... syndrome , chronic pancreatitis , hepatic steatosis (fatty liver), cystic fibrosis , several mitochondrial neuropathies and myopathies, myotonic dystrophy
, Friedreich's ataxia , some of the inherited forms of neonatal hyperinsulinism and many others. Risk of diabetes is higher with chronic use of ...
... to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis , hemophilia , muscular dystrophy
and sickle cell anemia . However, this has been much harder than modifying simple bacteria, primarily because of the problems involved in carrying ...
... or decreased muscle tone, respectively.
The myopathies are all diseases affecting the muscle itself, rather than its nervous control.
is a large group of diseases, many of them hereditary, where the muscle integrity is disrupted. It leads to progressive loss of strength, high ...
... to form specific types of brain cells. This could lead to new treatments and cures for diseases like Lou Gehrig's disease , muscular dystrophy
, and spinal cord injuries.
Researchers at the University of Wisconsin-Madison coaxed human embryonic stem cells into becoming neural stem ...
... X syndrome
Duchenne and Becker's muscular dystrophy
nonsyndromic deafness and X-linked nonsyndromic deafness
spinal and bulbar muscular atrophy
X-linked severe combined ...