In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
It is a detailed view of the brain like never seen before. In a significant technological leap, University of Florida ...
Secreted PD-L1 alleviates inflammatory arthritis in mice through local and systemic AAV gene therapy
We choose AAV8 due to the superior liver-directed transduction efficiency of AAV8 relative to other AAV serotypes (19). Reduced cytokine levels in serum from CIA mice intravenously injected with ...
A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.
Western blot analysis of the levels of FLAG-tagged AAV DddA or BspD61C (L) and HA-tagged AAV TadA8e (H) in skeletal muscle (quadriceps) of mice at 4 weeks post-injection. Coomassie Brilliant Blue (CBB ...
A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...
This vector was administered into the cerebral ventricular spaces of Scn1b-null mice. Following treatment, the AAV-Navβ1 vector successfully expressed the β1 subunit protein in both excitatory ...
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