Articles tagged with Sickle Cell Anemia
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Individuals with severe sickle cell disease express willingness to accept gene therapy risks for a potential cure. Gene therapies offer higher chances of eliminating symptoms and extending life expectancy, but also come with significant risks.
A recent study has shown that pneumococcal conjugate vaccines have significantly reduced the rate of invasive pneumococcal infection among children with sickle cell disease. The overall rate of IPD decreased by 87% in children under 4 years old, and by 80% in those aged 5 to 9.
A new clinical trial published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for sickle cell disease. The therapy was shown to decrease vaso-occlusive events and increase production of fetal hemoglobin, providing relief from the painful condition.
New research led by Nansi Boghossian found that sickle cell disease contributes to racial disparities in severe maternal morbidity, with Black individuals at least 1.5 times more likely to experience complications. The study suggests preconception care and management are critical for improving pregnancy outcomes for patients with sickl...
Researchers have made a significant discovery in gene therapy, showing that genetically engineered blood stem cells can be created while still in the bone marrow. This approach eliminates the need for chemotherapy and transplantation of corrected cells, cutting costs and improving access to critical treatments.
Gang Bao's lab receives a 4-year, $2.6 million grant from the National Institutes of Health to investigate the safety and efficacy of using gene editing treatments like CRISPR-Cas9 to treat sickle cell disease. The team aims to understand the mechanisms behind large gene modifications and their biological consequences.
The program found 1,695 persons in Indiana with sickle cell disease, with a median age of 21 and 87% Black. The data will inform clinical care, identify gaps in access to care, and guide lawmakers.
A recent study suggests that the global mortality burden of Sickle Cell Disease (SCD) is approximately 11 times higher than previously recorded, with an estimated 376,000 deaths in 2021. The Commission highlights the need for increased funding and access to treatments such as hydroxyurea, blood transfusions, and malaria prevention meth...
Researchers use base editing technology to restart fetal hemoglobin expression in SCD patient cells, achieving higher and more stable levels than other genome editing technologies. The approach has potential as a 'one-size-fits-all' treatment for all mutations that cause SCD and beta-thalassemia.
Researchers have discovered that gene editing technologies may introduce unintended mutations and damage to DNA in early human embryos. The study found that most cells repair breaks in the DNA using non-homologous end joining, which can lead to additional genetic abnormalities.
A new study reveals sickle cell disease is far deadlier than its textbook description, with a total mortality burden of 373,000 deaths in 2021. The condition disproportionately affects children, adolescents, and young adults worldwide, highlighting the need for universal newborn screening and early intervention.
This systematic analysis reports a significant global increase in sickle cell disease prevalence and mortality. The study reveals that sickle cell disease affects millions worldwide, with the highest burden in low- and middle-income countries.
An observational study of 329,000 Medicare admissions found that older persons receiving hospital care from allopathic (M.D.) or osteopathic (D.O.) physicians experience similar quality and cost of care. Researchers also highlight systemic health inequities faced by persons with sickle cell disease.
A cross-sectional analysis of 2,200 individuals with sickle cell disease found associations between employment status, sex, age, and depression with pain frequency. Comprehensive treatment for SCD must consider the full experiences of patients, including mental health impacts on pain reduction.
A team of researchers is exploring the potential of HDAC inhibitors to treat sickle cell disease by reactivating the fetal hemoglobin gene. Early evidence suggests that panobinostat, a specific inhibitor, can increase fetal hemoglobin levels in red blood cells and mouse models.
A new approach, STING-seq, combines genetic association studies, gene editing, and single-cell sequencing to identify causal variants and genetic mechanisms for blood cell traits. This method can help scientists identify drug targets for diseases with a genetic basis.
Researchers used prime editing to correct the mutation that causes sickle cell disease, restoring normal blood parameters in mice. The approach showed promising results with up to 41% conversion of mutated cells to healthy ones.
A new study published in Blood Advances found that the average life expectancy of publicly insured individuals with sickle cell disease is roughly 52.6 years, which is significantly lower than the overall US life expectancy of 73.5 years for men and 79.3 years for women. The study also revealed worse survival outcomes among those insur...
A new study found that less than 4% of people with sickle cell disease have prescriptions for newer FDA-approved pain-relieving drugs, despite their availability. The study also revealed significant gaps in prescription patterns by geographic region and age group, emphasizing the need for education and access to treatment options.
Researchers at University Hospitals Cleveland Medical Center discovered that S-nitrosohemoglobin senses areas with insufficient oxygen and restores blood flow for oxygenation. This breakthrough opens up a new line of drug development to correct poor tissue oxygenation, including peripheral artery disease and sickle cell disease.
Researchers discovered that bone marrow transplants can halt the development and progression of brain blood vessel disease in adults with sickle cell disease. The study found that receiving stem cell transplants led to positive changes in blood vessels, reducing the risk of stroke among patients with the condition.
A new study found that massage therapy significantly reduces pain, stress, and anxiety in children, adolescents, and young adults with cancer or sickle cell disease. The study published in Pediatric Blood & Cancer reports clinically significant benefits from massage therapy, highlighting its potential as a non-pharmacologic treatment o...
Researchers developed a microfluidic device to model the spleen's filtration function in patients with sickle cell disease. The study found that low oxygen levels can cause the spleen's filters to become clogged, while boosting oxygen levels can unclog them, potentially explaining how blood transfusions help patients.
A new study found that music therapy resulted in clinically significant reductions in pain and anxiety for patients with cancer and sickle cell disease. Patients with SCD who received music therapy also reported significantly higher pain and anxiety at baseline compared to those without SCD.
Researchers at Rice University have developed a procedure to quantify unintended changes that accompany on-target CRISPR-Cas9 gene editing, potentially threatening the efficacy and safety of therapies. The new method uses single-molecule sequencing with unique molecular identifiers to detect large deletions, insertions, and chromosomal...
Researchers found a direct connection between low oxygen conditions and increased fetal hemoglobin production in adults. A drug that activates the cellular response to low oxygen could potentially treat sickle cell disease by promoting HbF production, which has therapeutic benefits.
Researchers developed new educational materials to help individuals with sickle cell disease and their support networks learn about gene therapy clinical trials, benefits, risks, and treatment options. The project aims to facilitate engagement between the community and those conducting relevant clinical trials.
A new gene therapy for sickle cell disease was tested in mice but proved ineffective due to the complex genetic make-up of the Berkeley mice. The study suggests that researchers should carefully consider the genetics of the mice used to test human diseases.
The Brigham and Women's Hospital will implement and evaluate an integrated service delivery model (PEN-Plus) for severe chronic noncommunicable diseases, such as type 1 diabetes and rheumatic heart disease, in eight low- and lower-middle-income countries. The initiative aims to enable one million of the world's poorest children and you...
A study by RCSI University of Medicine and Health Sciences has identified barriers and motivators to blood donation for people from ethnic minority groups in Ireland. Factors that motivate people from minority ethnic backgrounds to give blood include religious reasons and a desire to help others in their own communities.
A new study reveals that individuals with sickle cell disease spend approximately $1.7 million on lifetime medical expenses, highlighting the significant economic burden of living with this disease. The study found that out-of-pocket costs for those with private insurance plans can be as high as 5-10% of their annual income.
Researchers found that people living with SCD have more medical appointments and urgent care visits, leading to higher out-of-pocket costs of $1,300 annually. The study suggests that curative therapies could reduce these costs, but accessing them will depend on conversations among policymakers, insurers, and patients.
A new study found that many adolescent and young adult men with sickle cell disease are unaware of potential fertility issues associated with the disorder. The research suggests a need for accessible fertility education and testing options to address this knowledge gap, particularly in how SCD and treatments may affect fertility.
A UCLA-led team has created a roadmap tracing each step in human blood stem cell development, providing a blueprint for producing fully functional blood stem cells. The map could help expand treatment options for blood cancers and inherited disorders.
A systematic review and meta-analysis found that SGLT2 inhibitors reduce hospitalizations and cardiovascular deaths in heart failure patients, regardless of diabetes status. The analysis also warned of increased genital infections associated with treatment.
Researchers used CRISPR gene editing to understand how deletions in one area of the genome affect nearby genes. They found that deleting a small region led to increased foetal globin expression and reduced adult globin levels, suggesting a key mechanism for asymptomatic patients with sickle cell disease.
The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors approved funding for 12 new comparative clinical effectiveness research studies focusing on maternal health, autism, and urinary incontinence. These studies will improve healthcare delivery and health outcomes in real-world settings.
A new study found that music therapy sessions improved patients' ability to manage chronic pain and enhance their quality of life. The intervention, which included music-based breathing exercises and progressive muscle relaxation, was feasible, acceptable, and beneficial among adults with Sickle Cell Disease.
A recent study suggests that gene therapy could be a possible cure for sickle cell disease, with the added benefit of preventing complications and extending lifespan. However, the high cost and limited accessibility of this treatment pose significant challenges for patients worldwide.
A recent study found that gene therapy delivery vectors were unlikely to cause the blood malignancies reported in trials; however, the exact cause remains a mystery. The study's results suggest that cancer risk in sickle cell disease may be more complex than initially thought, warranting further investigation.
A new gene therapy called LentiGlobin has been shown to completely eliminate episodes of severe pain caused by sickle cell disease and restore blood cells to their normal shape. The treatment uses a patient's own stem cells and has the potential to give people with this disease their life back.
A doctoral student at Texas A&M University has discovered blood outgrowth endothelial cells (BOECs) as an alternative to induced pluripotent stem cells (IPSCs) for organs-on-chips, offering a cheaper and more accessible option for patient-specific research. The new cells can be isolated from just 50-100 milliliters of blood and have sh...
Researchers at the University of Missouri have developed a free online resource that speeds up data analysis of human genomes three times faster than current methods. This enables scientists to see how an individual's genome makes them susceptible to different diseases in different ways, ultimately reducing associated costs and increas...
A team of Lehigh University undergraduates has won the National Institutes of Health's Healthcare Technologies for Low-Resource Settings Prize for developing a diagnostic device to detect sickle cell disease in infants. The $15,000 prize will support further development and testing of the device, which could improve healthcare outcomes...
A study published in Blood Advances found that hydroxyurea therapy can improve or even reverse cardiac complications in individuals living with sickle cell anemia. The longer duration of treatment was associated with better cardiac outcomes, suggesting the need for early screening and treatment initiation for this patient population.
A new study by Virginia Commonwealth University quantifies the financial impact of sickle cell disease, revealing $15k in lost wages per year per person, totaling $650,000 over a lifetime. The research found that 97% of respondents experienced severe and debilitating pain attacks from sickle cell.
Researchers found that COVID-19 vaccines are more than 95% effective in preventing confirmed infection. The study also revealed significant disparities among different age groups and comorbidities, with higher risks observed among older adults and those with underlying health conditions. Additionally, a separate cohort study showed tha...
A prospective cohort study found that treatment at an infusion center is associated with substantially better outcomes than treatment in the emergency department for patients with sickle cell disease. Patients treated in ICs received parenteral pain medication faster and were more likely to have their pain reassessed within 30 minutes.
Researchers found that children and adults with a history of severe pain episodes or coexisting organ conditions are at higher risk of severe COVID-19 illness. The study recommends vaccination for this medically vulnerable population, particularly those with these comorbidities.
Researchers at Children's Hospital of Philadelphia have developed a proof-of-concept treatment that can elevate adult and fetal hemoglobin levels simultaneously. The approach combines two tactics into a single gene therapy vector, which has shown promising results in vitro.
Researchers used OCT angiography to image patients with sickle cell retinopathy and found that sequential imaging can help assess disease progression and treatment effectiveness. The study showed that untreated patients had more flickering blood vessels, indicating a higher risk of permanent blockage.
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.
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.
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.