8 New Genetic Mutations Linked to Rare Cognitive Disorder …
WEBSep 27, 2024 · CSF1R-Related Disorder is a rare genetic condition causing cognitive decline. Mayo Clinic researchers recently identified eight new mutations, paving the way for personalized treatments and improved diagnosis. Further research is needed to enhance genetic counseling and develop interventions for …
Scientists restore brain cells impaired by a rare genetic disorder - NPR
WEBApr 30, 2024 · Scientists have found a way to restore brain cells impaired by a rare and life-threatening genetic disorder called Timothy syndrome. A type of drug known as an antisense oligonucleotide allowed...
The world's 1st CRISPR therapy has been approved. Here's …
WEBJan 18, 2024 · Drug regulators have approved a CRISPR therapy called Casgevy to treat inherited blood disorders. But what is it and how does it work?
CRISPR Gene-Editing Breakthrough Opens Door To Treating Broad …
WEBJun 26, 2021 · Scientists successfully treated a rare disease with the experimental gene-editing technique. It could open the door to new ways of treating more common disorders in the future.
Experimental gene therapy restores some vision in patients with ...
WEBMay 6, 2024 · The trial, which started in 2019, enrolled 12 adults, ages 17 to 63, and two children, ages 9 and 14, with inherited retinal degeneration caused by mutations in the CEP290 gene. That gene...
FDA approves first genetic treatments for sickle cell disease : …
WEBDec 8, 2023 · The FDA approved two gene therapies for anyone 12 and older suffering from the most severe form of sickle cell disease, a brutal blood disorder that has long been neglected by medical research.
Live-cell model system can decode genetic risk for psychiatric disorders
WEB3 days ago · For many years, scientists have known that genetic variants, or differences in DNA code across people, play some role in neurological and psychiatric disorders. But the details were murky. Now ...
New approach successfully traces genomic variants back to genetic disorders
WEBJan 5, 2023 · NHGRI researchers identified a person with a genomic variant associated with a known metabolic disorder. Further testing found that the individual had high levels of certain chemicals in their body associated with the …
WHO issues new recommendations on human genome editing for …
WEBDec 7, 2021 · Two new companion reports released today by the World Health Organization (WHO) provide the first global recommendations to help establish human genome editing as a tool for public health, with an emphasis on …
The first gene-editing treatment: 10 Breakthrough Technologies …
WEBJan 8, 2024 · The first gene-editing treatment: 10 Breakthrough Technologies 2024. Sickle-cell disease is the first illness to be beaten by CRISPR, but the new treatment comes with an expected price tag of...