A CRISPR/Cas9 gene editing system can be used to remove the repeat expansion in the C9ORF72 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), a new study reports. The system showed positive proof-of-concept effects in mouse models and in human cells, reducing the amount of faulty…
CRISPR/Cas9 Gene Editing Used to ‘Cut Out’ ALS Mutation: Early Study
ALS News Today brought consistent coverage of the latest research developments and advances in treatment related to amyotrophic lateral sclerosis (ALS) throughout 2022. We look forward to continuing to serve as a resource for the ALS community in the new year. Here, we’ve compiled a list of the…
Dependency is a difficult reality of ALS. My husband, Todd, fought to preserve his independence as long as possible, but he lost each battle to progressive disability. When his legs became noticeably weak, he got a walking stick, and then a scooter, followed by a power wheelchair. He lost…
Patient registries help record how many cases of a particular disease there are across the country, and many also log related genetic, clinical, and biological information. Some U.S. registries, including those dealing with infectious diseases, have mandatory reporting requirements. But for many other diseases, including amyotrophic lateral sclerosis (ALS),…
During my first year living with ALS, I spent a lot of time researching and learning whatever I could about the disease. I came across long lists of medical equipment that patients like me could expect to use when ALS symptoms progressed. Books written by medical experts with…
In amyotrophic lateral sclerosis (ALS), as in many diseases, patient registries, biorepositories, and natural history studies are helpful both in planning clinical trials and as tools for scientists to learn more about how a person’s lifestyle, genetics, and environment can potentially lead to…
Healthcare has long harnessed the power of big data. Examples range from the Human Genome Project, a worldwide 13-year effort to map DNA, to the adoption of electronic medical records— allowing doctors to quickly access patient information at points of care — and the rise of personalized medicine, which tailors…
Note: This column includes a mention of suicide. The most powerful force within people is the impulse to stay alive. Our survival instinct is so strong that we’re willing and inexplicably able to perform inhuman feats and take unimaginable risks. According to psychologist Abraham Maslow, survival is the foundation…
I feel compelled to seek out beauty and create a warm home, perhaps partly as an antidote to the difficulties we experience because my husband, Todd, has ALS. In a way, choosing to celebrate feels like a revolt against this devastating disease. A little over a week ago, I…
EpiSwitch, a non-invasive, blood-based test developed by Oxford BioDynamics, successfully stratified patients with amyotrophic lateral sclerosis (ALS) as fast versus slow progressors, according to an interim analysis of the REFINE-ALS study. These findings highlight the potential of the biomarker test to better classify disease progression in ALS patients…
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