UK first to approve CRISPR treatment for diseases: what you need to know
C Wong. Nature. November 16, 2023
From the article: "In a world first, the UK medicines regulator has approved a therapy that uses CRISPR gene editing as a treatment for diseases. The decision marks another high point for a biotechnology that has regularly been lauded as revolutionary in the decade since its discovery. The therapy will treat the blood conditions sickle-cell disease and ß-thalassaemia. Sickle-cell disease, also known as sickle-cell anaemia, can cause debilitating pain, and people with ß-thalassaemia can require regular blood transfusion. "
Genetic Variation and Sickle Cell Disease Severity: A Systematic Review and Meta-Analysis.
Justin K Kirkham et al. JAMA Netw Open 2023 10 (10) e2337484
From the abstract: " What genetic modifiers of sickle cell disease (SCD) are currently defined, and what are potential approaches to improve future studies?
In this systematic review and meta-analysis of 571 studies examining 29?670 individuals with SCD, 17?757 associations involving 1552 genes and 25 SCD phenotype categories were discovered; of these, only 173 associations met the study design, reporting, and phenotype or genotype harmonization required for meta-analysis. Gene variants regulating fetal hemoglobin and a-thalassemia were frequently identified, but other associations remained unconfirmed."
Pregnant and Living with Sickle Cell Disease: A Push for Better Outcomes
NIH, September 2023
From the website: " Experts say that medical advances in care and disease-modifying therapies have helped many people living with SCD survive well through their reproductive years. For parents-to-be, that means awareness is key. Individuals with SCD are at higher risk than the general population for preeclampsia, as Found discovered; but those with preeclampsia can go on to develop a condition called eclampsia, which can lead to seizures and even coma. People with SCD are also at higher risk for sepsis and blood clots. And there are risks for the fetus, such as lower-than-normal growth in the womb, preterm delivery, and stillbirth."
A Proclamation on National Sickle Cell Awareness Month, 2023
The White House, September 2023.
During National Sickle Cell Awareness Month, we recognize the perseverance and strength of the community of people living with this disease and recommit to developing more effective treatments. Approximately 100,000 Americans have Sickle Cell Disease (SCD) — a group of inherited red blood cell disorders that can cause acute, chronic pain and serious health complications, including infections, strokes, organ damage, vision problems, and serious fatigue. Living with SCD often means putting the goals and plans of everyday life on hold to accommodate the demands of the disease, enduring frequent unplanned hospital stays and struggling to pay for costly treatments not covered by insurance.