Virtual Collaboration: Exploring New Frontiers

Librarians looking for professional development opportunities, especially in states like North Dakota where many serve small populations in rural areas, will benefit from thinking creatively about organizing a conference. Two sections of the North Dakota Library Association (NDLA) used Blackboard Collaborate Ultra as a platform to produce a fully online conference. This virtual “unconference” was interactive and robust, with speakers from across the state, interactive whiteboards, poster rooms, meeting space for special interest groups, and more. In this workshop, we will share what we have learned about Collaborate Ultra, including its strengths and weaknesses. We hope to brainstorm and discuss other potential applications, including classrooms, professional development, online meetings, group work, and supporting distance students and colleagues. Please bring your device and join us in a working session where you will experience Collaborate Ultra first-hand and discover how this tool can facilitate learning and collaboration and impact the work you do.https://commons.und.edu/cfl-lpp/1005/thumbnail.jp

A New Frontier: Lessons Learned from NDLA\u27s First Online Un-Conference

NDLA’s Health Science Information and Academic & Special Libraries Sections held its inaugural fully online “un-conference” using Blackboard Collaborate Ultra. A fully online conference can be a robust, low-cost alternative to a traditional conference that could be an advantage to librarians looking for professional development opportunities in this rural state. In this session, the planners will discuss creating this experience and lessons learned.https://commons.und.edu/cfl-lpp/1002/thumbnail.jp

Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients

Pathogenesis and treatment of chronic pulmonary disease

Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.

BACKGROUND & AIMS:Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder causing pulmonary and liver disease. The PiZ mutation in AAT (SERPINA1) results in mis-folded AAT protein (Z-AAT) accumulating in hepatocytes, leading to fibrosis and cirrhosis. RNAi-based therapeutics silencing production of hepatic Z-AAT might benefit patients with AATD-associated liver disease. This study evaluated an RNAi therapeutic to silence production of AAT. METHODS:Part A of this double-blind first-in-human study randomized 54 healthy volunteers (HVs) into single dose cohorts (two placebo: four active), receiving escalating doses of the investigational agent ARC-AAT from 0.38 to 8.0 mg/kg or placebo. Part B randomized 11 patients with PiZZ (homozygous for Z-AAT) genotype AATD, who received up to 4.0 mg/kg of ARC-AAT or placebo. Patients with baseline FibroScan® >11 kPa or forced expiratory volume in one second (FEV1) <60% were excluded. Assessments included safety, pharmacokinetics, and change in serum AAT concentrations. RESULTS:A total of 36 HVs received ARC-AAT and 18 received placebo (part A). Seven PiZZ individuals received ARC-AAT and four received placebo (part B). A dose response in serum AAT reduction was observed at doses ≥4 mg/kg with similar relative reductions in PiZZ patients and HVs at 4 mg/kg and a maximum reduction of 76.1% (HVs) vs. 78.8% (PiZZ) at this dose. The time it took for serum AAT to return to baseline was similar for HV and PiZZ. There were no notable differences between HV and PiZZ safety parameters. The study was terminated early because of toxicity findings related to the delivery vehicle (ARC-EX1) seen in a non-human primate study. CONCLUSION:PiZZ patients and HVs responded similarly to ARC-AAT. Deep and durable knockdown of hepatic AAT production based on observed reduction in serum AAT concentrations was demonstrated. LAY SUMMARY:Accumulation of abnormal proteins in the livers of patients with alpha-1 antitrypsin deficiency may lead to decreased liver function and potentially liver failure. Therapeutics targeting the production of these abnormal proteins may be used to prevent or treat liver disease in patients with alpha-1 antitrypsin deficiency. CLINICAL TRIAL REGISTRATION NUMBER:NCT02363946