Blood Pressure Patterns and Factors Associated with Relative Hypertension among Steady State Sickle
Cell Disease Patients in Kinshasa, Democratic Republic of the Congo: A Cross-Sectional Study
Beta - Globin Haplotypes and Alpha - Thalassemia 3.7 kb Deletion in Sickle
Cell Disease Patients From the Occidental Brazilian Amazon
«When pain was reported as low, sickle
cell disease patients reported higher opioid use if they catastrophized, or focused their thinking on their pain, than if they didn't,» says Finan.
«These national guidelines are directed not just to hematologists but to all medical practitioners who might encounter sickle
cell disease patients, to inform them about hydroxyurea and how to best offer general medical care to them,» Dr. Buchanan said.
Salk researchers reprogrammed skins cells taken from a sickle
cell disease patient into induced pluripotent stem cells (iPSCs), immature cells capable of developing into any type of bodily tissue.
Not exact matches
The companies» R&D will focus on on a gene mutation present in a wide swath of
patients with ALS, a degenerative nervous system
disease that eats away at nerve
cells and weakens muscles.
His research has spanned hematopoiesis, gene therapy, stem
cell biology, genomics and cancer, consistently focusing on bringing the very latest research advances to
patients with heretofore incurable
diseases.
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 biotech specialist said that its updated phase 2 data in a study of its poziotinib candidate treatment for non-small
cell lung cancer resulted in a preliminary confirmed objective response rate and potential progression - free survival benefit in
patients with the EGFR Exon 20 Mutant form of the
disease.
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.
Lab - grown tissues derived from
patients» stem
cells may also allow researchers to screen drugs and test their effectiveness on
diseases like cancer.
«Altering Huntington's
patients» skin
cells into brain
cells sheds light on
disease: Reprogrammed brain
cells exhibit «symptoms» of fatal disorder.»
«If we want to treat the greatest number of
diseases, we need to figure out how to get these molecules inside the
cells of
patients — not just increasing the number of target
cells but also hitting tissues other than the liver,» says Anderson, who co-founded a company called CRISPR Therapeutics to pursue those goals.
Skin
cells are easy to collect from
patients and share the same genetic blueprint — and
disease - causing mutations — as brain
cells.
«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.
The long - term persistence of CD8αα + T
cells where initial infection occurs may explain why
patients have asymptomatic recurrences of genital herpes because these
cells constantly recognize and eliminate the virus, according to Jia Zhu, Ph.D., corresponding author, research assistant professor in Laboratory Medicine at the University of Washington and an affiliate investigator in the Fred Hutch Vaccine and Infectious
Disease Division.
«In effort to treat rare blinding
disease, researchers turn stem
cells into blood vessels:
Patients around the world contribute skin samples to test potential new therapy.»
They isolated blood
cells from HIV - positive
patients on antiretroviral therapy and at different stages of
disease progression, as well as
cells from non-infected individuals.
«This research represents an important step toward the goal of being able to better treat thyroid
diseases and being able to permanently rescue thyroid function through the transplantation of a
patient's own engineered pluripotent stem
cells,» explained co-corresponding author Anthony N. Hollenberg, MD, Chief of the Division of Endocrinology, Diabetes and Metabolism at BIDMC and Professor of Medicine at Harvard Medical School.
This knowledge is important, as iPSCs hold great promise in the field of regenerative medicine, as they can provide a single source of
patient - specific
cells to replace those lost to injury or
disease.
Creating a whole set of miniature new livers might take as little as obtaining liver
cells from healthy donors and placing them inside the lymph nodes of
patients suffering from liver
disease.
«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.
The idea to specifically study this group of
patients was based on groundbreaking research Garon published in the New England Journal of Medicine last year, which found that among
patients who received pembrolizumab, those with PD - L1 expression on at least 50 percent of their cancer
cells showed the longest survival and
disease control.
The Porteus team started with human stem
cells from the blood of
patients with sickle
cell disease, corrected the gene mutation using CRISPR and then concentrated the human stem
cells so that 90 percent carried the corrected sickle
cell gene.
Researchers used tissue and blood samples to show that the gammopathy (a precursor to myeloma) in both mice and
patients with Gaucher
disease is triggered by specific lipids, and that the antibodies made by tumor
cells in nearly a third of myeloma
patients are directed against such lipids.
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.
The trial enrolled 361
patients with recurrent or metastatic head and neck squamous
cell carcinoma who had not responded to platinum - based chemotherapy, a rapidly progressing form of the
disease with an especially poor prognosis, said Dr. Ferris.
An overabundance of the bacteria Veillonella in the digestive tract may increase pain in
patients with sickle
cell disease (SCD).
After exploring the molecular pathway in mice, the researchers focused on cardiac stem
cells in
patients with heart
disease.
For the new trial, hospitals enrolled
patients with advanced, squamous non-small
cell lung cancer whose
disease had progressed despite initial chemotherapy.
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.
Rashid's ultimate hope is that one day scientsts will be able to correct the
diseased cells in the lab and transplant them back into the
patient, but he cautions that
cell - based therapies won't be available anytime soon.
Cancer stem - like
cells are thought to be the root cause of chemotherapy resistance, leading to treatment failure in
patients with advanced
disease and the triggers of tumour recurrence and metastasis (regrowth).
«Cardiac stem
cells from heart
disease patients may be harmful: Researchers discover molecular pathway involved in toxic interaction between host
cells and immune system.»
Mice transplanted with
cells grown from a
patient suffering from Huntington's
disease (HD) develop the clinical features and brain pathology of that
patient, suggests a study published in the latest issue of Acta Neuropathologica by CHA University in Korea, in collaboration with researchers at Université Laval in Québec City, Canada.
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 new findings build on prior research from the Dhodapkar lab demonstrating that
patients with Gaucher
disease, an inherited lipid storage disorder, have a significant increased risk for developing myeloma; and the discovery of a subset of lipid - reactive immune
cells, called type II NKT - TFH, that promote the development of plasma
cells.
Scientists from the University of Cambridge's Institute for Medical Research obtained skin
cells from 10
patients — seven who had various forms of inherited liver
disease, and three healthy controls.
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.
A common feature of neurodegenerative
diseases such as Alzheimer's, Parkinson's or Huntington's
disease are deposits of aggregated proteins in the
patient's
cells that cause damage to cellular functions.
The presence of the dead, snarled nerve
cells and sticky protein fragments characteristic of Alzheimer's in the main smell - processing structures of these deceased
patients could suggest the
disease gets its start in the olfactory system.
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
cells were derived from eggs that had been injected with DNA from the
patients, so they could eventually be transplanted back to replace or correct the
patient's
diseased cells without fear of immune rejection.
«Stem
cell gene therapy could be key to treating Duchenne muscular dystrophy: Approach developed at UCLA holds promise for 60 percent of
patients with the deadly
disease.»
This accomplishment opens the door for
disease modeling and drug screening and brings personalized
cell therapy a step closer for
patients with diabetes.
Diabetes researchers are considering various replacements for insulin injections: Transplanting new pancreatic islet
cells that make insulin, coaxing the
patient's own islets to regenerate, or treating diabetics early in the
disease with immune - suppressing therapies to prevent their body from destroying the rest of their pancreatic islets.
The
disease commonly starts in childhood and causes the body's own immune system to attack and destroy the insulin - producing
cells in the pancreas, leaving the
patient dependent on life - long insulin injections.
In the next phase of the study, researchers will genetically sequence tumor
cells from at least 500
patients and follow the course of their
disease.