Editing scrambled
genes in human stem cells may help realize the promise of combined stem cell - gene therapy
To do this, they created a cellular model of Werner syndrome by using a cutting - edge gene - editing technology to delete WRN
gene in human stem cells.
Not exact matches
Then they would inject
human stem cells into the pig embryo
in hopes that the
human stem cells would bridge the gaps of the missing pancreas
gene and form a
human pancreas.
A team of researchers at the Stanford University School of Medicine has used a
gene - editing tool known as CRISPR to repair the
gene that causes sickle cell disease
in human stem cells, which they say is a key step toward developing a
gene therapy for the disorder.
BARE BONES Differences between the skeletons of modern
humans (back) and Neandertals (front) may
stem from the way the groups use some
genes involved
in bone growth.
«What's cool about this group of
genes is that they also get turned on
in human melanoma,» says Zon, who is also a member of the Harvard
Stem Cell Institute and a Howard Hughes Medical Institute investigator.
- Our results provide new insights into the mechanisms of how POLR3G
gene regulates
stem cell state, which
in turn sheds light on the complex mechanisms with which
human embryonic
stem cells both self - renew and maintain the ability to differentiate.
Researchers from Turku, Finland, have discovered new information about the mechanisms which maintain
gene activity
in human embryonic
stem cells.
Using single cell RNA sequencing analysis, the Cairns lab profiled cells individually, establishing the
gene expression profile
in human sperm
stem cells.
This image shows an expression of the
stem cell
gene Musashi
in human pancreatic cancer.
Recent research by neuroscientist Fred Gage and colleagues at the University of California (UC), San Diego, has shown that one of the most common types of jumping
gene in people, called L1, is particularly abundant
in human stem cells
in the brain that ultimately differentiate into neurons and plays an important role
in regulating neuronal development and proliferation.
Unlike OCT4, these
genes can only be studied
in human embryos because they are not expressed the same way, or at all,
in mouse embryos or immortalized lines of
human stem cells, says her colleague Robin Lovell - Badge, also at the Crick Institute.
The results, published
in the current issue of
Human Molecular Genetics, open the door for pursuing
gene editing
in nonhuman primates as models for new therapies, including pharmacological,
gene - and
stem cell - based therapies, said Keith Latham, MSU animal science professor and lead author of the study.
Asuri, Prashanth, et al. «Directed evolution of adeno - associated virus for enhanced
gene delivery and
gene targeting
in human pluripotent
stem cells.»
Since her recruitment at SR - Tiget as an independent Project Leader
in 2012, promoted to Group Leader
in 2016, her team investigates the molecular mechanisms of host - vector interplay during
gene transfer
in human hematopoietic
stem cells (HSC).
Neuronal developmental
gene and miRNA signatures induced by histone deacetylase inhibitors
in human embryonic
stem cells.
Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, Ando D, Urnov FD, Galli C, Gregory PD, Holmes MC, Naldini L.
Gene editing
in human stem cells using zinc finger nucleases and integrase - defective lentiviral vector delivery.
Thus, neural derivatives of disease - specific
human pluripotent
stem cells constitute a relevant biological resource for exploring the impact of adult - onset HD mutations of the HTT
gene on the division of neural progenitors, with potential applications
in HD drug discovery targeting HTT - dynein - p150Glued complex interactions.
Dr. Sonntag studies this concept on the molecular and cellular level using a translational research approach that integrates the analysis of
human material, such as postmortem brains, primary cell systems, and neural cell populations generated from patients» - or healthy individuals» - derived induced pluripotent
stem cells (iPSC), or induced neurons (iNs),
in combination with molecular, biochemistry, and lentivirus - mediated
gene - engineering technologies.
In an application for a prestigious «Pioneer Award» from NIH this year, he proposed injecting
human pluripotent
stem cells into pig embryos whose
genes for specific organs had been knocked out.
Importantly, 25 of the 115 transcripts, shared by EFTF - expressing pluripotent cells and the EF, encode for 15
genes that are both expressed
in retinal
stem / progenitor cells and required for normal eye formation
in frogs, fish, mice, or
humans (Figure 1C; Table S1).
Highly Synchronized Expression of Lineage - Specific
Genes during
In Vitro Hepatic Differentiation of
Human Pluripotent
Stem Cell Lines.
Induction of
stem cell
gene expression
in adult
human fibroblasts without transgenes.
These cells, first described
in humans in November 2007, are produced by inserting certain
stem - cell - associated
genes into regular adult cells (like skin cells).
Figure 2: Abnormal accumulation of the FGFR - TACC fusion protein (red)
in glioblastoma
stem cells isolated from a primary
human glioblastoma with fused FGFR - TACC
genes.
«Discovery of a
gene that could convert
human embryonic
stem cells into myocardial cells would be golden,» said Didier Stainier, PhD, UCSF assistant professor of biochemistry and biophysics, the senior author of the UCSF study and a pioneer
in the study of heart development
in the transparent zebrafish embryo.
HSV - sr39TK positron emission tomography and suicide
gene elimination of
human hematopoietic
stem cells and their progeny
in humanized mice.
High Throughput Screening for Inhibitors of Rest
in Neural Derivatives of
Human Embryonic
Stem Cells Reveals a Chemical Compound that Promotes Expression of Neuronal
Genes
2D nanoparticles known as nanosilicates can be used to grow bone and cartilage tissue from
human mesenchymal
stem cells, according to new
gene sequencing experiments by researchers at Texas A&M University
in the US.
In Lifemap, the HaemAtlas dataset provides
gene expression for adult
human B cells, helper T cells, natural killer cells, granulocytes, and monocytes while the Hematopoietic Fingerprints dataset provides
gene expression information for postnatal
stem cells and their progeny.
Human Stem Cell Institute (MICEX: ISKJ, Russia) HSCI just got an authorization for sales of the first
gene therapy drug
in Russia.
The molecules central to this balancing act, H3K4me3 and H3K27me3, are among the so - called epigenetic modifications that influence the activity patterns of
genes in both
human embryonic
stem (ES) cells and mature
human adult cells.
Human pluripotent stem cells (hPSCs), including human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs), are known to be vulnerable to apoptosis upon various technical manipulation, such as single cell dissociation, freezing and thawing, etc., which hinder their use for clonal isolation in gene transfer, differentiation and FACS cell sor
Human pluripotent
stem cells (hPSCs), including
human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs), are known to be vulnerable to apoptosis upon various technical manipulation, such as single cell dissociation, freezing and thawing, etc., which hinder their use for clonal isolation in gene transfer, differentiation and FACS cell sor
human embryonic
stem cells (hESCs) and
human induced pluripotent stem cells (hiPSCs), are known to be vulnerable to apoptosis upon various technical manipulation, such as single cell dissociation, freezing and thawing, etc., which hinder their use for clonal isolation in gene transfer, differentiation and FACS cell sor
human induced pluripotent
stem cells (hiPSCs), are known to be vulnerable to apoptosis upon various technical manipulation, such as single cell dissociation, freezing and thawing, etc., which hinder their use for clonal isolation
in gene transfer, differentiation and FACS cell sorting.
Stem cell researchers from UCLA used a high resolution technique to examine the genome, or total DNA content, of a pair of human embryonic stem cell lines and found that while both lines could form neurons, the lines had differences in the numbers of certain genes that could control such things as individual traits and disease susceptibil
Stem cell researchers from UCLA used a high resolution technique to examine the genome, or total DNA content, of a pair of
human embryonic
stem cell lines and found that while both lines could form neurons, the lines had differences in the numbers of certain genes that could control such things as individual traits and disease susceptibil
stem cell lines and found that while both lines could form neurons, the lines had differences
in the numbers of certain
genes that could control such things as individual traits and disease susceptibility.
And CRISPR has completely and utterly transformed the field, as we can do rapid
gene targeting now
in primary
human neural
stem cells.»
In findings appearing online today in Cell Stem Cell, researchers in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, describe how they transferred a single gene called Sox2 into both mouse and human skin cell
In findings appearing online today
in Cell Stem Cell, researchers in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, describe how they transferred a single gene called Sox2 into both mouse and human skin cell
in Cell
Stem Cell, researchers
in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, describe how they transferred a single gene called Sox2 into both mouse and human skin cell
in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, describe how they transferred a single
gene called Sox2 into both mouse and
human skin cells.
A dual yeast and
human stem cell discovery platform for Parkinson's disease: Investigations
in simple baker's yeast cells brought to light abnormalities
in Parkinson's patient neurons and identified
genes and small molecules that correct them.
A
human embryonic
stem cell is reined
in — prevented from giving up its unique characteristics of self - renewal and pluripotency — by the presence of a protein modification that stifles any
genes that would prematurely instruct the cell to develop into heart or other specialized tissue.
Targeted
Gene Addition to a Safe Harbor locus
in human CD34 + Hematopoietic
Stem Cells for Correction of X-linked Chronic Granulomatous Disease.
Recent developments
in StemBase: a tool to study
gene expression
in human and murine
stem cells.
«Basically, this study shows that the genetic makeup of individual
human embryonic
stem cell lines is unique
in the numbers of copies of certain
genes that may control traits and things like disease susceptibility,» said Teitell, who also is an associate professor of pathology and laboratory medicine and a researcher at UCLA's Jonsson Comprehensive Cancer Center.
«
Human Embryonic
Stem Cell Lines Differ
In Genes That Could Control Disease Susceptibility.»
However, advancements
in engineering
human stem cells are now allowing researchers to grow mini-organs
in labs, and
gene - editing tools can be used to insert specific mutations into these cells.
Researchers describe how a bacterial infection - based protein delivery strategy can mediate effective and safe
gene editing
in human pluripotent
stem cells.
«Having a very efficient and practical way of generating patient - specific
stem cells, which unlike
human embryonic
stem cells, wouldn't be rejected by the patient's immune system after transplantation brings us a step closer to the clinical application of
stem cell therapy,» says Belmonte, PhD., a professor
in the
Gene Expression Laboratory and director of the Center of Regenerative Medicine
in Barcelona, Spain.
A number of recent articles, however, have reported that hiPSCs are,
in fact, notably distinct from
human embryonic
stem cells
in terms of their
gene expression, epigenetic profile, proliferative capacity and the susceptibility of their differentiated progeny to cellular senescence and apoptosis [3 — 6].
Both mouse and
human iPSCs are similar to embryonic
stem cells (ESCs) with respect to their morphology, cell behavior,
gene expression, epigenetic status and differentiation potential both
in culture and
in vivo.
Created
in 2005 through a collaboration between Inserm — National Institute of Health and Medical Research — and AFM - Telethon — French Association against Myopathies — I -
Stem is the largest French laboratory for research and development dedicated to human pluripotent stem cells, of embryonic origin or obtained by reprogramming g
Stem is the largest French laboratory for research and development dedicated to
human pluripotent
stem cells, of embryonic origin or obtained by reprogramming g
stem cells, of embryonic origin or obtained by reprogramming
gene.
Abbreviations: Aβ, amyloid β - peptide; AD, Alzheimer's disease; ALS, amyotrophic lateral sclerosis; Ambra1, activating molecule
in Beclin -1-regulated autophagy; AMPK, AMP - activated protein kinase; APP, amyloid precursor protein; AR, androgen receptor; Atg, autophagy - related; AV, autophagic vacuole; Bcl, B - cell lymphoma; BH3, Bcl - 2 homology 3; CaMKKβ, Ca2 + - dependent protein kinase kinase β; CHMP2B, charged multivesicular body protein 2B; CMA, chaperone - mediated autophagy; 2 ′ 5 ′ ddA, 2 ′, 5 ′ - dideoxyadenosine; deptor, DEP - domain containing mTOR - interacting protein; DRPLA, dentatorubral pallidoluysian atrophy; 4E - BP1, translation initiation factor 4E - binding protein - 1; Epac, exchange protein directly activated by cAMP; ER, endoplasmic reticulum; ERK1 / 2, extracellular - signal - regulated kinase 1/2; ESCRT, endosomal sorting complex required for transport; FAD, familial AD; FDA, U.S. Food and Drug Administration; FIP200, focal adhesion kinase family - interacting protein of 200 kDa; FoxO3, forkhead box O3; FTD, frontotemporal dementia; FTD3, FTD linked to chromosome 3; GAP, GTPase - activating protein; GR, guanidine retinoid; GSK3, glycogen synthase kinase 3; HD, Huntington's disease; hiPSC,
human induced pluripotent
stem cell; hVps, mammalian vacuolar protein sorting homologue; IKK, inhibitor of nuclear factor κB kinase; IMPase, inositol monophosphatase; IP3R,
Ins (1,4,5) P3 receptor; I1R, imidazoline - 1 receptor; JNK1, c - Jun N - terminal kinase 1; LC3, light chain 3; LD, Lafora disease; L - NAME, NG - nitro - L - arginine methyl ester; LRRK2, leucine - rich repeat kinase 2; MIPS, myo - inositol -1-phosphate synthase; mLST8, mammalian lethal with SEC13 protein 8; MND, motor neuron disease; mTOR, mammalian target of rapamycin; mTORC, mTOR complex; MVB, multivesicular body; NAC, N - acetylcysteine; NBR1, neighbour of BRCA1
gene 1; NOS, nitric oxide synthase; p70S6K, ribosomal protein S6 kinase - 1; PD, Parkinson's disease; PDK1, phosphoinositide - dependent kinase 1; PE, phosphatidylethanolamine; PI3K, phosphoinositide 3 - kinase; PI3KC1a, class Ia PI3K; PI3KC3, class III PI3K; PI3KK, PI3K - related protein kinase; PINK1, PTEN - induced kinase 1; PKA, protein kinase A; PLC, phospholipase C; polyQ, polyglutamine; PS, presenilin; PTEN, phosphatase and tensin homologue deleted from chromosome 10; Rag, Ras - related GTP - binding protein; raptor, regulatory - associated protein of mTOR; Rheb, Ras homologue enriched
in brain; rictor, rapamycin - insensitive companion of mTOR; SBMA, spinobulbar muscular atrophy; SCA, spinocerebellar ataxia; SLC, solute carrier; SMER, small - molecule enhancer of rapamycin; SMIR, small - molecule inhibitor of rapamycin; SNARE, N - ethylmaleimide - sensitive factor - attachment protein receptor; SOD1, copper / zinc superoxide dismutase 1; TFEB, transcription factor EB; TOR, target of rapamycin; TSC, tuberous sclerosis complex; ULK1, UNC -51-like kinase 1; UVRAG, UV irradiation resistance - associated
gene; VAMP, vesicle - associated membrane protein; v - ATPase, vacuolar H + - ATPase; Vps, vacuolar protein sorting
That's right, scientists are people too and sometimes they get carried away by their enthusiasms as Emily Yoffe notes
in her excellent Slate essay, The Medical Revolution; Where are the cures promised by
stem cells,
gene therapy, and the
human genome?