Denise Griffiths

TL;DR: In this article, the effect of recombinant human osteogenic protein-1 (OP-1) for the treatment of acute renal failure after 60 min bilateral renal artery occlusion in rats was evaluated.

TL;DR: It is demonstrated for the first time that IGF-1 attenuates the pathological activity of non-neuronal cells that contribute to disease progression and attenuates glial cell-mediated release of tumor necrosis factor-alpha (TNF-alpha) and nitric oxide (NO).

TL;DR: AAV1 was superior to serotypes 2, 5, 7, and 8 in its relative ability to express hASM, alleviate storage accumulation, and correct behavioral deficits, and support the continued development of AAV based vectors for gene therapy of the CNS manifestations in Niemann-Pick type A disease.

TL;DR: Animals treated by combination therapy exhibited high levels of hASM in the viscera and brain, which resulted in near-complete correction of storage throughout the body, resulting in normal weight gain and superior recovery of motor and cognitive functions compared to animals treated by either brain or systemic injection alone.

TL;DR: The effectiveness of intracerebroventricular delivery of recombinant human ASM into ASMKO mice showed that ICV delivery of the enzyme led to widespread distribution of the hydrolase throughout the CNS and a significant reduction in lysosomal accumulation of sphingomyelin was observed throughout the brain and also within the spinal cord and viscera.