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Bioengineering & Translational Medicine

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https://www.readbyqxmd.com/read/30065971/how-will-the-field-of-gene-therapy-survive-its-success
#1
REVIEW
William F Kaemmerer
In August 2017, for the first time, a gene therapy was approved for market release in the United States. That approval was followed by two others before the end of the year. This article cites primary literature, review articles concerning particular biotechnologies, and press releases by the FDA and others in order to provide an overview of the current status of the field of gene therapy with respect to its translation into practice. Technical hurdles that have been overcome in the past decades are summarized, as are hurdles that need to be the subject of continued research...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065970/multivariate-techniques-enable-a-biochemical-classification-of-children-with-autism-spectrum-disorder-versus-typically-developing-peers-a-comparison-and-validation-study
#2
Daniel P Howsmon, Troy Vargason, Robert A Rubin, Leanna Delhey, Marie Tippett, Shannon Rose, Sirish C Bennuri, John C Slattery, Stepan Melnyk, S Jill James, Richard E Frye, Juergen Hahn
Autism spectrum disorder (ASD) is a developmental disorder which is currently only diagnosed through behavioral testing. Impaired folate-dependent one carbon metabolism (FOCM) and transsulfuration (TS) pathways have been implicated in ASD, and recently a study involving multivariate analysis based upon Fisher Discriminant Analysis returned very promising results for predicting an ASD diagnosis. This article takes another step toward the goal of developing a biochemical diagnostic for ASD by comparing five classification algorithms on existing data of FOCM/TS metabolites, and also validating the classification results with new data from an ASD cohort...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065969/detachment-of-ligands-from-nanoparticle-surface-under-flow-and-endothelial-cell-contact-assessment-using-microfluidic-devices
#3
Maria Jarvis, Michael Arnold, Jenna Ott, Vinu Krishnan, Kapil Pant, Balabhaskar Prabhakarpandian, Samir Mitragotri
Surface modification of nanoparticles is a well-established methodology to alter their properties to enhance circulation half-life. While literature studies using conventional, in vitro characterization are routinely used to evaluate the biocompatibility of such modifications, relatively little attention has been paid to assess the stability of such surface modifications in physiologically relevant conditions. Here, microfluidic devices were used to study the effect of factors that adversely impact surface modifications including vascular flow and endothelial cell interactions...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065968/lipid-nanoparticle-sirna-cocktails-for-the-treatment-of-mantle-cell-lymphoma
#4
Christopher M Knapp, Jia He, John Lister, Kathryn A Whitehead
Mantle cell lymphoma is an aggressive and incurable subtype of non-Hodgkin B cell lymphoma. Patients typically present with advanced disease, and most patients succumb within a decade of diagnosis. There is a clear and urgent need for novel therapeutic approaches that will affect mantle cell lymphoma through a unique mechanism compared to current therapies. This study examined the use of RNA interference (RNAi) therapy to attack mantle cell lymphoma at the mRNA level, silencing genes associated with cancer cell proliferation...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065967/clinical-translation-of-microbe-based-therapies-current-clinical-landscape-and-preclinical-outlook
#5
REVIEW
Ava M Vargason, Aaron C Anselmo
Next generation microbe-based therapeutics, inspired by the success of fecal microbiota transplants, are being actively investigated in clinical trials to displace or eliminate pathogenic microbes to treat various diseases in the gastrointestinal tract, skin, and vagina. Genetically engineered microbes are also being investigated in the clinic as drug producing factories for biologic delivery, which can provide a constant local source of drugs. In either case, microbe-therapeutics have the opportunity to address unmet clinical needs and open new areas of research by reducing clinical side effects associated with current treatment modalities or by facilitating the delivery of biologics...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065966/harnessing-nanostructured-systems-for-improved-treatment-and-prevention-of-hiv-disease
#6
REVIEW
Maya Monroe, Charles Flexner, Honggang Cui
Combination antiretroviral therapy effectively controls human immunodeficiency virus (HIV) viral replication, delaying the progression to acquired immune deficiency syndrome and improving and extending quality of life of patients. However, the inability of antiretroviral therapeutics to target latent virus and their poor penetration of viral reserve tissues result in the need for continued treatment for the life of the patient. Side effects from long-term antiretroviral use and the development of drug resistance due to patient noncompliance are also continuing problems...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065965/scalable-synthesis-and-validation-of-pamam-dendrimer-n-acetyl-cysteine-conjugate-for-potential-translation
#7
Rishi Sharma, Anjali Sharma, Siva P Kambhampati, Rajsekar Rami Reddy, Zhi Zhang, Jeffrey L Cleland, Sujatha Kannan, Rangaramanujam M Kannan
Dendrimer- N -acetyl cysteine (D-NAC) conjugate has shown significant promise in multiple preclinical models of brain injury and is undergoing clinical translation. D-NAC is a generation-4 hydroxyl-polyamidoamine dendrimer conjugate where N -acetyl cysteine (NAC) is covalently bound through disulfide linkages on the surface of the dendrimer. It has shown remarkable potential to selectively target and deliver NAC to activated microglia and astrocytes at the site of brain injury in several animal models, producing remarkable improvements in neurological outcomes at a fraction of the free drug dose...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065964/tgf-%C3%AE-responsive-car-t-cells-promote-anti-tumor-immune-function
#8
Andrew J Hou, ZeNan L Chang, Michael H Lorenzini, Eugenia Zah, Yvonne Y Chen
A chimeric antigen receptor (CAR) that responds to transforming growth factor beta (TGF-β) enables the engineering of T cells that convert this immunosuppressive cytokine into a potent T-cell stimulant. However, clinical translation of TGF-β CAR-T cells for cancer therapy requires the ability to productively combine TGF-β responsiveness with tumor-targeting specificity. Furthermore, the potential concern that contaminating, TGF-β?producing regulatory T (Treg) cells may preferentially expand during TGF-β CAR-T cell manufacturing and suppress effector T (Teff) cells demands careful evaluation...
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065963/biotm-buzz-volume-3-issue-2
#9
EDITORIAL
Aaron C Anselmo
No abstract text is available yet for this article.
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/30065962/engineering-clinical-translation-introduction-to-special-issue-dedicated-to-2017-bioengineering-and-translational-medicine-conference
#10
EDITORIAL
Rangaramanujam M Kannan, Samir Mitragotri
No abstract text is available yet for this article.
May 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29376134/engineering-of-a-miniaturized-robotic-clinical-laboratory
#11
Marilyn B Nourse, Kate Engel, Samartha G Anekal, Jocelyn A Bailey, Pradeep Bhatta, Devayani P Bhave, Shekar Chandrasekaran, Yutao Chen, Steven Chow, Ushati Das, Erez Galil, Xinwei Gong, Steven F Gessert, Kevin D Ha, Ran Hu, Laura Hyland, Arvind Jammalamadaka, Karthik Jayasurya, Timothy M Kemp, Andrew N Kim, Lucie S Lee, Yang Lily Liu, Alphonso Nguyen, Jared O'Leary, Chinmay H Pangarkar, Paul J Patel, Ken Quon, Pradeep L Ramachandran, Amy R Rappaport, Joy Roy, Jerald F Sapida, Nikolay V Sergeev, Chandan Shee, Renuka Shenoy, Sharada Sivaraman, Bernardo Sosa-Padilla, Lorraine Tran, Amanda Trent, Thomas C Waggoner, Dariusz Wodziak, Amy Yuan, Peter Zhao, Daniel L Young, Channing R Robertson, Elizabeth A Holmes
The ability to perform laboratory testing near the patient and with smaller blood volumes would benefit patients and physicians alike. We describe our design of a miniaturized clinical laboratory system with three components: a hardware platform (ie, the miniLab) that performs preanalytical and analytical processing steps using miniaturized sample manipulation and detection modules, an assay-configurable cartridge that provides consumable materials and assay reagents, and a server that communicates bidirectionally with the miniLab to manage assay-specific protocols and analyze, store, and report results (i...
January 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29376133/schedule-dependent-synergy-of-gemcitabine-and-doxorubicin-improvement-of-in-vitro-efficacy-and-lack-of-in-vitro-in-vivo-correlation
#12
Douglas R Vogus, Anusha Pusuluri, Renwei Chen, Samir Mitragotri
Combination chemotherapy is commonly used to treat late stage cancer; however, treatment is often limited by systemic toxicity. Optimizing drug ratio and schedule can improve drug combination activity and reduce dose to lower toxicity. Here, we identify gemcitabine (GEM) and doxorubicin (DOX) as a synergistic drug pair in vitro for the triple negative breast cancer cell line MDA-MB-231. Drug synergy and caspase activity were increased the most by exposing cells to GEM prior to DOX in vitro. While the combination was more effective than the single drugs at inhibiting MDA-MB-231 growth in vivo, the clear schedule dependence observed in vitro was not observed in vivo...
January 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29376132/visible-light-crosslinkable-human-hair-keratin-hydrogels
#13
Kan Yue, Yanhui Liu, Batzaya Byambaa, Vaishali Singh, Wanjun Liu, Xiuyu Li, Yunxia Sun, Yu Shrike Zhang, Ali Tamayol, Peihua Zhang, Kee Woei Ng, Nasim Annabi, Ali Khademhosseini
Keratins extracted from human hair have emerged as a promising biomaterial for various biomedical applications, partly due to their wide availability, low cost, minimal immune response, and the potential to engineer autologous tissue constructs. However, the fabrication of keratin-based scaffolds typically relies on limited crosslinking mechanisms, such as via physical interactions or disulfide bond formation, which are time-consuming and result in relatively poor mechanical strength and stability. Here, we report the preparation of photocrosslinkable keratin-polyethylene glycol (PEG) hydrogels via the thiol-norbornene "click" reaction, which can be formed within one minute upon irradiation of visible light...
January 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29376131/rna-peptide-nanoplexes-drug-dna-damage-pathways-in-high-grade-serous-ovarian-tumors
#14
Erik C Dreaden, Yi Wen Kong, Mohiuddin A Quadir, Santiago Correa, Lucia Suárez-López, Antonio E Barberio, Mun Kyung Hwang, Aria C Shi, Benjamin Oberlton, Paige N Gallagher, Kevin E Shopsowitz, Kevin M Elias, Michael B Yaffe, Paula T Hammond
DNA damaging chemotherapy is a cornerstone of current front-line treatments for advanced ovarian cancer (OC). Despite the fact that a majority of these patients initially respond to therapy, most will relapse with chemo-resistant disease; therefore, adjuvant treatments that synergize with DNA-damaging chemotherapy could improve treatment outcomes and survival in patients with this deadly disease. Here, we report the development of a nanoscale peptide-nucleic acid complex that facilitates tumor-specific RNA interference therapy to chemosensitize advanced ovarian tumors to frontline platinum/taxane therapy...
January 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29376130/ionic-liquids-for-addressing-unmet-needs-in-healthcare
#15
REVIEW
Christian Agatemor, Kelly N Ibsen, Eden E L Tanner, Samir Mitragotri
Advances in the field of ionic liquids have opened new applications beyond their traditional use as solvents into other fields especially healthcare. The broad chemical space, rich with structurally diverse ions, and coupled with the flexibility to form complementary ion pairs enables task-specific optimization at the molecular level to design ionic liquids for envisioned functions. Consequently, ionic liquids now are tailored as innovative solutions to address many problems in medicine. To date, ionic liquids have been designed to promote dissolution of poorly soluble drugs and disrupt physiological barriers to transport drugs to targeted sites...
January 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29376129/biotm-buzz-volume-3-issue-1
#16
EDITORIAL
Aaron C Anselmo
No abstract text is available yet for this article.
January 2018: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29313037/soluble-collagen-vi-treatment-enhances-mesenchymal-stem-cells-expansion-for-engineering-cartilage
#17
Piera Smeriglio, Jieun Lee, Nidhi Bhutani
Bone Marrow-derived mesenchymal stem cells (BM-MSC) are an attractive source for cell-based therapies in cartilage injury owing to their efficient differentiation into chondrocytes and their immune-suppressive abilities. However, their clinical use is hampered by a scarcity of cells leading to compromised efficacy. While expansion of human MSC ex vivo can potentially overcome the scarcity of cells, current methods lead to a rapid loss of the stem cell properties. In this study, we report soluble Collagen VI (cartilage pericellular matrix component) as a potential biologic that can expand the MSC population while maintaining the stem cell phenotype as confirmed by expression of the stem cell markers CD105 and CD90...
September 2017: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29313036/microfluidic-co-culture-devices-to-assess-penetration-of-nanoparticles-into-cancer-cell-mass
#18
Maria Jarvis, Michael Arnold, Jenna Ott, Kapil Pant, Balabhaskar Prabhakarpandian, Samir Mitragotri
In vitro and in vivo assessment of safety and efficacy are the essential first steps in developing nanoparticle-based therapeutic systems. However, it is often challenging to use the knowledge gained from in vitro studies to predict the outcome of in vivo studies since the complexity of the in vivo environment, including the existence of flow and a multicellular environment, is often lacking in traditional in vitro models. Here, we describe a microfluidic co-culture model comprising 4T1 breast cancer cells and EA...
September 2017: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29313035/peptide-delivery-with-poly-ethylene-glycol-diacrylate-microneedles-through-swelling-effect
#19
Shiying Liu, David C Yeo, Christian Wiraja, Hong Liang Tey, Milan Mrksich, Chenjie Xu
Transdermal delivery of therapeutic biomolecules (including peptides) can avoid enzymatic digestion that occurs in the oral route. (Polyethylene glycol) diacrylate (PEGDA)-based microneedles, with good biocompatibility, are easily fabricated through photo-polymerization with a precisely controlled structure. It has successfully been used for the transdermal delivery of small molecule drugs such as 5-fluorouracil. However, the delivery of peptide-based therapeutics using this platform is seldom reported. This is because of the potential damage to the peptide during the photo-polymerization process of PEGDA...
September 2017: Bioengineering & Translational Medicine
https://www.readbyqxmd.com/read/29313034/the-wyss-institute-a-new-model-for-medical-technology-innovation-and-translation-across-the-academic-industrial-interface
#20
REVIEW
Mary Tolikas, Ayis Antoniou, Donald E Ingber
The Wyss Institute for Biologically Inspired Engineering at Harvard University was formed based on the recognition that breakthrough discoveries cannot change the world if they never leave the laboratory. The Institute's mission is to discover the biological principles that Nature uses to build living things, and to harness these insights to create biologically inspired engineering innovations to advance human health and create a more sustainable world. Since its launch in 2009, the Institute has developed a new model for innovation, collaboration, and technology translation within academia, breaking "silos" to enable collaborations that cross institutional and disciplinary barriers...
September 2017: Bioengineering & Translational Medicine
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