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Full-Text Articles in Analytical, Diagnostic and Therapeutic Techniques and Equipment

Bridging From Intramuscular To Limb Perfusion Delivery Of Raav: Optimization In A Non-Human Primate Study, Alisha Gruntman, Gwladys Gernoux, Qiushi Tang, Guo-Jie Ye, Dave R. Knop, Gensheng Wang, Janet Benson, Kristen E. Coleman, Allison M. Keeler, Christian Mueller, Louis G. Chicoine, Jeffrey D. Chulay, Terence R. Flotte May 2019

Bridging From Intramuscular To Limb Perfusion Delivery Of Raav: Optimization In A Non-Human Primate Study, Alisha Gruntman, Gwladys Gernoux, Qiushi Tang, Guo-Jie Ye, Dave R. Knop, Gensheng Wang, Janet Benson, Kristen E. Coleman, Allison M. Keeler, Christian Mueller, Louis G. Chicoine, Jeffrey D. Chulay, Terence R. Flotte

Christian Mueller

Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of serum alpha-1 antitrypsin (AAT) deficiency have shown long-term (5-year) stable transgene expression at approximately 2% to 3% of therapeutic levels, arguing for the long-term viability of this approach to gene replacement of secreted serum protein deficiencies. However, achieving these levels required 100 IM injections to deliver 135 mL of vector, and further dose escalation is limited by the scalability of direct IM injection. To further advance the dose escalation, we sought to bridge the rAAV-AAT clinical development …


Effect Of Exercise Position On Percent Changes In Serratus Anterior Muscle Thickness, Joseph M. Day Mar 2019

Effect Of Exercise Position On Percent Changes In Serratus Anterior Muscle Thickness, Joseph M. Day

Joseph M. "Matt" Day

Objective: A reliable method for measuring SA thickness with ultrasound imaging has been reported, yet the ability to detect differences in levels of contractility has yet to be established. The purpose of this study was to determine if there are differences in the percent change in thickness of the SA during four different exercise positions performed by healthy individuals.

Design: Observational study

Setting: Laboratory

Participants: Twenty-eight healthy participants, mean age 26 (SD 4, 57% female, 100% right handed)

Methods: Volunteers were positioned in a standardized sitting posture and anatomical landmarks were marked for ultrasound probe positioning. Two resting and two …