• Title/Summary/Keyword: therapeutics

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A One-Step System for Convenient and Flexible Assembly of Transcription Activator-Like Effector Nucleases (TALENs)

  • Zhao, Jinlong;Sun, Wenye;Liang, Jing;Jiang, Jing;Wu, Zhao
    • Molecules and Cells
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    • v.39 no.9
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    • pp.687-691
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    • 2016
  • Transcription activator-like effector nucleases (TALENs) are powerful tools for targeted genome editing in diverse cell types and organisms. However, the highly identical TALE repeat sequences make it challenging to assemble TALEs using conventional cloning approaches, and multiple repeats in one plasmid are easily catalyzed for homologous recombination in bacteria. Although the methods for TALE assembly are constantly improving, these methods are not convenient because of laborious assembly steps or large module libraries, limiting their broad utility. To overcome the barrier of multiple assembly steps, we report a one-step system for the convenient and flexible assembly of a 180 TALE module library. This study is the first demonstration to ligate 9 mono-/dimer modules and one circular TALEN backbone vector in a one step process, generating 9.5 to 18.5 repeat sequences with an overall assembly rate higher than 50%. This system makes TALEN assembly much simpler than the conventional cloning of two DNA fragments because this strategy combines digestion and ligation into one step using circular vectors and different modules to avoid gel extraction. Therefore, this system provides a convenient tool for the application of TALEN-mediated genome editing in scientific studies and clinical trials.

Experiences of Precocious Puberty in Primary School Girls with Hormone Therapeutics (호르몬 치료를 받은 초등학교 여아의 성조숙증 경험)

  • Cheon, Soon-Mi;Jung, Hye-Young
    • The Journal of Korean Academic Society of Nursing Education
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    • v.25 no.4
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    • pp.459-470
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    • 2019
  • Purpose: The purpose of this qualitative study was to identify the nature of precocious puberty and to explore what it means in primary schools. Methods: The participants of this phenomenological study were nine primary school girls who were diagnosed with precocious puberty and experienced hormone therapeutics, applying a convenience sampling method. Data were collected from July 2017 to January 2018 through individual in-depth interviews of the participants, including gestures, facial expressions and nonverbal means. The data analysis followed the method of Giorgi. Results: The study identified 37 concepts, 12 clusters, and five themes from experiences of precocious puberty. The essential five themes were as follows: 'ashamed and concealing experience', 'there is no therapeutics option', 'difficulties in the process of therapeutics', 'difficulties in daily life', and 'ambivalence toward therapeutics'. Conclusion: The findings of this study indicate physical, psychological and social difficulties faced by girls with precocious puberty. Based on this results of the study, it is necessary to develop nursing intervention programs focusing on healthy growth and development for children with precocious puberty.

Ultrafiltration and Diafiltration Processes for Concentration and Formulation of Antibody-based Therapeutics (항체의약품 농축 및 제제화를 위한 한외여과 및 정용여과 공정)

  • Lee, Jieun;Lee, Jiyoon;Baek, Youngbin
    • Membrane Journal
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    • v.30 no.6
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    • pp.373-384
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    • 2020
  • Antibody-based therapeutics have been receiving great attention as a representative biopharmaceutical, in which many researches are also carried out for its commercialization. The downstream process is considered an important part of the manufacturing processes of antibody-based therapeutics since it directly affects the performance and stability of products. Ultrafiltration/diafiltration (UF/DF), mostly performed in final step during downstream process, are used for the final concentration and formulation of antibody-based therapeutics. This paper reviewed the major products of the UF membrane, process characteristics, and recent research trends in UF/DF.

Development of mRNA Vaccines/Therapeutics and Their Delivery System

  • Sora Son;Kyuri Lee
    • Molecules and Cells
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    • v.46 no.1
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    • pp.41-47
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    • 2023
  • The rapid development of mRNA vaccines has contributed to the management of the current coronavirus disease 2019 (COVID-19) pandemic, suggesting that this technology may be used to manage future outbreaks of infectious diseases. Because the antigens targeted by mRNA vaccines can be easily altered by simply changing the sequence present in the coding region of mRNA structures, it is more appropriate to develop vaccines, especially during rapidly developing outbreaks of infectious diseases. In addition to allowing rapid development, mRNA vaccines have great potential in inducing successful antigen-specific immunity by expressing target antigens in cells and simultaneously triggering immune responses. Indeed, the two COVID-19 mRNA vaccines approved by the U.S. Food and Drug Administration have shown significant efficacy in preventing infections. The ability of mRNAs to produce target proteins that are defective in specific diseases has enabled the development of options to treat intractable diseases. Clinical applications of mRNA vaccines/therapeutics require strategies to safely deliver the RNA molecules into targeted cells. The present review summarizes current knowledge about mRNA vaccines/ therapeutics, their clinical applications, and their delivery strategies.

Digital Therapeutics for Obesity Care (비만 관리를 위한 디지털 치료제)

  • Seo, Yoo Bin
    • Archives of Obesity and Metabolism
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    • v.1 no.2
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    • pp.47-52
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    • 2022
  • Digital therapeutics (DTx) are emerging as a novel solution to improve lifestyle and prevent non-communicable diseases. Obesity is a complex, multi-factorial, chronic condition that requires patient-centered lifestyle modification. DTx, such as mobile applications and wearables, may offer easily accessible, efficient, and personalized care in the field of obesity and metabolic diseases. Yet, there is controversy over its clinical usefulness. This review will provide a comprehensive overview of DTx, including its potential role and current limitation in obesity care, based on recent literature.

Nomenclature of emerging therapeutics in neurology

  • Shin, Jin-Hong;Park, Young-Eun;Kim, Dae-Seong
    • Annals of Clinical Neurophysiology
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    • v.23 no.1
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    • pp.29-34
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    • 2021
  • New therapeutics in neurology are expanding at an unprecedented pace. In addition to the classic enzyme-replacement therapies, monoclonal antibodies are increasingly being used to modulate autoimmunity. RNA therapeutics are an emerging class, together with gene and cell therapies. The nomenclature of international nonproprietary names helps us to recognize these new drugs according to their class and function. Suffixes denote major categories of the drug, while infixes provide additional information such as the source and target.

Cholesterol conjugated spermine as a delivery modality of antisense oligonucleotide

  • Im, Yoon Kyung;Kim, Myung Su;Yoo, Hoon
    • International Journal of Oral Biology
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    • v.38 no.4
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    • pp.155-160
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    • 2013
  • The major issue in the development of nucleic acid based therapeutics is the inefficient delivery of these agents into cells. We prepared cholesterol conjugated spermine and evaluated its usefulness as a delivery modality for antisense oligonucleotides in HeLa-Luc cells. A 2'-O-methyl antisense oligonucleotide sequence, designed to correct splicing at an aberrant intron inserted into a normal luciferase reporter gene, was used for complex formation with cholesterol conjugated spermine. Effective delivery of this antisense agent into nucleus would results in the expression of a luciferasereporter gene product. The cholesterol-spermine formed stable complexes with the antisense oligonucleotide and showed modest delivery activity. Furthermore, this delivery activity was maintained even in the presence of serum proteins, mimicking in vivo conditions. Cholesterol-spermine thus has potential as a delivery system for antisense oligonucleotides into cells.

Therapeutic aptamers: developmental potential as anticancer drugs

  • Lee, Ji Won;Kim, Hyun Jung;Heo, Kyun
    • BMB Reports
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    • v.48 no.4
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    • pp.234-237
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    • 2015
  • Aptamers, composed of single-stranded DNA or RNA oligonucleotides that interact with target molecules through a specific three-dimensional structure, are selected from pools of combinatorial oligonucleotide libraries. With their high specificity and affinity for target proteins, ease of synthesis and modification, and low immunogenicity and toxicity, aptamers are considered to be attractive molecules for development as anticancer therapeutics. Two aptamers - one targeting nucleolin and a second targeting CXCL12 - are currently undergoing clinical trials for treating cancer patients, and many more are under study. In this mini-review, we present the current clinical status of aptamers and aptamer-based cancer therapeutics. We also discuss advantages, limitations, and prospects for aptamers as cancer therapeutics. [BMB Reports 2015; 48(4): 234-237]