LIG4 syndrome is an exceptionally rare disorder caused by a genetic mutation that prevents the body from repairing damaged ...
Marcelo was diagnosed with Treacher Collins syndrome, a rare condition affecting facial bone structure and cartilage. The ...
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
A new TLC reality series, Jay & Pamela, is just around the corner—and its stars are an engaged couple born with the same ...
The early stage company is developing a new approach to gene editing to treat rare genetic diseases ... investment and must achieve a lot with very little until they prove themselves and gain ...
Ozzy Osbourne has been battling a rare form of Parkinson's Disease known as Parkin 2. In 2020, he disclosed to his fans that ...
a rare and fatal genetic disorder affecting the central nervous system. On the very day Noa was diagnosed, an experimental gene therapy and clinical trial were posted to treat Canavan disease.
A new study shows that long-read sequencing has the potential to improve the rate of diagnosis while reducing the time to diagnosis from years to days -- in a single test and at a much lower cost.
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...
"This type of gene editing should also allow for the treatment of diseases whose origin is not genetic or whose origin cannot ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback