Search Results - "Zhao, Michael A"

Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse by Passini, Marco A., Bu, Jie, Fidler, Jonathan A., Ziegler, Robin J., Foley, Joseph W., Dodge, James C., Yang, Wendy W., Clarke, Jennifer, Taksir, Tatyana V., Griffiths, Denise A., Zhao, Michael A., O'Riordan, Catherine R., Schuchman, Edward H., Shihabuddin, Lamya S., Cheng, Seng H.

“…Niemann-Pick disease (NPD) is caused by the loss of acid sphingomyelinase (ASM) activity, which results in widespread accumulation of undegraded lipids in…”
Get full text

Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease by James C. Dodge, Jennifer Clarke, Antonius Song, Jie Bu, Yang, Wendy, Tatyana V. Taksir, Denise Griffiths, Michael A. Zhao, Schuchman, Edward H., Cheng, Seng H., Catherine R. O'Riordan, Shihabuddin, Lamya S., Marco A. Passini, Stewart, Gregory R.

“…Niemann-Pick type A disease is a lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. Previously we showed that storage…”
Get full text

Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice by Ziegler, Robin J, Bercury, Scott D, Fidler, Jonathan, Zhao, Michael A, Foley, Joseph, Taksir, Tatyana V, Ryan, Susan, Hodges, Bradley L, Scheule, Ronald K, Shihabuddin, Lamya S, Cheng, Seng H

“…The availability of a murine model of Pompe disease has enabled an evaluation of the relative merits of various therapeutic paradigms, including gene therapy…”
Get more information

417. Combination Brain and Systemic Injections of AAV Results in Whole Body Therapy and Extension of Lifespan in the Niemann-Pick Mouse by Passini, Marco A, Bu, Jie, Fidler, Jonathan A, Foley, Joseph W, Dodge, James C, Ziegler, Robin J, Yang, Wendy W, Clarke, Jennifer, Taksir, Tatyana V, Griffiths, Denise A, Zhao, Michael A, O'riordan, Catherine R, Shihabuddin, Lamya S, Schuchman, Edward H, Cheng, Seng H

“…The majority of lysosomal storage diseases contain both CNS and visceral pathology. Many experimental designs use either intracranial injection of viral…”
Get full text