• Vineti, a San Francisco -
based cell and gene therapy software and analytics company, raised $ 13.75 million in Series A funding.
Not exact matches
US -
based pharmaceutical company Gilead Sciences entered the chimeric antigen receptor (CAR) T -
cell therapy business through its acquisition of Kite Pharma,
and Australian biopharma company CSL Behring acquired US -
based Calimmune, a company that develops clinical - stage
gene therapy solutions.
«We are very excited about the prospect of using these more physiologically relevant
cell -
based models for
gene and small molecule drug discoveries,» says Dr. Andrew Liu, Assistant Professor of Biology at the University of Memphis.
In these
and other inherited diseases, 10 to 15 percent of the single -
base pair mutations that cause the disease create a misplaced, premature «stop» codon in the middle of the
gene — causing the machinery of the
cell to prematurely halt synthesis of the protein, which destroys its ability to function.
Base oxidation regulates gene activity In cooperation with colleagues at LMU, as well as researchers based in Berlin, Basel and Utrecht, Carell and his group have now shown, for the first time, that a standard base other than cytosine is also modified in embryonic stem cells of m
Base oxidation regulates
gene activity In cooperation with colleagues at LMU, as well as researchers
based in Berlin, Basel
and Utrecht, Carell
and his group have now shown, for the first time, that a standard
base other than cytosine is also modified in embryonic stem cells of m
base other than cytosine is also modified in embryonic stem
cells of mice.
Biochemist Radhey Gupta of McMaster University in Canada proposes that a bacterium
and an archaean fused to form the first eukaryote,
based on his analysis of a pair of slow - changing
genes found in what may be one of the oldest
cells with a nucleus, Giardia lamblia.
The process, reported in Human Reproduction, utilizes DNA fingerprinting (an assessment of active
genes in a given
cell) to boost the success rate of IVF
and lower the chances of risky multiple births by identifying which of several five - day - old embryos are most likely to result in pregnancy The new method, which will replace unproved alternatives such as choosing embryos
based on their shape, is likely to up the success of women becoming pregnant
and lower their chances of having multiple births.
But where atomic research had a single, explosive debut,
gene -
and cell -
based treatments have emerged in fits, after many false starts.
Biomolecular model
based on the
gene expression data analyses support the reduction of glucose molecules (blue gradient)
and acid buildup (gold gradient) proposed to occur in the boundary layer around the
cell.
Based on studies of
gene activity
and immune
cells, he found that these mammals have «domesticated» implantation's inflammatory response.
The resulting «map» of
gene - drug interactions allowed the researchers to accurately predict the responses of multiple human cancer
cell lines to different chemotherapy agents
based on the
cell lines» genetic profiles
and also revealed new genetic factors that appear to determine the response of breast
and ovarian tumor
cells to common classes of chemotherapy treatment.
Since the discovery of the genetic
basis for cystic fibrosis in 1989, scientists have developed a variety of viral
and non-viral vector systems for delivering a corrected CFTR
gene back into lung
cells.
In this new study, researchers employed a
cell -
based reporter system to screen a collection of over 1000
gene - regulatory factors called microRNAs for the ability to influence this transition
and identified some with particularly potent effects.
That DNA includes slightly less than 21,000 protein - coding
genes (some researchers once estimated we had more than 100,000 such
genes); «
genes» for 8800 small RNA molecules
and 9600 long noncoding RNA molecules, each of which is at least 200
bases long;
and 11,224 stretches of DNA that are classified as pseudogenes, «dead»
genes now known to really be active in some
cell types or individuals.
In a series of
cell -
based studies, they showed that it both blocked a family of iron - containing, oxygen - sensing enzymes called hypoxia - inducible factor prolyl - hydroxylases (HIF - PHD)
and, critically, protected neurons by activating
genes that protect them from oxidative stress.
Boston Children's Hospital has offered non-exclusive licenses to for - profit entities on a patent developed by Orkin's laboratory regarding BCL11A, a genetic switch regulating hemoglobin production that is expected to form the
basis of clinical trials for
gene therapy
and gene editing for sickle
cell disease
and thalassemia.
After preclinical studies, a
gene therapy trial for SCID - X1 was initiated,
based on the use of complementary DNA containing a defective γc Moloney retrovirus — derived vector
and ex vivo infection of CD34 +
cells.
«Understanding how Cas9 is able to locate specific 20 -
base - pair target sequences within genomes that are millions to billions of
base pairs long may enable improvements to
gene targeting
and genome editing efforts in bacteria
and other types of
cells,» says Doudna who holds joint appointments with Berkeley Lab's Physical Biosciences Division
and UC Berkeley's Department of Molecular
and Cell Biology
and Department of Chemistry,
and is also an investigator with the Howard Hughes Medical Institute (HHMI).
It is the first time that a
gene therapy
based on genetically modified stem
cells is tested in a multicenter, international clinical trial that shows a reproducible
and robust therapeutic effect in different centers
and different countries.
Still, scientists have been able to piece together some viral histories,
based on the fact that the
genes of many viruses — such as those that cause herpes
and mono — seem to share some properties with
cells» own
genes.
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.
Most notably, the studies are
based on transient transfection of wild - type
and mutant
gene constructs into cultured
cells or into zebrafish embryos
and are unlikely to reproduce the specific mutant
gene dose that is present in heterozygous mutant
cells in FOP patients.
Based on analyses of over 600 drug
and breast cancer
cell pairings, researchers showed that, for some
cells, drug exposure can cause significant changes in
gene expression — indicating the successful action of a drug on its target — without affecting
cell growth or survival.
Exon -
Based Transcriptome Profiling Reveals
Genes That Have Prognostic Impact on the Survival of Young High Risk Diffuse Large B -
Cell / Follicular Grade 3 Lymphoma Patients Treated with Dose - Dense Chemoimmuno - therapy
and CNS Prophylaxis.
Use of exon -
based transcriptome profiling to identify novel signaling pathways
and survival - associated
genes in diffuse large B -
cell lymphoma.
The advent of molecular cloning, DNA sequencing
and the many tools of molecular genetics
and cell biology has given us sufficient knowledge of the
basis for disease
and the
genes to target, but what has limited the application of
gene therapy has been efficient
gene delivery systems.
Furthermore, high - resolution analysis of cytosine methylation in primary
and transformed
cells has found less aberrant methylation of CpG island promoters in transformed
cells than had been previously hypothesized
based on candidate
gene studies [58].
The mouse embryonic stem
cell knockout resource provides a
basis for the characterization of relationships between
genes and phenotypes.
Martonvásár Development of new generic plant genotypes to satisfy the needs of the future,
based on an internationally acknowledged plant
gene pool,
and using up - to - date genetic, physiological,
cell and reproduction biological, functional genomic, biotechnological, plant breeding
and crop production methods.
A physician with a longstanding interest in
gene therapy, Dr. High was formerly a Professor at the Perelman School of Medicine of the University of Pennsylvania, an Investigator of the Howard Hughes Medical Institute,
and the Founding Director of the Center for Cellular
and Molecular Therapeutics at The Children's Hospital of Philadelphia, a Center focused on developing novel
cell and gene -
based therapies for genetic disease.
Topics for the scientific sessions are: Single
cell genomics
and gene expression Genetic interactions RNAi
and somatic
cell genetics Protein - DNA interactions Cancer The meeting also highlights existing opportunities for academic
and industrial researchers to access automated
cell -
based and biochemical technologies
based at the Karolinska High Throughput Center; home to one of the most sophisticated, state - of - the - art, ultra-high performance liquid handling
and analysis platforms in Europe.
This work is one
basis for the use in the scientific community of transgenes as a major technique to study
gene regulation in normal
and malignant
cells.
Remember that a
gene is a set of instructions, spelled using the chemical «letters» A, C, G
and T. Each
gene has a different sequence of letters,
and cells use zinc - containing proteins to help control individual
genes based on their specific sequence of letters.
Based on data from Robinson
and other people who had similar experiences, Newell has been able to identify a pattern of
genes turned on in their immune
cells that may predict whether someone could be able to become «tolerant.»
Caribou recently cofounded Intellia Therapeutics for the development of human
gene and cell therapies
based on their proprietary CRISPR - Cas9 technology platform.
A novel way of approaching protection -
based therapeutics for glaucoma should derive from evidence accumulating over two decades in stroke
and cardiac arrest: That simultaneously activating a variety of self - defense responses in
cells with stressful «conditioning» stimuli induces the expression of a host of
genes that promote
cell survival.
However, when forced to express the eye field transcription factor (EFTF)
genes, the
cells differentiate into all seven retinal
cell classes
and eventually organize themselves into a functioning eye that can detect light
and guide tadpoles in a vision -
based behavior.
The NIH Common Fund's Library of Integrated Network -
based Cellular Signatures (LINCS) program aims to create a network -
based understanding of biology by cataloging changes in
gene expression
and other cellular processes that occur when
cells are exposed to a variety of perturbing agents.
Vaxwave ® is
based on lymphocytic choriomeningitis virus (LCMV)
and in this vector the
gene encoding the LCMV envelope protein, normally responsible for virus entry into target
cells, has been deleted
and replaced with a target
gene of interest.
Having spent hundreds of millions of dollars recently in its
gene -
based cell therapies for cancer, Strimvelis
and its other
gene therapies for rare conditions sit better with Orchard Therapeutics.
The next step
based on these novel head
and neck cancer discoveries, the scientists agree, is to tease out how the
genes function in normal
cells, whether they form the lining of the larynx, pharynx, or another anatomical site affected by head
and neck cancer.
These features enable gesicles to knock out
genes with high efficiency
and in a broader range of
cell types than plasmid -
based delivery methods.
In MMEJ pathway, we achieve efficient
gene disruption in human
cell lines
and animals by developing a computer program that assists the choice of nuclease target sites
based on microhomology prediction.
Using genetic
and epigenetic analyses coupled with powerful perturbation technologies to test
gene functions in human
cells and mouse models, we hope to identify the critical drivers of this disease
and the
basis for therapeutic responses.
Determining how ES
cell genes are modified by these epigenetic markers may explain these
cells» unique characteristics, said the scientists, who are
based at the Genome Institute of Singapore (GIS)
and the Bioprocessing Technology Institute (BTI), both under the Agency for Science, Technology
and Research (A * STAR), as well as at the National University of Singapore (NUS).
He was a Helen Hay Whitney fellow with Nobel Laureate Dr. Phillip Sharp at the Center for Cancer Research at the Massachusetts Institute of Technology (M.I.T.), where he developed
gene transfer technologies
based on
gene amplification
and expression in mammalian
cells.
iTeos focuses upon selected key suppressive mechanisms of immunity in cancer
based on
gene expression, association with immune
cell subsets, protein expression in human tumors
and biological validation.
We confirmed the purity of ZP - free oocytes
and cumulus
cell samples
based on the exclusive expression of known oocyte - specific
and cumulus
cell - specific
genes.
Researchers describe how a bacterial infection -
based protein delivery strategy can mediate effective
and safe
gene editing in human pluripotent stem
cells.
One early form of CRISPR -
based gene therapy could involve editing the
genes responsible for blood disorders like sickle -
cell anemia in bone marrow
cells, growing them into mature blood
cells and injecting them back into patients.