(BOSTON, September 6, 2016) -- A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle ...

Researchers have cleared the last scientific hurdle to a clinical trial of gene therapy to cure sickle cell disease, they reported on Tuesday, fueling hopes that they will begin enrolling patients ...

The neurotransmitter dopamine influences the activity of a wide variety of brain areas. A deficiency of this substance can have drastic consequences: The death of dopamine-producing nerve cells in the ...

Scientists have found a gene responsible for an intellectual disability disorder and proven how it works. The research, details the role of a gene called BCL11A in a new intellectual disability ...

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...

A team at Dana Farber/Boston Children’s has been able to selectively silence a gene, BCL11A, found in 2008 to be an integral part of a potential Sickle Cell cure using gene therapy. Sickle Cell ...

Sickle cell disease is the most common red blood cell disorder in the United States, having been diagnosed in approximately 100,000 African Americans alone. The lack of oxygen from sickle cell buildup ...

Doctors who treated patients with sickle cell disease who received an infusion of gene-edited treatment have called it "transformative" for those who live with a chronic condition. The technique calls ...

A new study identifies a gene that is especially active in aggressive subtypes of breast cancer. The research suggests that an overactive BCL11A gene drives triple-negative breast cancer development ...

The switch from fetal to adult hemoglobin expression in humans is related to the active repression of HbF expression by BCL11A. A self-inactivating lentivirus vector with a short hairpin RNA designed ...

A direct consequence of natural selection among Homo sapiens, sickle cell disease (SCD) has afflicted a large segment of the population for several millennia. However, with the rapid rise of new ...