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UrduUCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 gene-editing technology in humans to directly correct the genetic mutation that causes the disease.
Tag: Sickle Cell Anemia
Non-invasive eye test for multiple diseases to advance under $4.8M NIH award
Researchers at the IU School of Optometry and IU Luddy School of Informatics, Computing and Engineering will advance next-generation technology that uses a simple eye scan to assist in the early detection of conditions such as Alzheimer’s disease, diabetes and heart disease.
Researchers Publish Final Results of Key Clinical Trial for Gene Therapy for Sickle Cell Disease
An international consortium published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
Children’s Hospital Los Angeles Study Finds Many Kids With Sickle Cell Anemia Lack Preventative Care
Press Release EMBARGOED until Wednesday, March 6, 12:05 AM EST LOS ANGELES (March 6, 2024)—Children with sickle cell anemia are vulnerable to serious infections and stroke, but many do not receive the preventative care that could help them stay healthier…
Sickle Cell Disease Continues to Face Underfunding, Lack of Research
Over the years, dedicated sickle cell disease programs and research initiatives have greatly improved patient care and life expectancy. But, giving these patients the care they require still presents a number of challenges, including inadequate funding for sickle cell programs, lack of research, and limited access to healthcare.
New study shows promising evidence for sickle cell gene therapy
UChicago Medicine Comer Children’s Hospital was one of three sites to enroll patients in a clinical trial to test a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.
CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin
Researchers 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.
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.
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.