It’s only been seven years since scientists first learned how to precisely and reliably splice the human genome using a tool called CRISPR, making it possible to think about snipping out disease-causing mutations and actually cure, once and for all, genetic diseases ranging from sickle cell anemia to certain types of cancer and even blindness. …
BACKGROUND: Pain is the hallmark of sickle cell disease (SCD) and it can be severe, frequent and unpredictable. Although nociceptive pain is more common, at times, people with SCD may have neuropathic pain. The latter can occur due to peripheral or central nerve injury. This review is focused on identifying treatment of only painful sensory …
Abstract Recently, gene therapy clinical trials have been successfully applied to hemoglobinopathies, such as sickle cell disease (SCD) and β-thalassemia. Among the great discoveries that led to the design of genetic approaches to cure these disorders is the discovery of the β-globin locus control region (LCR) and several associated transcription factors, which determine hemoglobin switching …
OBJECTIVES: To capture evidence of the efficacy and safety of pharmacological analgesia for uncomplicated acute sickle-cell pain in pediatric patients compared to placebo. SOURCES OF DATA: Searches for key evidence were performed from March 1 to 31, 2018, for randomized controlled trials of pharmacological analgesia compared to placebo for uncomplicated acute sickle-cell pain in a …
Garrett: ‘It’s like a double whammy. Making it to the league and doing it with the disease. I hope it’s an inspiration to a lot of the kids with sickle cell.’ About 100,000 Americans are affected by sickle cell disease, according to the Centers for Disease Control and Prevention. Around 500 athletes play in the NBA …
The first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder. “It is just amazing how far things have come,” says Victoria Gray, 34, of Forest, Miss. “It is wonderful,” she told NPR in an exclusive interview after undergoing the landmark treatment …
