Month: January 2021

Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently used in gene therapy trials for those conditions. The vector, an engineered vehicle for delivering functional copies of the hemoglobin gene to correct a genetic abnormality, leads to the production of more hemoglobin with a lower dose, minimizing the risk of toxic side effects.

The findings were published today in Molecular Therapy.

“These results have many potential benefits for the successful treatment of

What if scientists knew exactly what impact the SARS-CoV-2 virus had inside our lung cells, within the first few hours of being infected? Could they use that information to find drugs that would disrupt the virus’ replication process before it ever gets fully underway? The discovery that several existing FDA-approved drugs—including some originally designed to fight cancer—can stop coronavirus in its tracks indicates the answer is a resounding yes.

A team of Boston University researchers—hailing from BU’s National Emerging Infectious Diseases Laboratories (NEIDL),

Scientists have broadened our understanding of how ‘weak’ cells bond with their more mature cellular counterparts to boost the body’s production of insulin, improving our knowledge of the processes leading to type 2 diabetes—a significant global health problem.

Type 2 diabetes mellitus occurs when β-cells cannot release enough insulin—a tightly controlled process requiring hundreds of such cells clustered together to co-ordinate their response to signals from food, such as sugar, fat and gut hormones.

An international research team—led by scientists at the University of Birmingham—have discovered that immature β-cells (PDX1LOW/MAFALOW) are able to overcome their relative