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The Effects Of Continuous Insulin Pump Therapy On Glycemic Control In Pregnant Type 1 Diabetics, Kimberly Kelsey 2017 Dominican University of California

The Effects Of Continuous Insulin Pump Therapy On Glycemic Control In Pregnant Type 1 Diabetics, Kimberly Kelsey

Senior Theses and Capstone Projects

Type 1 Diabetics have various ways of managing their diabetes that have been studied for their effectiveness. Pregnancy in the Type 1 Diabetic has been understudied. It is known that Diabetics in general have poorer pregnancy outcomes because of poor glycemic control. This coupled with the fact that the body needs 3-4 times more insulin as pregnancy comes to an end makes managing blood glucose levels challenging for diabetics (Lowdermilk, 2016, p. 689). For the Type 1 Diabetic, there are two main ways to control diabetes: multiple daily injections using at least two type of insulin and continuous subcutaneous insulin ...


The Effect Of Photoactivated Tmp On Burkholderia Cepacia Biofilms, Reyna G. Osorio, Chandra N. Swiech, Tracy L. Collins 2017 Cedarville University

The Effect Of Photoactivated Tmp On Burkholderia Cepacia Biofilms, Reyna G. Osorio, Chandra N. Swiech, Tracy L. Collins

Tracy Collins, Ph.D.

Burkholderia cepacia is an opportunistic pathogen that causes infections in immunocompromised individuals such as cystic fibrosis patients. B. cepacia infections are typically characterized by the formation of complex communities of cells known as biofilms. Because B. cepacia biofilms are difficult to eradicate using antibiotics, it is important to pursue alternative treatment methods. Photodynamic therapy (PDT) is a type of therapy that uses light, a photosensitizer, and oxygen to elicit cell death through the production of reactive oxygen species. PDT has been shown in previous studies to be successful in killing both Pseudomonas aeruginosa and Staphylococcus aureus. In this study, we ...


Reframing Outcome Measures For Thrombolytics In Acute Ischemic Stroke, Brandon Baker, Charles W. Kropf, Nicholus Yee, James Peter Meza 2017 DMC-Sinai Grace Hospital Emergency Medicine Residency

Reframing Outcome Measures For Thrombolytics In Acute Ischemic Stroke, Brandon Baker, Charles W. Kropf, Nicholus Yee, James Peter Meza

Clinical Research in Practice: The Journal of Team Hippocrates

An informed consent conversation examining relevant research and discussing the potential benefits and harms of thrombolytic therapy in acute stroke.


Anticoagulation Increases Survival In Patients With Idiopathic Pulmonary Arterial Hypertension, Marvin L. Kajy 2017 Department of Internal Medicine, Detroit Receiving Hospital/Detroit Medical Center

Anticoagulation Increases Survival In Patients With Idiopathic Pulmonary Arterial Hypertension, Marvin L. Kajy

Clinical Research in Practice: The Journal of Team Hippocrates

A critical appraisal and clinical application of Olsson KM, Delcroix M, Ghofrani HA, et al. Anticoagulation and survival in pulmonary arterial hypertension: Results from the Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA). Circulation, 2014;129(1):57-65. doi: 10.1161/CIRCULATIONAHA.113.004526


Evolution Of The Alpha-1 Antitrypsin Muscle Gene Therapy: Translation From Clinical Trial To Benchtop And Back Again, Alisha M. Gruntman, Gwladys Gernoux, Gensheng Wang, Janet Benson, Jeff Chulay, Dave Knop, Christian Mueller, Terence R. Flotte 2017 University of Massachusetts Medical School

Evolution Of The Alpha-1 Antitrypsin Muscle Gene Therapy: Translation From Clinical Trial To Benchtop And Back Again, Alisha M. Gruntman, Gwladys Gernoux, Gensheng Wang, Janet Benson, Jeff Chulay, Dave Knop, Christian Mueller, Terence R. Flotte

Christian Mueller

Alpha-one antitrypsin (AAT) deficiency is a genetic disease affecting the lungs due to inadequate anti-protease activity in the pulmonary interstitium. On-going human trials use intra-muscular delivery of adeno-associated virus (rAAV1), allowing expressing myofibers to secrete normal (M)AAT protein. In the Phase IIa trial, patients in the highest dose cohort (6x1012vg/kg) were given 100 intra-muscular (IM) injections of undiluted vector, with serum AAT levels still substantially below target levels. Previous work has shown that delivering rAAV vector to the musculature via limb perfusion leads to widespread gene expression in myofibers. We hypothesize that widespread delivery would result in an ...


Alzheimer’S Disease: Dawn Of A New Era?, Farideh Amirrad, Emira Bousoik, Kiumars Shamloo, Hassan Al-Shiyab, Viet-Hong Nguyen, Hamidreza Montazeri Aliabadi 2017 Chapman University

Alzheimer’S Disease: Dawn Of A New Era?, Farideh Amirrad, Emira Bousoik, Kiumars Shamloo, Hassan Al-Shiyab, Viet-Hong Nguyen, Hamidreza Montazeri Aliabadi

Pharmacy Faculty Articles and Research

Alzheimer’s disease (AD) is an irreversible neurodegenerative disease characterized by a progressive decline in cognition and memory, leading to significant impairment in daily activities and ultimately death. It is the most common cause of dementia, the prevalence of which increases with age; however, age is not the only predisposing factor. The pathology of this cognitive impairing disease is still not completely understood, which has limited the development of valid therapeutic options. Recent years have witnessed a wide range of novel approaches to combat this disease, so that they greatly increased our understanding of the disease and of the unique ...


Oncology Section Edge Task Force On Urogenital Cancer Outcomes: Clinical Measures Of Lymphedema—A Systematic Review, Joy C. Cohn, Hannah Geyer, Jeannette Lee, Mary Insana Fisher 2017 University of Pennsylvania Health System

Oncology Section Edge Task Force On Urogenital Cancer Outcomes: Clinical Measures Of Lymphedema—A Systematic Review, Joy C. Cohn, Hannah Geyer, Jeannette Lee, Mary Insana Fisher

Physical Therapy Faculty Publications

Background: Valid and reliable tools to assess lymphedema are necessary to accurately evaluate status and to objectively document and measure the results of interventions. Understanding the advantages and disadvantages of each measure can inform the clinician's choice of the appropriate tool to be used in the clinic or research setting.

Purpose: To identify reliable and valid measurement techniques that are sensitive to change for assessing edema volume or soft tissue change in the lower extremities or genital region of patients with lymphedema.

Methods: A systematic review of the literature was conducted to assess the published psychometric properties and clinical ...


Clinical Application Of A Cancer Genomic Profiling Assay To Guide Precision Medicine Decisions, Cheryl Eifert, Angeliki Pantazi, Ruobai Sun, Jia Xu, Pablo Cingolani, Joerg Heyer, Meaghan Russell, Maria Lvova, Jennifer Ring, Julie Y. Tse, Stephen Lyle, Alexei Protopopov 2017 KEW, Inc.

Clinical Application Of A Cancer Genomic Profiling Assay To Guide Precision Medicine Decisions, Cheryl Eifert, Angeliki Pantazi, Ruobai Sun, Jia Xu, Pablo Cingolani, Joerg Heyer, Meaghan Russell, Maria Lvova, Jennifer Ring, Julie Y. Tse, Stephen Lyle, Alexei Protopopov

Open Access Articles

AIM: Develop and apply a comprehensive and accurate next-generation sequencing based assay to help clinicians to match oncology patients to therapies.

MATERIALS and METHODS: The performance of the CANCERPLEX(R) assay was assessed using DNA from well-characterized routine clinical formalin-fixed paraffin-embedded (FFPE) specimens and cell lines.

RESULTS: The maximum sensitivity of the assay is 99.5% and its accuracy is virtually 100% for detecting somatic alterations with an allele fraction of as low as 10%. Clinically actionable variants were identified in 93% of patients (930 of 1000) who underwent testing.

CONCLUSION: The test's capacity to determine all of the ...


Safe And Efficient Silencing With A Pol Ii, But Not A Pol Lii, Promoter Expressing An Artificial Mirna Targeting Human Huntingtin, Edith L. Pfister, Kathryn O. Chase, Huaming Sun, Lori A. Kennington, Faith Conroy, Emily S. Johnson, Rachael Miller, Florie Borel, Neil Aronin, Christian Mueller 2017 University of Massachusetts Medical School

Safe And Efficient Silencing With A Pol Ii, But Not A Pol Lii, Promoter Expressing An Artificial Mirna Targeting Human Huntingtin, Edith L. Pfister, Kathryn O. Chase, Huaming Sun, Lori A. Kennington, Faith Conroy, Emily S. Johnson, Rachael Miller, Florie Borel, Neil Aronin, Christian Mueller

Pediatric Publications and Presentations

Huntington's disease is a devastating, incurable neurodegenerative disease affecting up to 12 per 100,000 patients worldwide. The disease is caused by a mutation in the Huntingtin (Htt) gene. There is interest in reducing mutant Huntingtin by targeting it at the mRNA level, but the maximum tolerable dose and long-term effects of such a treatment are unknown. Using a self-complementary AAV9 vector, we delivered a mir-155-based artificial miRNA under the control of the chicken beta-actin or human U6 promoter. In mouse brain, the artificial miRNA reduced the human huntingtin mRNA by 50%. The U6, but not the CbetaA promoter ...


5 Year Expression And Neutrophil Defect Repair After Gene Therapy In Alpha-1 Antitrypsin Deficiency, Christian Mueller, Gwladys Gernoux, Alisha M. Gruntman, Florie Borel, Emer P. Reeves, Roberto Calcedo, Farshid N. Rouhani, Anthony Yachnis, Margaret Humphries, Martha Campbell-Thompson, Louis M. Messina, Jeffrey D. Chulay, Bruce Trapnell, James M. Wilson, Noel G. McElvaney, Terence R. Flotte 2017 University of Massachusetts Medical School

5 Year Expression And Neutrophil Defect Repair After Gene Therapy In Alpha-1 Antitrypsin Deficiency, Christian Mueller, Gwladys Gernoux, Alisha M. Gruntman, Florie Borel, Emer P. Reeves, Roberto Calcedo, Farshid N. Rouhani, Anthony Yachnis, Margaret Humphries, Martha Campbell-Thompson, Louis M. Messina, Jeffrey D. Chulay, Bruce Trapnell, James M. Wilson, Noel G. Mcelvaney, Terence R. Flotte

Christian Mueller

Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due principally to the unopposed effects of neutrophil elastase. We previously reported achieving plasma wild-type alpha-1 antitrypsin concentrations at 2.5%-3.8% of the purported therapeutic level at 1 year after a single intramuscular administration of recombinant adeno-associated virus serotype 1 alpha-1 antitrypsin vector in alpha-1 antitrypsin deficient patients. We analyzed blood and muscle for alpha-1 antitrypsin expression and immune cell response. We also assayed previously reported markers of neutrophil function known to be altered in alpha-1 antitrypsin deficient patients. Here, we report sustained expression at 2.0%-2 ...


5 Year Expression And Neutrophil Defect Repair After Gene Therapy In Alpha-1 Antitrypsin Deficiency, Christian Mueller, Gwladys Gernoux, Alisha M. Gruntman, Florie Borel, Emer P. Reeves, Roberto Calcedo, Farshid N. Rouhani, Anthony Yachnis, Margaret Humphries, Martha Campbell-Thompson, Louis M. Messina, Jeffrey D. Chulay, Bruce Trapnell, James M. Wilson, Noel G. McElvaney, Terence R. Flotte 2017 University of Massachusetts Medical School

5 Year Expression And Neutrophil Defect Repair After Gene Therapy In Alpha-1 Antitrypsin Deficiency, Christian Mueller, Gwladys Gernoux, Alisha M. Gruntman, Florie Borel, Emer P. Reeves, Roberto Calcedo, Farshid N. Rouhani, Anthony Yachnis, Margaret Humphries, Martha Campbell-Thompson, Louis M. Messina, Jeffrey D. Chulay, Bruce Trapnell, James M. Wilson, Noel G. Mcelvaney, Terence R. Flotte

University of Massachusetts Medical School Faculty Publications

Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due principally to the unopposed effects of neutrophil elastase. We previously reported achieving plasma wild-type alpha-1 antitrypsin concentrations at 2.5%-3.8% of the purported therapeutic level at 1 year after a single intramuscular administration of recombinant adeno-associated virus serotype 1 alpha-1 antitrypsin vector in alpha-1 antitrypsin deficient patients. We analyzed blood and muscle for alpha-1 antitrypsin expression and immune cell response. We also assayed previously reported markers of neutrophil function known to be altered in alpha-1 antitrypsin deficient patients. Here, we report sustained expression at 2.0%-2 ...


Diverse Stimuli Engage Different Neutrophil Extracellular Trap Pathways, Elaine F. Kenny, Alf Herzig, Renate Krüger, Aaron Muth, Santanu Mondal, Paul R. Thompson, Volker Brinkmann, Horst Von Bernuth, Arturo Zychlinsky 2017 Max Planck Institute for Infection Biology

Diverse Stimuli Engage Different Neutrophil Extracellular Trap Pathways, Elaine F. Kenny, Alf Herzig, Renate Krüger, Aaron Muth, Santanu Mondal, Paul R. Thompson, Volker Brinkmann, Horst Von Bernuth, Arturo Zychlinsky

Thompson Lab Publications

Neutrophils release neutrophil extracellular traps (NETs) which ensnare pathogens and have pathogenic functions in diverse diseases. We examined the NETosis pathways induced by five stimuli; PMA, the calcium ionophore A23187, nigericin, Candida albicans and Group B Streptococcus. We studied NET production in neutrophils from healthy donors with inhibitors of molecules crucial to PMA induced NETs including protein kinase C, calcium, reactive oxygen species, the enzymes myeloperoxidase (MPO) and neutrophil elastase. Additionally, neutrophils from chronic granulomatous disease patients, carrying mutations in the NADPH oxidase complex or a MPO-deficient patient were examined. We show that PMA, C. albicans and GBS use a ...


Identification Of A Nucleoside Analog Active Against Adenosine Kinase-Expressing Plasma Cell Malignancies, Utthara Nayar, Jouliana Sadek, Jonathan Reichel, Denise Hernandez-Hopkins, Gunkut Akar, Peter J. Barelli, Michelle A. Sahai, Hufeng Zhou, Jennifer Totonchy, David Jayabalan, Ruben Niesvizky, Ilaria Guasparri, Duane Hassane, Yifang Liu, Shizuko Sei, Robert H. Shoemaker, J. David Warren, Olivier Elemento, Kenneth M. Kaye, Ethel Cesarman 2017 Weill Cornell Medical College

Identification Of A Nucleoside Analog Active Against Adenosine Kinase-Expressing Plasma Cell Malignancies, Utthara Nayar, Jouliana Sadek, Jonathan Reichel, Denise Hernandez-Hopkins, Gunkut Akar, Peter J. Barelli, Michelle A. Sahai, Hufeng Zhou, Jennifer Totonchy, David Jayabalan, Ruben Niesvizky, Ilaria Guasparri, Duane Hassane, Yifang Liu, Shizuko Sei, Robert H. Shoemaker, J. David Warren, Olivier Elemento, Kenneth M. Kaye, Ethel Cesarman

Pharmacy Faculty Articles and Research

Primary effusion lymphoma (PEL) is a largely incurable malignancy of B cell origin with plasmacytic differentiation. Here, we report the identification of a highly effective inhibitor of PEL. This compound, 6-ethylthioinosine (6-ETI), is a nucleoside analog with toxicity to PEL in vitro and in vivo, but not to other lymphoma cell lines tested. We developed and performed resistome analysis, an unbiased approach based on RNA sequencing of resistant subclones, to discover the molecular mechanisms of sensitivity. We found different adenosine kinase–inactivating (ADK-inactivating) alterations in all resistant clones and determined that ADK is required to phosphorylate and activate 6-ETI. Further ...


Rod Outer Segment Development Influences Aav-Mediated Photoreceptor Transduction After Subretinal Injection, Lolita Petit, Shan Ma, Shun-Yun Cheng, Guangping Gao, Claudio Punzo 2017 University of Massachusetts Medical School

Rod Outer Segment Development Influences Aav-Mediated Photoreceptor Transduction After Subretinal Injection, Lolita Petit, Shan Ma, Shun-Yun Cheng, Guangping Gao, Claudio Punzo

UMass Metabolic Network Publications

Vectors based on the adeno-associated virus (AAV) are currently the preferred tools for delivering genes to photoreceptors (PR) in small and large animals. AAVs have been applied successfully in various models of PR dystrophies. However, unknown barriers still limit AAV's efficient application in several forms of severe PR degenerations due to insufficient transgene expression and/or treated cells at the time of injection. Optimizations of PR gene therapy strategies will likely benefit from the identification of the cellular factors that influence PR transduction. Interestingly, recent studies have shown that the AAV transduction profile of PRs differs significantly between neonatal ...


Role Of Peptidylarginine Deiminase 2 (Pad2) In Mammary Carcinoma Cell Migration, Sachi Horibata, Katherine E. Rogers, David Sadegh, Lynne J. Anguish, John L. McElwee, Pragya Shah, Paul R. Thompson, Scott A. Coonrod 2017 Cornell University

Role Of Peptidylarginine Deiminase 2 (Pad2) In Mammary Carcinoma Cell Migration, Sachi Horibata, Katherine E. Rogers, David Sadegh, Lynne J. Anguish, John L. Mcelwee, Pragya Shah, Paul R. Thompson, Scott A. Coonrod

Thompson Lab Publications

BACKGROUND: Penetration of the mammary gland basement membrane by cancer cells is a crucial first step in tumor invasion. Using a mouse model of ductal carcinoma in situ, we previously found that inhibition of peptidylarginine deiminase 2 (PAD2, aka PADI2) activity appears to maintain basement membrane integrity in xenograft tumors. The goal of this investigation was to gain insight into the mechanisms by which PAD2 mediates this process.

METHODS: For our study, we modulated PAD2 activity in mammary ductal carcinoma cells by lentiviral shRNA-mediated depletion, lentiviral-mediated PAD2 overexpression, or PAD inhibition and explored the effects of these treatments on changes ...


Recombinant Aav Serotype And Capsid Mutant Comparison For Pulmonary Gene Transfer Of Alpha-1-Antitrypsin Using Invasive And Noninvasive Delivery, Rejean Liqun Wang, Thomas J. McLaughlin, Travis Cossette, Qiushi Tang, Kevin Foust, Martha Campbell-Thompson, Ashley Martino, Pedro Cruz, Scott Loiler, Christian Mueller, Terence R. Flotte 2017 University of Florida

Recombinant Aav Serotype And Capsid Mutant Comparison For Pulmonary Gene Transfer Of Alpha-1-Antitrypsin Using Invasive And Noninvasive Delivery, Rejean Liqun Wang, Thomas J. Mclaughlin, Travis Cossette, Qiushi Tang, Kevin Foust, Martha Campbell-Thompson, Ashley Martino, Pedro Cruz, Scott Loiler, Christian Mueller, Terence R. Flotte

Christian Mueller

Recombinant adeno-associated viral (rAAV) vectors have been widely used in pulmonary gene therapy research. In this study, we evaluated the transduction and expression efficiencies of several AAV serotypes and AAV2 capsid mutants with specific pulmonary targeting ligands in the mouse lung. The noninvasive intranasal delivery was compared with the traditional intratracheal lung delivery. The rAAV8 was the most efficient serotype at expressing alpha-1-antitrypsin (AAT) in the lung among all the tested serotypes and mutants. A dose of 1 x 1010 vg of rAAV8-CB-AAT transduced a high percentage of cells in the lung when delivered intratrachealy. The serum and the broncho-alveolar ...


Modeling Hiv Dynamics Following 3bnc117 Antibody Infusion, Samantha Erwin 2017 Virginia Polytechnic Institute and State University

Modeling Hiv Dynamics Following 3bnc117 Antibody Infusion, Samantha Erwin

Biology and Medicine Through Mathematics Conference

No abstract provided.


Evolution Of The Alpha-1 Antitrypsin Muscle Gene Therapy: Translation From Clinical Trial To Benchtop And Back Again, Alisha M. Gruntman, Gwladys Gernoux, Gensheng Wang, Janet Benson, Jeff Chulay, Dave Knop, Christian Mueller, Terence R. Flotte 2017 University of Massachusetts Medical School

Evolution Of The Alpha-1 Antitrypsin Muscle Gene Therapy: Translation From Clinical Trial To Benchtop And Back Again, Alisha M. Gruntman, Gwladys Gernoux, Gensheng Wang, Janet Benson, Jeff Chulay, Dave Knop, Christian Mueller, Terence R. Flotte

UMass Center for Clinical and Translational Science Research Retreat

Alpha-one antitrypsin (AAT) deficiency is a genetic disease affecting the lungs due to inadequate anti-protease activity in the pulmonary interstitium. On-going human trials use intra-muscular delivery of adeno-associated virus (rAAV1), allowing expressing myofibers to secrete normal (M)AAT protein. In the Phase IIa trial, patients in the highest dose cohort (6x1012vg/kg) were given 100 intra-muscular (IM) injections of undiluted vector, with serum AAT levels still substantially below target levels. Previous work has shown that delivering rAAV vector to the musculature via limb perfusion leads to widespread gene expression in myofibers. We hypothesize that widespread delivery would result ...


Combination Photodynamic Therapy And Chemotherapy For Temozolomide-Resistant Glioblastoma, Janel Kydd, Rahul Jadia, Prakash Rai 2017 University of Massachusetts Lowell

Combination Photodynamic Therapy And Chemotherapy For Temozolomide-Resistant Glioblastoma, Janel Kydd, Rahul Jadia, Prakash Rai

UMass Center for Clinical and Translational Science Research Retreat

Polymer based nanoparticles (NPs) are useful vehicles for drug therapy in treating glioblastoma because of their ideal characteristics such as small size, to cross the blood-brain barrier, and bind to overexpressed transferrin receptors via peptide conjugation and surface modification of NPs. The use of a photosensitizer drug such as verteporfin, or BPD, in combination with a repurposed drug, Cediranib (CED), prepared as a nanoparticle therapy will provide the medical field with new research on the possible ways to treat glioblastoma. BPD-CED-loaded NPs have the potential to induce cytotoxicity in glioblastoma cells by 1) remotely triggering BPD through photodynamic therapy by ...


Matrix Assisted Laser Desorption Ionization (Maldi) Mass Spectrometry: An Imaging Modality To Monitor The Effects Of Gene Therapy In A Murine Model Of Gm1 Gangliosidosis, Khaja Muneeruddin, Bindesh Shrestha, Hernando Olivos, Sophia H. Todeasa, Eleonora D'Ambrosio, Miguel Sena-Esteves, Scott A. Shaffer 2017 University of Massachusetts Medical School

Matrix Assisted Laser Desorption Ionization (Maldi) Mass Spectrometry: An Imaging Modality To Monitor The Effects Of Gene Therapy In A Murine Model Of Gm1 Gangliosidosis, Khaja Muneeruddin, Bindesh Shrestha, Hernando Olivos, Sophia H. Todeasa, Eleonora D'Ambrosio, Miguel Sena-Esteves, Scott A. Shaffer

UMass Center for Clinical and Translational Science Research Retreat

GM1 gangliosidosis is an autosomal recessive lysosomal storage disorder caused by an enzyme deficiency of β-galactosidase (β-gal) leading to toxic accumulation of GM1 ganglioside in the central nervous system (CNS) and progressive neurodegeneration. Adeno-associated virus (AAV) mediated gene delivery of lysosomal enzymes to the CNS has shown great potential for the treatment of lysosomal storage diseases with neurological involvement. In this work we use MALDI mass spectrometry imaging (MSI) to assess the spatial distribution of gangliosides, ganglioside metabolites and related lipids in a GM1 gangliosidosis mouse brain model following adeno-associated virus (AAV) gene therapy.

Deficiency of β-galactosidase enzyme in a ...


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