“On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition.

For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.

For a lucky few, like Kendric, the treatment could make possible lives they have longed for.”

From New York Times.

“An artificial intelligence system has proven it can save lives by warning physicians to check on patients whose heart test results indicate a high risk of dying. In a randomised clinical trial with almost 16,000 patients at two hospitals, the AI reduced overall deaths among high-risk patients by 31 per cent.”

From New Scientist.

“For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she could not make out people’s faces, only their silhouettes.

But after receiving an experimental gene-editing treatment to one of her eyes, she now can see things she never saw before.

Cook was born with an inherited retinal disorder that causes blindness, a rare type of eye disorder historically called Leber congenital amaurosis or LCA. A few years ago, she decided to participate in a clinical trial that involved using the gene-editing tool CRISPR to correct the form of inherited blindness that she has…

 This study is the first time that CRISPR has been used in the eyes of living people.

‘The results of this study provide proof of concept that CRISPR-Cas9 gene editing can be used safely and effectively to treat inherited retinal disorders,’ said the study’s first author Dr. Eric Pierce.”

From CNN.

“In a first-ever human clinical trial of four adult patients, an mRNA cancer vaccine developed at the University of Florida quickly reprogrammed the immune system to attack glioblastoma, the most aggressive and lethal brain tumor…

Reported May 1 in the journal Cell, the discovery represents a potential new way to recruit the immune system to fight notoriously treatment-resistant cancers using an iteration of mRNA technology and lipid nanoparticles, similar to COVID-19 vaccines, but with two key differences: use of a patient’s own tumor cells to create a personalized vaccine, and a newly engineered complex delivery mechanism within the vaccine.”

From Medical Xpress.