The pace of research can seem unreasonably slow; here are a few reasons why
posted on January 1, 2012 - 3:11pm
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human...
posted on January 1, 2012 - 1:42pm
Stem cells have been much in the news lately, including for neuromuscular diseases.
One extremely promising approach has been to create stem cells from the adult (differentiated) cells of humans or animals, and then reprogram them back to a stemlike state, after which they can be redifferentiated into a desired cell type, such as muscle or nerve cells. Stem cells made from differentiated cells...
The search for therapies and cures must be accompanied by the cultivation of the best and brightest researchers
posted on October 1, 2011 - 3:33pm
All MDA grants have the same basic goal: moving promising treatments through the “drug development pipeline,” from early discoveries in the lab, to clinical trials, to actual therapies that can be prescribed in the clinic.
But some MDA grants are designed to accomplish this goal not only by funding research but by encouraging the “best and the brightest” young scientists to become future...
Quest takes a look at gene therapy, 'antisense' and other cutting-edge scientific approaches and how they're being applied to diseases in MDA's program
posted on July 1, 2011 - 4:15pm
Antisense oligonucleotides block flawed genetic instructions
Antisense oligonucleotides — also called antisense, oligos, or simply AONs — are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.
Progress is ongoing in coaxing stem cells along specific paths, altering their genes and understanding the immune response to stem cell transplantation
posted on June 13, 2011 - 2:23pm
Stem cells are a hot topic these days in medicine, science and law, although the term has multiple meanings and it's easy to get confused.
In short, stem cells are cells at an early stage of development from which specialized cells, such as muscle or nerve cells, can develop (in other words, from which these specialized cells "stem").
Different kinds of stem cells are referred to as:
Strategies to fight muscle-fiber fragility have the potential to treat many forms of muscular dystrophy
posted on March 31, 2011 - 11:58am
We hear a lot these days about genes being destiny, about what can and can’t be done to go beyond predetermined biological limits.
A child born with a mutation in the gene for the muscle protein dystrophin, for instance, is destined to develop Duchenne or Becker muscular dystrophy, depending on the precise mutation and other factors.
Video games can serve the higher public good when players work alone or with a team to solve genetic and biochemical puzzles
posted on March 31, 2011 - 11:57am
Note: This article was updated Sept. 19, 2011.
Video games have a reputation for being big time-wasters. But what if you could help solve scientific mysteries — such as unraveling the origins of a genetic disease — at the same time as you’re having fun?
That’s the idea behind several new online games designed to harness the collective brain power of computer users — a process known as “...
