Amyotrophic lateral sclerosis (ALS) is a fatal neuromuscular disease in which patients gradually become paralyzed due to loss of motor function. Many genetically inheritable mutations have been linked to ALS; however, the majority of ALS patients are considered sporadic.
Amyotrophic lateral sclerosis (ALS) is a late-onset neuromuscular disease with no cure and limited treatment options. Patients experience a gradual paralysis leading to death from respiratory complications on average only 2-5 years after diagnosis.
Human induced-pluripotent stem cells are a promising resource for propagation of myogenic progenitors. Our group recently reported a unique protocol for the derivation of myogenic progenitors directly (without genetic modification) from human pluripotent cells using free-floating spherical culture.
Our current research is to apply stem cell technology to disease modeling and therapeutic applications for neuromuscular diseases such as ALS (also known as Lou Gehrig’s disease).
Title: Skeletal muscle cells derived from human pluripotent stem cells Legend: Suzuki lab recently initiated a new project to establish skeletal muscle progenitor/stem cells derived from human pluripotent sources. Our culture method can produce skeletal …
“The novelty is that this is a combined cell and gene therapy approach,” Suzuki explains, noting that the bone marrow stem cells on their own had a modest effect, possibly by releasing their own protective factors. “But only when we engineered the cells to release GDNF did we see a significant improvement. The cells turned out to be quite an important component. It’s this combination of cells and drug delivery that seems to be so effective.”
Human neural stem cells, exposed in a lab dish to the steroid DHEA, exhibit a remarkable uptick in growth rates, suggesting that the hormone may play a role in helping the brain produce new cells.