A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells don't develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received experimental gene therapy co-developed by NIH scientists. They experienced substantial improvements in immune system function and normal growth up to two years after treatment.
Gene therapy developed at St. Jude Children's Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time. The gene therapy involved collecting patients' bone marrow, then using a virus as a vector to insert a correct copy of the IL2RG gene into the genome (DNA) of patients' blood stem cells.
A new Canadian study of more than 2,400 families suggests that among preschoolers, spending two hours or more of screen time per day is linked to clinically significant behavioural problems.
Using CRISPR gene editing, a team has thwarted a lethal lung disease, in an animal model, in which a harmful mutation causes death within hours after birth. This proof-of-concept study showed that in utero editing could be a promising new approach for treating lung diseases before birth.
Using CRISPR gene editing, researchers have thwarted a lethal lung disease in an animal model in which a harmful mutation causes death within hours after birth. This proof-of-concept study showed that in utero editing could be a promising new approach for treating lung diseases before birth.
Offering universal late pregnancy ultrasounds at 36 weeks would benefit mothers and babies and could be cost saving. A new study shows that an additional routine ultrasound could eliminate undiagnosed breech presentation of babies, lower the rate of emergency caesarean sections, and improve the health of mothers and babies.
An NYU pediatrician and researcher writes in JAMA Pediatrics that new recommendations on testing children for lead are inconclusive, but do not mean that we should abandon screening children for elevated lead levels.
This recommendation from the US Preventive Services Task Force (USPSTF) updates its 2006 statement regarding screening for elevated blood lead levels in children and pregnant women.
Researchers from Children's Hospital Colorado and the University of Colorado School of Medicine in Aurora, Colorado, have found that abnormal performance on the Romberg balance test can indicate that children and adolescents will experience prolonged symptoms following sports-related concussion.
Intimate partner homicide among teens does occur and 90% of the victims are girls, according to a new study in JAMA Pediatrics.