«The AAV - DJ / 8 subtype combines multiple serotypes, giving us a broader range of
cell targets for the delivery of our CRISPR / Cas9 system,» Dr. Hu said.
Not exact matches
Gene therapy,
for example, frequently involves using a virus to deliver a gene to the
cells that scientists are
targeting.
The technology's possibilities are staggering — in theory, allowing medical scientists to do everything from cure genetic disorders like sickle
cell disease to identify gene
targets for combating HIV.
I won't reveal yet who my favorites are, but I will say that these young scientist - founders came up with very creative solutions
for preventing infections in some common surgeries, tackling resistance in
targeted antibody drugs, improving gene vectors
for cell therapies, helping the vision - impaired «see» faces and better read their environments, imaging hard - to - see spots in the lungs and other organs, improving genetic risk analysis, and expediting the logistical operations of hospitals.
Most of the action is coordinated by white blood
cells, which direct the lines of attack like football coaches, churning out antibodies that
target the opponent
for destruction.
One recent example: Stemcentrx, which rode an unproven approach
targeting cancer stem
cells to a summertime financing round of nearly $ 250 million and a $ 5 billion valuation, the most
for a venture - backed drug maker and second to Theranos in health care.
For example, CRAC channels in T
cells have been clinically validated as important drug
targets through human mutations and the use of calcineurin inhibitors that act downstream from CRAC channels.
Cambridge, MA — February 6, 2017 — Aura Biosciences, a biotechnology company developing a new class of therapies to
target and selectively destroy cancer
cells using viral nanoparticle conjugates, announced today that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug application (IND)
for the company's lead program, light - activated AU - 011 in ocular melanoma (OM).
His work indicates that this
cell surface marker could serve as a
target for a novel brain cancer vaccine or T -
cell therapies engineered to recognize and kill tumors carrying that neoantigen.
Cambridge, MA — March 30, 2017 — Aura Biosciences, a biotechnology company developing a new class of therapies to
target and selectively destroy cancer
cells using viral nanoparticle conjugates, announced today that it has enrolled and dosed the first patient in its Phase 1b clinical trial of light - activated AU - 011, an investigational, first - in - class
targeted therapy in development
for the treatment of ocular melanoma, a rare and life - threatening disease.
The Japanese government has set a
target price of ¥ 2.2 million — around US $ 18,000 —
for fuel -
cell vehicles by 2025.
What Stephen Hawking Missed: Small Biotechs Developing Promising
Cell Therapies for Devastating Disease Source: Streetwise Reports (5/2/18) In the second of a two - part series exploring the disruptive cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies targeting big - ticket indications and their potential to drive blockbuster value for both patients and invest
Cell Therapies
for Devastating Disease Source: Streetwise Reports (5/2/18) In the second of a two - part series exploring the disruptive
cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies targeting big - ticket indications and their potential to drive blockbuster value for both patients and invest
cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies
targeting big - ticket indications and their potential to drive blockbuster value
for both patients and investors.
At the end of September, the European Commission approved a marketing application
for Lutathera, a radioactive molecule that
targets specific receptors found on the surface of neuroendocrine tumor
cells.
A 17 - year - old California girl won $ 100,000 in a national science competition
for her creation of a nanoparticle that can
target and kill cancer
cells.
Eilersen has announced it is releasing a new series of digital load
cells in a hygienic (aseptic) design
for dynamic and process weighing, filling, packaging, and general weighing applications
targeted primarily at food and pharmaceutical customers.
The load
cells transmit weight gain information to the controller, which steps down the feed rate to a trickle and stops the conveyor immediately before reaching the
target weight to compensate
for material in flight.
These are the main
target cells for HIV.
CoolScultping,
for instance, uses an applicator to
target, cool and eliminate fat
cells without damaging your skin.
And it CAN be a good
for political organizing, if your
targets are in populations with high
cell - phone and / or Twitter usage (
for instance, younger — and plugged - in — urban black and latino voters).
Because the genes that are massively amplified in the neochromosomes are essential
for the
cell's survival, drugs that turn those off are an obvious
target for research.
«xIAP has other roles in the
cell, such as regulation of
cell death, so it is a tricky
target for treating inflammatory diseases.
A research team at the University of California, Riverside has discovered a way
for chemotherapy drug paclitaxel to
target migrating, or circulating, cancer
cells, which are responsible
for the development of tumor metastases.
These results significantly advance understanding of how cancer
cells are made to move during metastasis and may provide more precise
targets for drugs to stop cancer metastasis in patients where there are oncogenic mutations.
Still, «
for something that really tends to interfere with your everyday life, I would think that a treatment that
targets just these
cells could be appropriate,» she said.
In SIF - seq, hundreds or thousands of DNA fragments to be tested
for enhancer activity are coupled to a reporter gene and
targeted into a single, reproducible site in embryonic
cell genomes.
The autophagy machinery in yeast
cells is similar to that in human
cells, and Ohsumi's work, which began in the 1990s, gives scientists new
targets for possible treatments.
Nevertheless, our results show that RNA modules have great potential in biotechnology
for controlling processes in bacterial
cells in a
targeted manner.»
The new technology, described in the journal Molecular
Cell, offers a new way to seek drug
targets for many diseases, not just Parkinson's, says Timothy Lu, an MIT associate professor of electrical engineering and computer science and of biological engineering.
«These predictive algorithms seem to do a good job when CRISPR is performed in
cells or tissues in a dish, but whole genome sequencing has not been employed to look
for all off -
target effects in living animals,» says co-author Alexander Bassuk, MD, PhD, professor of pediatrics at the University of Iowa.
Similarly, it may be possible to identify and validate new
targets for drugs that would selectively kill tumor
cells with a particular molecular context.
«Several major advances in recent years have been good news
for multiple myeloma patients, but those new drugs only
target terminally differentiated cancer
cells and thus can only reduce the bulk of the tumor,» said Jamieson, who is also deputy director of the Sanford Stem
Cell Clinical Center, director of the CIRM Alpha Stem Cell Clinic at UC San Diego and director of stem cell research at Moores Cancer Center at UC San Diego Hea
Cell Clinical Center, director of the CIRM Alpha Stem
Cell Clinic at UC San Diego and director of stem cell research at Moores Cancer Center at UC San Diego Hea
Cell Clinic at UC San Diego and director of stem
cell research at Moores Cancer Center at UC San Diego Hea
cell research at Moores Cancer Center at UC San Diego Health.
If it does, he will then study BCCA chemical networks in living
cells to determine promising
targets for new drugs.
Carlo Croce, a cancer researcher at Ohio State University in Columbus, and his colleagues created a diagram of interacting miRNAs
for normal body
cells by connecting them according to which genes they
target and the function of those genes, in a way similar to analyses of human social networks.
To hunt
for drugs that
target these
cells, Piyush Gupta, a molecular biologist at the Broad Institute at the Massachusetts Institute of Technology, and colleagues genetically engineered ordinary human
cells so that they acquired some of the properties of cancer stem
cells, including being impervious to chemotherapy.
«The paper shows that the timing of development is important
for the mechanism that a
cell uses to find a
target,» he says.
But while Venter's synthetic genome will be housed within an existing bacterial
cell, other scientists are aiming
for the even more ambitious
target of building an entire living
cell from the basic chemical ingredients.
Yang said the study not only indicated which genes are affected by traumatic brain injury and linked to serious disease, but also might point to the genes that govern metabolism,
cell communication and inflammation — which might make them the best
targets for new treatments
for brain disorders.
Dr. McCabe said nanoparticles are a leading - edge technology also being studied
for delivery of drugs
for other conditions, such as cancer, heart disease, and bacterial infections, in order to
target specific
cells to reduce toxicity and side effects of those medications and to make them more effective.
Our work suggests that
targeting a specific stem
cell phenotype,
for example with immunotherapies, could be beneficial in patients with oligodendroglioma.»
In this study, the SIgN team discovered
for the first time that the integrity of p53 affects the production of a special
cell surface protein called Major Histocompatibility Complex (MHC) class I. MHC class I molecules on the cancer
cell surface serve as
targets for the immune system.
«Our work strongly supports that cancer stem
cells are the main source of growth in these tumors and, as such, should be considered promising
targets for treatment,» says Mario Suvà, MD, PhD, of the MGH Department of Pathology, co-senior author of the Nature paper.
For example, an MDM2 -
targeting drug shrank deep - tissue fat
cell tumors in just one out of 20 patients in a phase I safety trial, published in 2012.
In a paper published in the current online issue of the journal Small, titled «Immune
Cell - Mediated Biodegradable Theranostic Nanoparticles
for Melanoma
Targeting,» the researchers report the use of a novel biodegradable and photoluminescent poly (lactic acid) nanoparticle, loaded with melanoma - specific drugs with immune
cells as the nanoparticle carriers.
«
For years, researchers have focused on delivering more chemotherapy drugs into cancer
cells using nanoparticles, without
targeting the root of drug resistance,» He said.
We designed chimeric antigen receptor (CAR) T
cells that specifically
target podoplanin (PDPN), a key protein
for the progression of solid tumors, including GBM.»
By combining this strategy with cancer
cell -
targeting materials, we should be able to develop a therapy
for glioblastoma and other challenging cancers in the future.»
To begin the research, the scientists screened human DIPG tumor cultures
for surface molecules that could act as
targets for CAR - T
cells.
Targeting exhausted immune
cells may change the prognosis
for patients with acute myeloid leukemia (AML) relapse after a stem
cell transplant, according to Penn State College of Medicine researchers.
The network provides an invaluable resource
for identifying
targets to treat the disease and has been used to implicate a particular signaling pathway involved in
cell motility.
Artist's conception of nanoparticle - carrying immune
cells that
target tumors and release drug - loaded nanoparticles
for cancer treatment.