News

Understanding one gene's role in different neurodevelopmental disorders
Researchers have identified how variations in a gene called TRIO can influence brain functions and result in distinct ...
Rolling Out1d
How genetic disease imprisons your backbone
Ankylosing spondylitis causes chronic back pain and joint fusion, affecting millions. Learn the early warning signs and what ...
Healio2d
FDA grants rare pediatric disease designation for Stargardt disease gene therapy
The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated ...
News-Medical.Net on MSN52m
Genetic analysis shows leprosy was endemic in pre-colonial America
Long considered a disease brought to the Americas by European colonizers, leprosy may actually have a much older history on ...
ZME Science on MSN3d
This Baby’s One-in-a-Million Genetic Disorder Had No Cure. So Scientists Designed One Just for Him
KJ Muldoon came into the world with a genetic time bomb ticking inside him.
Good News Network4d
Infant With Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment
In medical breakthrough, a baby diagnosed with rare genetic disorder is thriving after customized CRISPR gene editing therapy ...
Smithsonian Magazine on MSN9d
In a Remarkable First, a Baby With a Rare Disease Receives Personalized Gene Therapy
Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases ...
Doctors Achieve Medical Breakthrough Using Gene-Editing Therapy to Heal Baby with Rare Disorder
Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the ...
BioSpace2d
Rocket’s Danon Disease Gene Therapy on Hold After Patient Death
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of ...
Ocugen wins FDA rare pediatric disease status for genetic eye disorder treatment
Ocugen ( NASDAQ: OCGN) disclosed on Tuesday that its gene therapy candidate, OCU410ST, has been granted rare pediatric ...
How much of your disease risk is genetic? It's complicated.
Environmental factors such as lifestyle and the medications you take influence the effects your genes have on your body — and can clarify how diseases develop.
Hawai‘i Public Radio57m
Hilo High graduate and Nobel Prize winner on pioneering CRISPR gene editing
In the news earlier this month was a story about baby KJ, who made headlines because of a custom treatment he received for a ...