Novel gene therapy platform restores muscle function in models of Duchenne muscular dystrophy
Duchenne muscular dystrophy is a genetic disorder that leaves people unable to make an important protein called dystrophin, resulting in muscle weakness and degeneration
Current viral-based gene therapies are not very effective, in part because they cannot carry the full set of genetic instructions due to their limited size
New platform uses natural extracellular vesicles that are large enough to carry the full set of genetic instructions...
EHA: Oral combination shows high response rates in patients with hard-to-treat acute myeloid leukemia
Relapsed or refractory acute myeloid leukemia (AML) represents one of the most challenging hematologic cancers
The menin inhibitor revumenib...
Researchers uncover how aging cells may trigger heart attacks and strokes
Some cancer treatments come with a risk of cardiovascular side effects, highlighting a need to understand underlying causes
Researchers discovered...
Early immunotherapy aids in treating potentially fatal fungal pneumonias in preclinical models
Lung infections caused by opportunistic molds can “paralyze” the immune system
Researchers tested adding immunotherapy on top of standard antifungal treatments
This approach reversed immune system paralysis, leading to better survival and less severe illness in lab models, especially when immunotherapy was administered early
Study demonstrates a potentially clinically relevant strategy for deploying...