• 제목/요약/키워드: clinical therapeutics

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Clinical Pharmacology in Children

  • Park Min-Soo
    • 대한임상약리학회:학술대회논문집
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    • 대한임상약리학회 1996년도 정기총회 및 추계학술대회
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    • pp.12-12
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    • 1996
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Therapeutic aptamers: developmental potential as anticancer drugs

  • Lee, Ji Won;Kim, Hyun Jung;Heo, Kyun
    • BMB Reports
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    • 제48권4호
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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]

Manufacturing Therapeutic Exosomes: from Bench to Industry

  • Ahn, So-Hee;Ryu, Seung-Wook;Choi, Hojun;You, Sangmin;Park, Jun;Choi, Chulhee
    • Molecules and Cells
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    • 제45권5호
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    • pp.284-290
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    • 2022
  • Exosome, a type of nanoparticles also known as small extracellular vesicles are gaining attention as novel therapeutics for various diseases because of their ability to deliver genetic or bioactive molecules to recipient cells. Although many pharmaceutical companies are gradually developing exosome therapeutics, numerous hurdles remain regarding manufacture of clinical-grade exosomes for therapeutic use. In this mini-review, we will discuss the manufacturing challenges of therapeutic exosomes, including cell line development, upstream cell culture, and downstream purification process. In addition, developing proper formulations for exosome storage and, establishing good manufacturing practice facility for producing therapeutic exosomes remains as challenges for developing clinical-grade exosomes. However, owing to the lack of consensus regarding the guidelines for manufacturing therapeutic exosomes, close communication between regulators and companies is required for the successful development of exosome therapeutics. This review shares the challenges and perspectives regarding the manufacture and quality control of clinical grade exosomes.

Sex Differences in the Preventive Effect of Cardiovascular and Metabolic Therapeutics on Dementia

  • Sun Ah Choi;Hye Jin Jee;Katrina Joy Bormate;Yeonjae Kim;Yi-Sook Jung
    • Biomolecules & Therapeutics
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    • 제31권6호
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    • pp.583-598
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    • 2023
  • Dementia is a clinical syndrome characterized by progressive impairment of cognitive and functional abilities. As currently applied treatments for dementia can only delay the progression of dementia and cannot fundamentally cure it, much attention is being paid to reducing its incidence by preventing the associated risk factors. Cardiovascular and metabolic diseases are well-known risk factors for dementia, and many studies have attempted to prevent dementia by treating these risk factors. Growing evidence suggests that sex-based factors may play an important role in the pathogenesis of dementia. Therefore, a deeper understanding of the differences in the effects of drugs based on sex may help improve their effectiveness. In this study, we reviewed sex differences in the impact of therapeutics targeting risk factors for dementia, such as cardiovascular and metabolic diseases, to prevent the incidence and/or progression of dementia.

Acute Radiation Syndrome in an Irradiated Minipig Model for Patients with Radiation Exposure

  • Jang, Hyosun;Kim, Joong-sun;Shim, Sehwan;Jang, Won-seok;Lee, Sun-Joo;Myung, Jae Kyung;Lee, Seung-Sook;Park, Sunhoo
    • Journal of Radiation Protection and Research
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    • 제42권3호
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    • pp.146-153
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    • 2017
  • Background: Acute radiation syndrome (ARS) primarily refers to damage to the hematopoietic system, myeloid system, and gastrointestinal (GI) system caused by radiation exposure. Such damage progresses to become life-threatening. In particular, as the syndrome develops very rapidly-within several hours from radiation exposure-prompt and accurate diagnosis and treatment are needed, as is further research into appropriate diagnostic and treatment modalities. Materials and Methods: Minipigs, which display human-like properties, underwent whole-body irradiation at 2 or 4 Gy (doses causing hematopoietic ARS) or at higher doses of 7 or 12 Gy. Changes in the blood cells and clinical symptoms were analyzed and we performed a necropsy when the animals succumbed to ARS. Results and Discussion: The minipig irradiated with 2 Gy showed a decrease in white blood cells, including neutrophils, lymphocytes, and platelets in the early stages. However, the blood cell counts gradually increased and returned to normal values. The minipig irradiated with 4 Gy succumbed due to hematopoietic ARS. In contrast, the minipigs irradiated with 7 or 12 Gy exhibited clinical symptoms of combined GI damage and hematopoietic syndrome. Moreover, a characteristic pattern of platelet changes was observed in the 7 and 12 Gy irradiated minipigs. Conclusion: The changes in the platelet count caused by radiation exposure observed in minipigs, which are hematologically and pathohistologically similar to humans, suggest that they can be used as a novel diagnostic criterion.

Development of mRNA Vaccines/Therapeutics and Their Delivery System

  • Sora Son;Kyuri Lee
    • Molecules and Cells
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    • 제46권1호
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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.