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CRISPR is a promising treatment for blood disorders, but it comes with challenges, such as off-target effects.
Cleveland Clinic researchers depleted BACE1 to reverse amyloid plaque formation in mice with Alzheimer's disease.
Two recent advances are focused on using engineered T cells and drug nanocrystals to combat the HIV “reservoirs” that hide in the body and cause the disease to…
A stem cell pathway could be targeted to create more effective treatments for triple-negative breast cancer.
A novel therapy enhances the reception of the cartilage growth signals while blocking the inflammatory ones that lead to joint degeneration.
Targeting "clusters" of mutations with CRISPR could lead to a treatment that could work for up to 60% of patients with DMD.
French scientists discovered that an X chromosome-linked abundance of a toll-like receptor could be the root cause of lupus.
European researchers discovered how drugs bind to the enzyme DHODH, which could lead to more selective cancer drugs.
A team at Duke University has created the first functioning human muscle from skin cell-derived induced pluripotent stem cells.
Alnylam, which put its hemophilia program on hold in September, has gotten the all-clear from the FDA to resume trials.