Sickle Cell Disease is an awful genetic disease that disproportionally affects black people. It's caused by a single-point mutation in DNA, which results in a modified hemoglobin protein, differing by only one amino acid. While this may sound insignificant, it's anything but. Simple organic chemistry explains why this change profoundly affects those unfortunate enough to inherit the disease, which is characterized by abnormal hemoglobin.
sickle cell disease
Exa-cel, a new CRISPR-based treatment, modifies the genes of the patient's stem cells to induce them to produce fetal hemoglobin.
To underscore how important the battle for its eradication still is, misperceptions are clarified and key aspects of the inherited illness are addressed here.
Here are the final four exciting developments in science, health and technology of 2017. And, a prediction for what innovation could be truly disruptive in the future.
The actor and musician knows how to convey a message that effects medical change, helped in part by his visit top be with sick kids at Children's Hospital Los Angeles. Now, let's see if Hollywood follows suit.
ACSH's Director of Medicine, Dr. Jamie Wells, traveled to Washington, DC to tape at Al Jazeera for a live television program. It engaged a global discussion on sickle cell anemia, its perils and the advances in gene therapy that are showing great promise for this genetic disease.
A number of recent headlines imply that a new case study in the New England Journal of Medicine proves that gene therapy has cured sickle cell disease — a genetic disorder that causes tremendous pain, suffering and diminished life expectancy. Let's unpack the significance of the researchers’ findings.
A small study conducted by the National Institutes of Health shows bone marrow transplantation as an effective means to treat severe sickle cell