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UrduResearchers at Children’s Hospital of Philadelphia (CHOP) 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 adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. The results were published in Haematologica.
Tag: Sickle Cell Disease
Mount Sinai Ophthalmologists Develop New Technique to Assess Progression of Sickle Cell Retinopathy
(New York, NY – May 10, 2021)- Ophthalmologists at New York Eye and Ear Infirmary of Mount Sinai have created a new technique to evaluate patients with sickle cell retinopathy and assess the disease before it progresses and leads to…
Cecelia Calhoun Named Director of the Adolescent-Young Adult Sickle Cell Program at Yale Cancer Center and Smilow Cancer Hospital
Cecelia Calhoun, MD, MSPH, MBA, has been appointed Assistant Professor of Medicine (Hematology) and Assistant Professor of Pediatrics (Hematology/Oncology) at Yale Cancer Center and Smilow Cancer Hospital
Patients with Sickle Cell–Related Kidney Failure Benefit from Transplants But Are Less Likely to Receive Them
• Patients with kidney failure associated with sickle cell disease benefit from kidney transplants, but they’re less likely than other patients to receive them.
Gene therapy gives man with sickle cell disease the chance for a better future
In July 2019, Evie Junior enrolled in a clinical trial for an experimental stem cell gene therapy for sickle cell disease. The study is led by UCLA Broad Stem Cell Research Center physician-scientists Dr. Donald Kohn and Dr. Gary Schiller and funded by the California Institute for Regenerative Medicine.
Intellia Therapeutics Receives Grant to Develop Curative CRISPR/Cas9 In Vivo Sickle Cell Disease Treatments
Intellia Therapeutics Receives Grant to Develop Curative CRISPR/Cas9 In Vivo Sickle Cell Disease Treatments
Lost in Transition: Sickle Cell Clinic Helps Adults Continue Care
Networking California for Sickle Cell Care Initiative supports expansion of San Diego’s only adult sickle cell disease clinic at UC San Diego Health which offers child-to-adult transition health services to patients like Vanessa Hughes.
Understanding Sickle Cell Disease
Sickle cell disease is a lifelong inherited disorder of the red blood cells that poses many challenges for the people and families living with it. Rutgers Cancer Institute expert shares more on the basics of sickle cell disease and how the illness is managed.
Gut Microbiome Translates Stress Into Sickle Cell Crises
A new study shows how chronic psychological stress leads to painful vessel-clogging episodes—the most common complication of sickle-cell disease (SCD) and a frequent cause of hospitalizations. The findings, made in mice, show that the gut microbiome plays a key role in triggering those episodes and reveals possible ways to prevent them. The research was conducted by scientists at Albert Einstein College of Medicine and published online today in Immunity.
COVID-19: What you should know if you have sickle cell disease
If you have sickle cell disease, you are at higher risk than the general population for severe complications from COVID-19.
NIH announces $1 million prize competition to target global disease diagnostics
The National Institutes of Health has launched a $1 million Technology Accelerator Challenge (TAC) to spur the design and development of non-invasive, handheld, digital technologies to detect, diagnose and guide therapies for diseases with high global and public health impact. The Challenge is focused on sickle cell disease, malaria and anemia and is led by NIH’s National Institute of Biomedical Imaging and Bioengineering (NIBIB).
While Promoting Diseases Like Cancer, These Enzymes Also Cannibalize Each Other
In diseases like cancer, atherosclerosis, and sickle cell anemia, cathepsins promote their propagation. Drug trials to inhibit these enzymes have failed due to baffling side effects. Now a new study examines cathepsins in systems to remove some of the bafflement.
Yale Cancer Center researchers show identifying type of chronic pain in adults with sickle cell disease may lead to better outcomes
Identifying the type of pain an adult with sickle cell disease (SCD) experiences may be useful in improving treatment, according to a new study by researchers at Yale Cancer Center (YCC) and Smilow Cancer Hospital.
Likelihood of Prenatal Screening for Blood Disorders Varies Between Type of Healthcare Provider
A Rutgers Cancer Institute of New Jersey investigator and other collaborators examined prenatal screening practices for blood disorders between different types of care providers and found different variations in prenatal guidance.