Free Republic

The Food and Drug Administration has approved the world's first medicine employing CRISPR gene-editing technology to treat sickle cell disease, giving thousands new hope to treat the painful disease. "Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today," said Nicole Verdun, director of the Office of Therapeutic Products within the FDA's Center for Biologics Evaluation and Research. Two new treatments, Casgevy and Lyfgenia, were approved on Friday...

BOSTON (Reuters) – Bone marrow transplants, already used to treat some children with sickle cell disease, also may cure some adults with this deadly genetic defect that causes red blood cells to contort, U.S. scientists said on Wednesday. > Destroying a patient's bone marrow and replacing it with healthy marrow from a donor, often a sibling, is considered too risky for adults. > This approach leaves enough space inside the patient's bones for the donated marrow to find a home and produce enough healthy red blood cells to compensate for the defective ones. >

In a study to be published in the January 2006 issue of Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells. This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology. To prevent the production of the abnormal hemoglobin that causes sickle cell disease, a viral vector was introduced in cell cultures of patients who have the disease. The vector carried...

<p>Washington, DC, May. 11 (UPI) -- The U.S. Senate approved Sen. Jim Talent's, R-Mo., legislation expanding services and treatment for patients afflicted with sickle cell blood disorder.</p> <p>The Sickle Cell Treatment Act authorizes federal matching funds for disease-related services under Medicaid as well as increasing state funding for physician and laboratory services.</p>

WASHINGTON, Feb. 25 — High blood pressure in the lungs has been identified as a major risk factor for death in adults with sickle-cell disease, and all people with the disease should be screened for this complication, according to a study being published Thursday. Almost one-third of adult sickle-cell patients who were screened with a noninvasive ultrasound method were found to have moderate to severe pulmonary hypertension and high blood pressure in the lungs that had not previously been detected, the study found. The results from the tests confirm earlier suggestions from studies of patient records that pulmonary hypertension is...