Blood diseases cured with bone marrow transplant
Doubling the low amount of total body radiation delivered to patients undergoing bone marrow transplants with donor cells that are only 'half-matched' increased the rate of engraftment from only about...
View ArticleMost precise measurements of sickle cell disease building blocks could lead...
In a breakthrough study of sickle cell disease, biomedical engineers have revealed that the building blocks of the disease are much less efficient at organizing than previously thought. The findings...
View ArticleResearchers optimize gene editing for SCD and beta thalassemia
Gene editing of patients' blood stem cells can potentially cure many blood disorders. But introducing targeted edits into these cells has been challenging, and the edits aren't always stable once the...
View ArticleAthletes with sickle cell traits are at more risk to collapse: here's why
A genetic variation known to affect sickle cell disease might be the reason why some college football players experience adverse clinical outcomes during periods of extreme physical exertion and others...
View ArticleNew genetic weapons challenge sickle cell disease
Researchers advancing gene-editing techniques to help patients with sickle cell disease discover an unexpected boost in fetal hemoglobin production, which mutes the effect of the disease.
View ArticleNew method to rapidly, reliably monitor sickle cell disease
Researchers have developed a rapid and reliable new method to continuously monitor sickle cell disease using a microfluidics-based electrical impedance sensor. This novel technology can characterize...
View ArticleSickle cell drug showing promise in clinical trial
An investigational drug for the treatment of sickle cell disease is showing early promise in clinical trials for impacting biomarkers of the disease in patients, reported researchers.
View ArticleThe way sickle cells form may be key to stopping them
Chemists are reporting a new finding on how sickle cells are formed, which may lead not only to stopping their formation, but to new avenues for making uniformly-sized nanoparticles for industry.
View ArticleOver-conditioning kills: Non-traumatic fatalities in football is preventable
Most non-traumatic fatalities among high school and college football athletes do not occur while playing the game of football, but rather during conditioning sessions which are often associated with...
View ArticleTargeting a blood stem cell subset shows lasting, therapeutically relevant...
Researchers have used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several blood disorders, including sickle cell disease and beta-thalassemia.
View ArticleComputer model could help test new sickle cell drugs
A new computer model that captures the dynamics of the red blood cell sickling process could help in evaluating drugs for treating sickle cell disease.
View ArticleNew method reveals how damage occurs in human biological cells due to...
Researchers have developed a novel way to measure how mechanical fatigue affects biological cells. They also have established the important role of this effect in influencing physical properties of...
View ArticleFor the first time, professor observes crystallized iron product, hemozoin,...
For the first time ever, a professor has observed a crystallized iron product called hemozoin being made in mammals, with widespread implications for future research and treatment of blood disorders....
View ArticleResearchers create new viral vector for improved gene therapy in sickle cell...
Researchers have developed a new and improved viral vector -- a virus-based vehicle that delivers therapeutic genes -- for use in gene therapy for sickle cell disease. In advanced lab tests using...
View ArticleBlood transfusions: Fresh red blood cells no better than older ones
Findings from the ABC-PICU study on critically ill children may alter policies at hospitals where fresh red cells are preferentially used.
View ArticleExploring mechanisms of resistance to HIV in people with sickle cell disease
A new analysis supports prior reports that people with sickle cell disease have lower rates of human immunodeficiency virus (HIV) infection, but follow-up cell studies did not reveal a mechanism to...
View ArticleNew mobile health tool measures hemoglobin without drawing blood
Researchers have developed a way to use smartphone images of a person's eyelids to assess blood hemoglobin levels. The ability to perform one of the most common clinical lab tests without a blood draw...
View ArticleCannabis shows potential for mitigating sickle cell disease pain
Cannabis appears to be a safe and potentially effective treatment for the chronic pain that afflicts people with sickle cell disease, according to a new clinical trial.
View ArticleHow Dantu blood group protects against malaria, and how all humans could benefit
The secret of how the Dantu genetic blood variant helps to protect against malaria has been revealed for the first time. The team found that red blood cells in people with the rare Dantu blood variant...
View ArticleAs genome-editing trials become more common, informed consent is changing
As public interest and expanded research in human genome editing grows, many questions remain about ethical, legal and social implications of the technology. People who are seriously ill may...
View ArticleCRISPR technology to cure sickle cell disease
A new article reports two patients appear to have been cured of beta thalassemia and sickle cell disease after their own genes were edited with CRISPR-Cas9 technology. The two researchers who invented...
View ArticleNutrition, companionship reduce pain in mice with sickle cell disease
Researchers have found that an enriched diet and companionship can reduce pain in mice with sickle cell disease by increasing serotonin. They also discovered that duloxetine, an antidepressant that...
View Articlesickle cell disease: Addressing a complex world of pain in a single gene...
A new review analyzes disparities in experience and treatment of sickle cell disease.
View ArticleStandard vital signs could help estimate people's pain levels
A new study demonstrates that machine-learning strategies can be applied to routinely collected physiological data, such as heart rate and blood pressure, to provide clues about pain levels in people...
View ArticleA better treatment for sickle cell disease
Sickle cell disease is the most prevalent inherited blood disorder in the world, affecting 70,000 to 100,000 Americans. However, it is considered an orphan disease, meaning it impacts less than 200,000...
View ArticleToward a reliable oral treatment for sickle cell disease
For those who have sickle cell disease, there are only a few treatment options, which include bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Now,...
View ArticleResearchers speed identification of DNA regions that regulate gene expression
Scientists have developed a highly efficient method to address a major challenge in biology -- identifying the genetic 'switches' that regulate gene expression.
View ArticleResearchers develop proof-of-concept treatment that elevates adult and fetal...
Researchers have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and...
View ArticleGenetic base editing treats sickle cell disease in mice
Sickle cell disease leads to chronic pain, organ failure, and early death in patients worldwide. A team has demonstrated a gene editing approach that efficiently corrects the mutation underlying SCD in...
View ArticleStudy identifies risk factors for severe COVID-19 in individuals with sickle...
New research finds that certain factors, such as a history of severe pain episodes and coexisting organ conditions, increase the risk of severe COVID-19 illness, including hospitalization, in...
View ArticleTrials reveal efficacy and safety of oral drug for treating anemia associated...
Pooled results from phase 3 clinical trials demonstrate that roxadustat is efficacious and may be comparable to placebo in studies done to date for treating anemia in patients with chronic kidney...
View ArticleNovel assay finds new mechanism underlying red blood cell aging
A multifaceted microfluidic in vitro assay is helping to identify the role of hypoxia on red blood cell aging via the biomechanical pathways. It holds promise for investigating hypoxic effects on the...
View ArticleNew technique may lead to safer stem cell transplants
Studying mice, researchers have developed a method of stem cell transplantation that does not require radiation or chemotherapy. Instead, the strategy takes an immunotherapeutic approach, combining the...
View ArticleDoctoral student finds alternative cell option for organs-on-chips
Organ-on-a-chip technology has provided a push to discover new drugs for a variety of rare and ignored diseases for which current models either don't exist or lack precision. In particular, these...
View ArticleExperimental gene therapy reverses sickle cell disease for years
An experimental gene therapy for sickle cell disease restored blood cells to their normal shape and eliminated severe pain crises for years after treatment, a multicenter study has found.
View ArticleBeing in space destroys more red blood cells
A world-first study has revealed how space travel can cause lower red blood cell counts, known as space anemia. Analysis of 14 astronauts showed their bodies destroyed 54 percent more red blood cells...
View ArticleA possible cure for sickle cell?
Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed...
View ArticleCRISPR gene editing reveals biological mechanism behind common blood disorder
Researchers have used CRISPR gene editing -- a type of 'molecular scissors' -- to understand how deletions in one area of the genome can affect the expression of nearby genes. The work will help...
View ArticlePhase 3 clinical trial results lead to approval of oral drug for red blood...
Results from a phase 3 clinical indicate that the oral drug mitapivat is safe and effective for treating adults with pyruvate kinase deficiency, a genetic condition that causes red blood cell...
View ArticleA link between hypoxia and fetal hemoglobin provides hope for sickle cell...
Scientists found the cellular response to low oxygen also increases fetal hemoglobin expression in adults, which could lead to novel treatments for some common genetic anemias.
View ArticleCRISPR-Cas9: Even good gene edits can go bad
Researchers are working to reveal potential threats to the efficacy of CRISPR/Cas9 gene editing, even when it appears to be working as planned.
View Article'Spleen-on-a-chip' yields insight into sickle cell disease
Researchers have designed a microfluidic device, or 'spleen-on-a-chip,' that can model how the spleen's filters become clogged by sickled red blood cells, leading to a life-threatening condition known...
View ArticleQuantifying the life expectancy gap for people living with sickle cell disease
While research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps. A new study finds that the average...
View ArticlePrime editing shows proof of concept for treating sickle cell disease
Scientists showed how prime editing can correct mutations that cause sickle cell disease in a potentially curative approach.
View ArticleCRISPR and single-cell sequencing pinpoint causal genetic variants for traits...
A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98%...
View ArticleResearchers develop 'in vivo' RNA-based gene editing model for blood disorders
In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification...
View ArticleResearch sheds new light on gene therapy for blood disorders
A study shows new steps toward more patients getting gene therapy.
View ArticlePromising evidence for sickle cell gene therapy
A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.
View ArticleResearchers chart the contents of human bone marrow
A team has mapped the location and spatial features of blood-forming cells within human bone marrow. Their findings confirm hypotheses about the anatomy of this tissue and provide a powerful new means...
View ArticleNew research advances understanding of cancer risk in gene therapies
Researchers have discovered that 'cell competition' following gene therapy results in the accumulation of stem cells with genetic mutations which make them grow faster. Some of these genetic mutations...
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