A human-on-a-chip approach to tackling rare diseases

Mello, Camilly P Pires de; Rumsey, John W.; Slaughter, Victoria L.; Hickman, James J.

doi:10.1016/j.drudis.2019.08.001

Cited by 41 publications

(33 citation statements)

References 141 publications

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“…Scaling down of bioanalytical activities, in order to decrease production expenses, as well as simplifying transport and saving space in the laboratory has led to a focus on laboratory-on-a-chip technology. Overall, scaling down improves the efficiency of required screening [ 56 , 57 ]. However, this could be complicated by extensive time implications, error-recovery rates, and complex experimental design often involving an error-prone robotic operation.…”

Section: High-throughput Screening (Hts)mentioning

confidence: 99%

High-Throughput Screening Platforms in the Discovery of Novel Drugs for Neurodegenerative Diseases

et al. 2021

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Neurodegenerative diseases (NDDs) are incurable and debilitating conditions that result in progressive degeneration and/or death of nerve cells in the central nervous system (CNS). Identification of viable therapeutic targets and new treatments for CNS disorders and in particular, for NDDs is a major challenge in the field of drug discovery. These difficulties can be attributed to the diversity of cells involved, extreme complexity of the neural circuits, the limited capacity for tissue regeneration, and our incomplete understanding of the underlying pathological processes. Drug discovery is a complex and multidisciplinary process. The screening attrition rate in current drug discovery protocols mean that only one viable drug may arise from millions of screened compounds resulting in the need to improve discovery technologies and protocols to address the multiple causes of attrition. This has identified the need to screen larger libraries where the use of efficient high-throughput screening (HTS) becomes key in the discovery process. HTS can investigate hundreds of thousands of compounds per day. However, if fewer compounds could be screened without compromising the probability of success, the cost and time would be largely reduced. To that end, recent advances in computer-aided design, in silico libraries, and molecular docking software combined with the upscaling of cell-based platforms have evolved to improve screening efficiency with higher predictability and clinical applicability. We review, here, the increasing role of HTS in contemporary drug discovery processes, in particular for NDDs, and evaluate the criteria underlying its successful application. We also discuss the requirement of HTS for novel NDD therapies and examine the major current challenges in validating new drug targets and developing new treatments for NDDs.

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Section: High-throughput Screening (Hts)mentioning

confidence: 99%

High-Throughput Screening Platforms in the Discovery of Novel Drugs for Neurodegenerative Diseases

et al. 2021

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“…Finding new therapeutics for rare diseases [162,163], which affect a small population, is another expected application for OOC devices in the future. Small market size and high expenses to develop new treatments for rare disease are the main reasons that most pharmaceutical companies are not interested in investing to develop treatments for rare diseases.…”

Section: Discussion and Future Perspectivesmentioning

confidence: 99%

Evolution of Biochip Technology: A Review from Lab-on-a-Chip to Organ-on-a-Chip

et al. 2020

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Following the advancements in microfluidics and lab-on-a-chip (LOC) technologies, a novel biomedical application for microfluidic based devices has emerged in recent years and microengineered cell culture platforms have been created. These micro-devices, known as organ-on-a-chip (OOC) platforms mimic the in vivo like microenvironment of living organs and offer more physiologically relevant in vitro models of human organs. Consequently, the concept of OOC has gained great attention from researchers in the field worldwide to offer powerful tools for biomedical researches including disease modeling, drug development, etc. This review highlights the background of biochip development. Herein, we focus on applications of LOC devices as a versatile tool for POC applications. We also review current progress in OOC platforms towards body-on-a-chip, and we provide concluding remarks and future perspectives for OOC platforms for POC applications.

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“…Although the research in engineered models of rare diseases is at its preliminary state, there have already been successful pilot studies. For instance, various multi-organ-on-a-chip platforms based on iPSCs and CRISPR technology have been proposed to induce specific mutations in an engineered cell population to study the autoimmune response and to understand the complex mechanism of autoimmune diseases, such as type I diabetes, rheumatoid arthritis, and celiac disease ( de Mello et al, 2019 ). Since the severity of autoimmune diseases varies considerably amongst individuals, the potential to model combinations of genetic, environmental, and cellular components makes multi-organ-on-a-chip platforms uniquely able to determine the most relevant factors in disease progression for specific patients.…”

Section: Tackling Current Biomedical Challenges With Frontier Technologiesmentioning

confidence: 99%

Tackling Current Biomedical Challenges With Frontier Biofabrication and Organ-On-A-Chip Technologies

Celikkin

Presutti

Maiullari

et al. 2021

Front. Bioeng. Biotechnol.

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In the last decades, biomedical research has significantly boomed in the academia and industrial sectors, and it is expected to continue to grow at a rapid pace in the future. An in-depth analysis of such growth is not trivial, given the intrinsic multidisciplinary nature of biomedical research. Nevertheless, technological advances are among the main factors which have enabled such progress. In this review, we discuss the contribution of two state-of-the-art technologies–namely biofabrication and organ-on-a-chip–in a selection of biomedical research areas. We start by providing an overview of these technologies and their capacities in fabricating advanced in vitro tissue/organ models. We then analyze their impact on addressing a range of current biomedical challenges. Ultimately, we speculate about their future developments by integrating these technologies with other cutting-edge research fields such as artificial intelligence and big data analysis.

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A human-on-a-chip approach to tackling rare diseases

Cited by 41 publications

References 141 publications

High-Throughput Screening Platforms in the Discovery of Novel Drugs for Neurodegenerative Diseases

High-Throughput Screening Platforms in the Discovery of Novel Drugs for Neurodegenerative Diseases

Evolution of Biochip Technology: A Review from Lab-on-a-Chip to Organ-on-a-Chip

Tackling Current Biomedical Challenges With Frontier Biofabrication and Organ-On-A-Chip Technologies

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