Cardiology
FDA advisors to weigh new gene therapy for sickle cell anemia
Patients with sickle cell disease may soon have two new treatments to try.
Oct 30, 2023
0
1
Page 11 - News tagged with gene therapy
Diseases, Conditions, Syndromes
First ever gene therapy trial for children with Hunter syndrome opens
The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.
Oct 30, 2023
0
0
Psychology & Psychiatry
Why do people with hoarding disorder hoard, and how can we help?
Hoarding disorder is an under-recognized serious mental illness that worsens with age. It affects 2.5% of the working-age population and 7% of older adults. That's about 715,000 Australians.
Oct 25, 2023
0
1
Genetics
Team looking at gene therapy for children paralyzed by rare mutations
The 50 families stretch from the Netherlands and the United Kingdom to the United States and China. Each family has a child who is paralyzed from a mutation in a single gene named Contactin-Associated Protein 1 (Cntnap1).
Oct 20, 2023
1
33
Diseases, Conditions, Syndromes
Human lung chip leveraged to faithfully model radiation-induced lung injury
The lung is one of the tissues most sensitive to radiation in the human body. People exposed to high radiation doses following nuclear incidents develop radiation-induced lung injury (RILI), which affects the function of ...
Oct 17, 2023
0
22
Genetics
Researchers work to preserve fertility for people undergoing gene therapy
Researchers at the National Institutes of Health have created a novel gene therapy procedure that could preserve fertility in people with sickle cell disease and other genetic blood conditions. Infertility is a high-risk ...
Oct 12, 2023
0
12
Medical research
Restoring nerve-muscle connections boosts strength of aging mice, study finds
A small molecule previously shown to enhance strength in injured or old laboratory mice does so by restoring lost connections between nerves and muscle fibers, Stanford Medicine researchers have found.
Oct 12, 2023
0
96
Genetics
3D genome architecture influences SCID-X1 gene therapy success
Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), sometimes called "bubble boy disease," are born with a defective gene that prevents them from producing immune cells. Gene therapy from St. Jude ...
Oct 6, 2023
0
26
Oncology & Cancer
An ancient anti-cancer mechanism: DISE
A new editorial paper titled "DISE, an ancient anti-cancer mechanism that senses mutational load in cancerous cells?" has been published in Oncotarget.
Oct 3, 2023
0
1
Medical research
Katalin Kariko, scientific maverick who paved way for mRNA vaccines
Hungarian-born scientist Katalin Kariko's obsession with researching a substance called mRNA to fight disease once cost her a faculty position at a prestigious US university, which dismissed the idea as a dead end.
Oct 2, 2023
0
46
