.
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.
Furthermore, new genome - editing technologies such as CRISPR / Cas9 now enable the efficient derivation
of precision
disease models incorporating patient -
specific genetic variants as a means
of recapitulating essential aspects
of human disease in mouse and other
model organisms.
In recent years, researchers have developed so - called «senolytic» drugs that wipe out senescent cells in aging mice and mouse
models of age - related
disease, exploiting the high dependence
of these cells on
specific biochemical survival pathways.9, 10 In these studies, senolytic drugs have restored exercise capacity9 and formation
of new blood and immune precursor cells11 in aging mice to near youthful norms, and prevented or treated mouse
models of diseases of aging like osteoarthritis, 12 fibrotic lung
disease, 13 hair loss, 14 atherosclerosis, 15,16 and age - related
diseases of the heart itself.9 UNITY Biotechnology is leading a growing charge toward the clinic, with
human clinical trials expected to begin in 2019.
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.
In addition, projects to perform genetic interaction screens on
disease genes in
model organisms (yeast, worm, fly, fish) will not be considered, unless the project includes substantive
specific aims that investigate the
disease relevance
of any new genes discovered in
human or mammalian
model systems.
Working with Dr. Weiskopf, we established a
model of human dengue
disease using HLA transgenic mouse strains, and characterized
human dengue -
specific CD8 + and CD4 + T - cell responses in natural infection as well as following vaccination.
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.
The technology also has the potential to knock out
specific genes to create pig
models of human diseases.
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.