Articles tagged with Sickle Cell Anemia
Scientists at Beam Therapeutics have created a redesigned base editor that successfully repairs the single-base mutation causing sickle-cell disease. The therapy targets an upstream regulatory pathway to express fetal hemoglobin, offering a potential solution for this genetic disorder.
A new oral treatment, FTX-6058, has the potential to address the root cause of sickle cell disease by increasing fetal hemoglobin levels. The drug could be formulated into a daily tablet and has shown better results than current treatments in preclinical experiments.
Researchers found that adaptive cognitive training program Cogmed Working Memory Training improved visual working memory, verbal short-term memory, and math fluency in children with SCD. Patients who completed at least 10 training sessions showed the most significant benefits.
A clinical trial combining CRISPR technology with UCLA and UCSF expertise aims to directly correct the sickle mutation in blood stem cells, addressing the underlying cause of debilitating sickle cell disease. The goal is to out-compete native sickle cells by correcting 20% of genes.
A team of researchers from Texas A&M University developed an organ-on-a-chip device that can model the disease physiology of patients with sickle cell disease. The device uses blood outgrowth endothelial cells to mimic a patient's vessel, allowing for personalized treatment and predicting disease progression.
A new study evaluates the economic impact of gene therapy on severe sickle cell disease, suggesting substantial costs and potential cost savings for Medicaid programs. The analysis estimates a high prevalence of sickle cell disease among these programs, which may affect treatment affordability.
Scientists have discovered that sugars called mannoses play a crucial role in the immune system's response to malaria and sickle cell disease. This breakthrough could lead to new therapeutic approaches for treating these diseases.
Researchers developed an AI algorithm to mine physiological data from patients with chronic pain, detecting changes in pain levels and atypical fluctuations. The study aims to provide a more precise treatment method by supplementing subjective pain assessments with objective data-driven approaches.
A new study shows that machine-learning strategies can be applied to routinely collected physiological data to provide clues about pain levels in people with sickle cell disease. The researchers found that these vital signs give clues into the patients' reported pain levels, outperforming baseline models.
A single letter difference in a gene leads to sickle cell disease affecting 20 million worldwide, with varying life expectancy depending on social and environmental factors. A new review calls for integrative studies to better understand the disease globally and develop locally-appropriate interventions.
A new analysis indicates that patients with kidney failure associated with sickle cell disease benefit from kidney transplantation to a similar extent as patients with kidney failure from other causes. Despite this, the sickle cell population is less likely to receive transplants.
Researchers found that an enriched diet and companionship can reduce pain in mice with sickle cell disease by increasing serotonin levels. Duloxetine, an antidepressant boosting serotonin, also showed similar pain-reducing effects.
Researchers have successfully treated two patients with beta thalassemia and sickle cell disease using CRISPR-Cas9 gene editing, a promising treatment for these severe red blood congenital diseases. The new approach uses the patient's own cells and eliminates the risk of rejection or graft-versus-host disease.
A recent study presented at the 2020 ASH meeting demonstrated that Gazelle's integrated anemia-SCD test has a high sensitivity and specificity for detecting anemia, with over 92.3% accuracy. The test is expected to be available in mid-2021 and has the potential to improve disease management in low-resource settings.
Scientists at ChristianaCare's Gene Editing Institute are developing a novel gene therapy for inherited blood disorders like sickle cell disease using CRISPR technology. The team aims to identify genetic variations that affect treatment efficacy and create a tailored approach for personalized medicine.
A new study examines the risk of acute kidney injury among Black individuals with sickle cell trait and disease, finding a higher risk for acute kidney injury and faster loss of kidney function. The study adds important information to sparse data on the risk of acute kidney injury in those carrying the sickle cell gene.
Researchers have developed a new technology to diagnose sickle cell disease with increased sensitivity and speed, using only a small droplet of blood and costing less than traditional methods. The Acousto Thermal Shift Assay (ATSA) measures protein interactions and mutations in under one minute.
Researchers at NHGRI conducted a study to better understand the needs of patients with sickle cell disease regarding genome editing clinical trials. Participants showed higher genetic literacy levels than expected, but emphasized the need for accessible information about risks and benefits.
A five-year study led by University of Illinois Chicago researchers will assess the effectiveness of acupuncture and guided meditation in managing sickle cell disease pain, aiming to reduce opioid use. The study, funded at $7.1M, will explore non-pharmacological pain management interventions for approximately 360 patients.
A new study found that adults with sickle cell disease who received medical marijuana required fewer hospital visits than those who did not. Receiving medical marijuana was also associated with an increase in the use of edible cannabis products, which may help individuals better tolerate their pain at home.
Researchers found that the gut microbiome plays a key role in triggering painful vessel-clogging episodes, or VOE, in people with sickle-cell disease. Stress triggers increased gut permeability, allowing beneficial bacteria to stimulate pro-inflammatory molecules, which promote inflammation and damage internal organs.
A new clinical trial suggests that vaporized cannabis is safe and potentially effective in mitigating chronic pain caused by sickle cell disease. The study found that patients experienced reduced pain levels and improved mood over time, paving the way for further research on medical cannabis as a treatment option.
A study in the New England Journal of Medicine has found that dose escalation of hydroxyurea treatment is more effective and has similar side effects than a lower fixed dose for children with sickle cell anemia in Uganda. The results have global implications and can potentially improve treatment outcomes worldwide.
A new study finds that dose escalation of hydroxyurea treatment for children in Uganda with sickle cell anemia is more effective and has similar side effects than a lower fixed dose. The findings have global implications, removing a major barrier to expanding the use of hydroxyurea worldwide.
A study published in the New England Journal of Medicine has found that dose escalation of hydroxyurea treatment is more effective and has similar side effects than a lower fixed dose for children with sickle cell anemia in Uganda. The findings have global implications and could impact use of hydroxyurea worldwide.
Rice University has secured a $2.45 million NIH grant to develop CRISPR-Cas9 gene editing for sickle cell disease treatment. The study aims to address the risks and mechanisms of gene editing in hematopoietic stem/progenitor cells.
A new analysis supports prior reports that people with sickle cell disease experience lower rates of HIV infection. However, the molecular characteristics of immune system cells do not explain the reduced risk.
The NIH Technology Accelerator Challenge aims to develop handheld, digital technologies for detecting diseases with high global impact. The challenge will focus on sickle cell disease, malaria, and anemia, addressing the need for accessible diagnostic tools in low-resource settings.
In 2019, the pharmaceutical industry approved 48 new medicines, including treatments for various cancers, sickle cell disease, and migraines. The majority of these drugs had novel mechanisms of action, such as antibody-drug conjugates and small interfering RNA.
Researchers at the National Institutes of Health have developed a new and improved viral vector that can deliver therapeutic genes more efficiently than conventional vectors. The new vector shows promising results in animal models, with up to 10 times higher efficiency and six times higher carrying capacity.
A team of Brown University researchers developed a new computer model to simulate the way red blood cells become misshapen in sickle cell disease. The model can be used to quickly and inexpensively pre-screen new drug candidates, potentially identifying promising treatments for the genetic disorder that affects millions worldwide.
Non-traumatic fatalities in football are often linked to over-exertion during conditioning sessions, which can be prevented with standardized workout design and accountability. The NCAA's acclimatization model has been ineffective in reducing exertional heat-related fatalities.
Researchers at Pitt/UPMC are testing a technique called red cell exchange transfusion to prolong life and slow organ damage in sickle cell disease patients. The SCD-CARRE trial aims to determine whether red cell exchange can lower deaths and reduce hospitalizations.
A UH researcher reports that the formation of abnormal hemoglobin clusters in red blood cells may be a crucial step in developing treatments for sickle cell disease. The study suggests that inducing similar-sized clusters in liquids and solids could have applications in nanotechnology and industry.
A clinical trial for a new sickle cell disease treatment, IMR-687, has shown early promise with an increase in F-cells and a reduction in pain, improving biomarkers of the disease.
A new non-invasive prenatal test has been developed to diagnose sickle cell disease in unborn babies. The test uses cell-free fetal DNA and has shown promising results, with successful diagnoses achieved in 21 out of 24 pregnancies. Further development and validation are needed before the test can be introduced into clinical practice.
Researchers developed a rapid and reliable method to continuously monitor sickle cell disease using microfluidics-based electrical impedance sensor. This technology characterizes dynamic cell sickling and unsickling processes, providing important contributing factors of abnormal blood flow and vaso-occlusion.
Recent research on sickle cell disease highlights major progress in diagnosis and treatment, but warns that more screening of newborns is needed, particularly in sub-Saharan Africa. The study emphasizes the importance of investing in health policies to address the growing number of cases.
A new study uses CRISPR-Cas9 to repair the sickle cell mutation in patients' hematopoietic stem cells, boosting their own protective fetal hemoglobin. The approach showed promising results, with up to 40% of edited cells fixed and a significant increase in fetal hemoglobin expression.
A recent study found significant associations between genetic markers, body mass index, and symptom variation in collegiate football players with sickle cell trait. Higher levels of fetal hemoglobin are linked to better outcomes, particularly for those with higher body weight.
A new study finds that routine sickle cell disease screening among migrants can help prevent severe complications and death. Researchers estimate that up to 80% of newborns with SCD are born in sub-Saharan countries, where the condition is highly prevalent.
Researchers found that chronic morphine treatment decreased survival in control mice, but not in sickle mice with sickle cell disease. Chronic opioid therapy also caused hyperalgesia, a condition of increased pain sensitivity, in sickle mice. The study suggests that opioids may still be effective for managing pain in SCD patients despi...
A breakthrough study has revealed that the building blocks of sickle cell disease are much less efficient at organizing than previously thought. This finding could lead to new treatments, including medicines prescribed at lower doses, and improve outcomes for approximately 20 million people worldwide who suffer from the lifelong disease.
A new protocol has increased the rate of successful bone marrow transplants from half-matched donors to nearly 100%, offering a higher chance of cure for patients with severe inherited blood disorders. Patients experienced reduced symptoms and no longer required immunosuppressive medications.
Researchers have developed a gene therapy treatment that is reversing disease symptoms in two adults with Sickle Cell Anemia. The treatment, which uses reduced-intensity preconditioning, shows promise for transportability to resource-challenged parts of the world where SCA is most common.
Researchers present findings on acute erythroid leukemia, identifying five subtypes with distinct genomic features and survival rates. St. Jude also shares updates on gene therapy for hemophilia B and advances in sickle cell disease treatment.
A study of 73 adults with sickle cell disease found that even high-dose opioid treatment did not effectively manage pain in a subset of patients. The researchers emphasize the need for non-opioid pain relievers to address this challenge.
The Doris Duke Charitable Foundation has awarded a grant to the Critical Path Institute (C-Path) to accelerate the development of therapies for sickle cell disease. C-Path will plan a consortium of stakeholders to identify and work collaboratively on jointly beneficial drug development tools and novel methodologies.
Researchers found that immature reticulocytes are more prone to stick to blood vessel walls, leading to vaso-occlusive pain crises. A new microfluidic system mimicked post-capillary vessels, revealing how low oxygen levels cause sickle red cells to form stiff fibers that increase adhesion.
Researchers at Mount Sinai will investigate the use of inhaled corticosteroids to treat sickle cell disease, a condition that affects 100,000 Americans and has high healthcare costs. The IMPROVE 2 study aims to improve patient outcomes and reduce ED visits.
Doctors at the University of Illinois Hospital have cured seven adult patients with sickle cell disease using compatible donor stem cells. The new transplant protocol allows for half-matched donors, significantly increasing potential donors and improving treatment outcomes.
Researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells, boosting their production of foetal haemoglobin. This approach may lead to new therapies for sickle cell anaemia and thalassaemia by targeting precise changes in the genome.
Researchers at UIC will study molecular neurobiology of chronic pain and develop new drug treatments for sickle cell disease patients experiencing chronic pain. The funding aims to understand the role of protein kinases in chronic pain and identify potential therapeutic targets.
Scientists at Rice University have successfully used CRISPR/Cas9-based gene editing to repair 20-40% of stem and progenitor cells from patients with sickle cell disease. The study, led by bioengineer Gang Bao, aims to find a cure for the hereditary disease.
A new study finds that only 18% of children with sickle cell anemia receive daily antibiotics to reduce the risk of deadly infections. The condition affects racial and ethnic minority populations and can lead to serious complications like meningitis or death if left untreated.
Researchers found a link between negative emotions and higher opioid use in people with sickle cell disease, particularly with low pain levels. Negative thinking also played a role in short-acting opioid use, especially when pain was reported as high.
A high dose of hydroxyurea significantly increased fetal hemoglobin levels and reduced hospitalizations among young sickle cell anemia patients. The study found that maximizing the dose resulted in a substantial reduction in hospitalizations, with nearly all patients experiencing improved health outcomes.
A large NIH-funded clinical trial will determine if bone marrow transplantation can be part of standard care for patients with severe sickle cell disease. The study aims to balance health risks with the promise of being cured, and participants will be followed for two years.
Researchers are launching a national study to understand why fewer than 30% of children with sickle cell disease receive transcranial Doppler screening, which can prevent strokes. The goal is to identify barriers and implement strategies to increase access to the life-saving test.
A year-long trial suggests that hydroxyurea treatment is safe and effective for Ugandan children with sickle cell anemia, contrary to concerns about increased malaria risk. The study found no correlation between hydroxyurea treatment and the rate or severity of malaria infection.