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Voyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies

Fri, 05/20/2016 - 2:15pm

As part of the mini-symposium entitled "Shark Tank-UMass Spin-out Life Sciences Start-ups," Dr. Gao discusses an example of UMass life sciences technology that has been spun out into a start-up company -- Voyager Therapeutics from UMass Worcester.

Advancing Translational Research at the UMass Amherst Center for Personalized Health Monitoring

Fri, 05/20/2016 - 2:15pm

This is the moderator's introductory presentation for the mini-symposium entitled "Advancing Translational Research at the UMass Amherst Center for Personalized Health Monitoring," in which she discusses the mission of the Center, which is to advance life sciences research to improve human health.

InVitroMetrix QCM-Based Cell Biosensor: Research tool to accelerate pharmaceutical drug discovery success

Fri, 05/20/2016 - 2:15pm

As part of the mini-symposium entitled "Shark Tank-UMass Spin-out Life Sciences Start-ups," Dr. Dewilde discusses an example of UMass life sciences technology that has been spun out into a start-up company -- Cell QCM from UMass Lowell.

Increasing Physical Activity Amounts and Intensity in Older Adults Using Low Cost Wearable Devices - “Cadence Training”

Fri, 05/20/2016 - 1:00pm

As part of the mini-symposium entitled "Biomechanical Gait Analysis for Improving Clinical Outcomes: Applications for Orthopedics, Geriatrics and Community Based Research," this presentation discusses wearable devices.

Electronic Communication Systems: Energizing the Patient with Diabetes to Engage in Their Own Health Care

Fri, 05/20/2016 - 1:00pm

As part of the mini-symposium entitled "Interdiscipllinary Mobile Health and Sensing Research," Dr. Thompson discusses his work in mobile diabetes monitoring.

Using Mobile-Based Technology to Screen for Atrial Fibrillation in India

Fri, 05/20/2016 - 1:00pm

As part of the mini-symposium entitled "Interdiscipllinary Mobile Health and Sensing Research," this presentation discusses use of a novel smartphone app for cardiovascular screening in rural India.

Patient Reported Outcomes in Arthritis, TJR, and Physical Activity Research

Fri, 05/20/2016 - 1:00pm

As part of the mini-symposium entitled "Biomechanical Gait Analysis for Improving Clinical Outcomes: Applications for Orthopedics, Geriatrics and Community Based Research," this presentation discusses the importance of patient reported outcomes (PRO) in clinical research and the PRO/physical activity translational research in osteoarthritis and and total joint replacement at UMass Medical School.

Innovations for Vulnerable Populations in Massachusetts

Fri, 05/20/2016 - 1:00pm

This is the moderator's introductory presentation for the mini-symposium entitled "Innovations for Vulnerable Populations in Massachusetts," in which she places the session presentations in the context of an ongoing collaboration between Commonwealth Medicine, a division of the University of Massachusetts Medical School, and MassHealth.

Examining Movement Function in Patients with Knee Osteoarthritis

Fri, 05/20/2016 - 1:00pm

As part of the mini-symposium entitled "Biomechanical Gait Analysis for Improving Clinical Outcomes: Applications for Orthopedics, Geriatrics and Community Based Research," this presentation explores research on gait analysis and pain for patients with knee osteoarthritis.

Medical, Social, and Other Determinants of Health Care Costs in MassHealth

Fri, 05/20/2016 - 1:00pm

As part of the mini-symposium entitled "Innovations for Vulnerable Populations in Massachusetts," this presentation explores research into expanding the State’s existing risk models to include social determinants of health variables. Potential variables for inclusion in payment models (such as unstable housing, defined as having three or more addresses during a calendar year) have been identified. These models are being developed in support of alternative payment mechanisms for integrated delivery systems.

Keynote Address: The Future of Cardiovascular Epidemiology: Current Trends?

Fri, 05/20/2016 - 12:00pm

This is the Research Retreat's Keynote presentation by Vasan S. Ramachandran, MD, who is Principal Investigator and Co-Director, Echocardiography/Vascular Laboratory, Framingham Heart Study. Dr. Ramachandran is also Chief, Section of Preventive Medicine and Epidemiology and Professor of Medicine, Boston University School of Medicine. Dr. Ramachandran discusses the future of cardiovascular epidemiology, including the roles of: cHealth (community), sHealth (social), mHealth (mobile), eHealth (electronic), and gHealth (genomic).

UMass Center for Clinical and Translational Science: Past, Present and Future

Fri, 05/20/2016 - 11:30am

Katherine Luzuriaga, MD, is PI and Director, UMass Center for Clinical and Translational Science and Vice Provost, Clinical and Translational Research at UMass Medical School. Dr. Luzuriaga is also the UMass Memorial Health Care Chair in Biomedical Research and Professor, Program in Molecular Medicine, Pediatrics and Medicine. In her presentation, she reviews the history, goals, programs and achievements of the UMass Center for Clinical and Translational Science.

The Challenge of Maintaining our Physician-Scientist Workforce

Fri, 05/20/2016 - 11:20am

Dr. Flotte, who is Dean, Provost and Executive Deputy Chancellor at the University of Massachusetts Medical School, discusses the value of physician-scientists to the mission of academic medicine, and the challenges and rewards. Specific purposeful mechanisms must be developed to ensure the ongoing viability of the physician-scientist role.

Mini Symposia Program: 2016 UMass Center for Clinical and Translational Science Research Retreat

Fri, 05/20/2016 - 10:30am

Mini Symposia Program for the 6th annual UMass Center for Clinical and Translational Science Research Retreat, held Friday, May 20, 2016 at the University of Massachusetts Medical School, Worcester, MA.

Agenda: 2016 UMass Center for Clinical and Translational Science Research Retreat

Fri, 05/20/2016 - 10:30am

Agenda for the 6th annual UMass Center for Clinical and Translational Science Research Retreat, held Friday, May 20, 2016 at the University of Massachusetts Medical School, Worcester, MA.

Developing an Adeno-Associated Viral Vector (AAV) Toolbox for CNS Gene Therapy: A Dissertation

Thu, 05/19/2016 - 4:08pm

Neurological disorders – disorders of the brain, spine and associated nerves – are a leading contributor to global disease burden with a sizable economic cost. Adeno-associated viral (AAV) vectors have emerged as an effective platform for CNS gene therapy and have shown early promise in clinical trials. These trials involve direct infusion into brain parenchyma, an approach that may be suboptimal for treatment of neurodegenerative disorders, which often involve more than a single structure in the CNS. However, overall neuronal transduction efficiency of vectors derived from naturally occurring AAV capsids after systemic administration is relatively low. We have developed novel capsids AAV-AS and AAV-B1 that lead to widespread gene delivery throughout the brain and spinal cord, particularly to neuronal populations. Both transduce the adult mouse brain >10-fold more efficiently than the clinical gold standard AAV9 upon intravascular infusion, with gene transfer to multiple neuronal sub-populations. These vectors are also capable of neuronal transduction in a normal cat. We have demonstrated the efficacy of AAV-AS in the context of Huntington's disease by knocking down huntingtin mRNA 33-50% after a single intravenous injection, which is better than what can be achieved by AAV9 at the particular dose. AAVB1 additionally transduces muscle, beta cells, pulmonary alveoli and retinal vasculature at high efficiency, and has reduced sensitivity to neutralizing antibodies in human sera. Generation of this vector toolbox represents a major step towards gaining genetic access to the entire CNS, and provides a platform to develop new gene therapies for neurodegenerative disorders.

Systematic Dissection of Roles for Chromatin Regulators in Dynamics of Transcriptional Response to Stress in Yeast: A Dissertation

Thu, 05/19/2016 - 4:08pm

The following work demonstrates that chromatin regulators play far more pronounced roles in dynamic gene expression than they do in steady-state. Histone modifications have been associated with transcription activity. However, previous analyses of gene expression in mutants affecting histone modifications show limited alteration. I systematically dissected the effects of 83 histone mutants and 119 gene deletion mutants on gene induction/repression in response to diamide stress in yeast. Importantly, I observed far more changes in gene induction/repression than changes in steady-state gene expression. The extensive dynamic gene expression profile of histone mutants and gene deletion mutants also allowed me to identify specific interactions between histone modifications and chromatin modifiers. Furthermore, by combining these functional results with genome-wide mapping of several histone modifications in the same time course, I was able to investigate the correspondence between histone modification occurrence and function. One such observation was the role of Set1-dependent H3K4 methylation in the repression of ribosomal protein genes (RPGs) during multiple stresses. I found that proper repression of RPGs in stress required the presence, but not the specific sequence, of an intron, an element which is almost unique to this gene class in Saccharomyces cerevisiae. This repression may be related to Set1’s role in antisense RNA-mediated gene silencing. Finally, I found a potential role for Set1 in producing or maintaining uncapped mRNAs in cells through a mechanism that does not involved nuclear exoribonucleases. Thus, deletion of Set1 in xrn1Δ suppresses the accumulation of uncapped transcripts observed in xrn1Δ. These findings reveal that Set1, along with other chromatin regulators, plays important roles in dynamic gene expression through diverse mechanisms and thus provides a coherent means of responding to environmental cues.

Distracted Practice and Patient Safety: The Healthcare Team Experience: A Dissertation

Thu, 05/19/2016 - 3:20pm

Purpose: The purpose of this study was to explore the experiences of distracted practice across the healthcare team.

Definition: Distracted practice is the diversion of a portion of available cognitive resources that may be needed to effectively perform/carry out the current activity.

Background: Distracted practice is the result of individuals interacting with the healthcare team, the environment and technology in the performance of their jobs. The resultant behaviors can lead to error and affect patient safety.

Methods: A qualitative descriptive (QD) approach was used that integrated observations with semi-structured interviews. The conceptual framework was based on the distracted driving model and a completed concept analysis.

Results: There were 22 observation sessions and 32 interviews (12 RNs, 11 MDs, and 9 Pharmacists) completed between December, 2014 and July 2015. Results suggested that distracted practice is based on the main theme of cognitive resources which varies by the subthemes of individual differences; environmental disruptions; team awareness; and “rush mode”/time pressure.

Conclusions and Implications: Distracted practice is an individual human experience that occurs when there are not enough cognitive resources available to effectively complete the task at hand. In that moment an individual shifts from thinking critically, being able to complete their current task without error, to not thinking critically and working in an automatic mode. This is when errors occur. Additional research is needed to evaluate intervention strategies to reduce and prevent distracted practice.

Techniques in distal access of wide-necked giant intracranial aneurysms during treatment with flow diversion

Tue, 05/17/2016 - 12:38pm

BACKGROUND: Accessing the normal distal vessel in treatment of wide-necked giant intracranial aneurysms with flow diversion can be difficult.

CASE DESCRIPTION: Through illustrative cases, the authors present several useful techniques in distal access of wide-necked giant aneurysms during flow diversion treatment. Obtaining an optimal projection that separates the outflow limb from the aneurysm is most critical. Each of the three techniques described enabled the distal access to giant intracranial aneurysms during treatment with flow diversion.

CONCLUSION: The looped-around technique, balloon-assisted technique, and retrograde access are valuable strategies in crossing the aneurysm if direct distal access cannot be obtained.

Biosimilars in rheumatology: what the clinician should know

Tue, 05/17/2016 - 12:37pm

Biosimilars are now a reality in rheumatology. Although analytical and non-clinical procedures to establish similarity have evolved significantly, clinical trials demonstrating equivalent efficacy and safety are absolutely required for all biosimilars. The design of such trials, including equivalence and non-inferiority statistical approaches, are discussed. Clinical evidence on biosimilars that have been approved recently or are presently being developed for use in rheumatology is also reviewed and contrasted with that available for biomimics (or intended copies), which are non-innovator biologics that are marketed in several countries but have not undergone review according to a regulatory pathway for biosimilars.