• Title/Summary/Keyword: Therapeutic Proteins

Search Result 593, Processing Time 0.029 seconds

PIWI Proteins and piRNAs in the Nervous System

  • Kim, Kyung Won
    • Molecules and Cells
    • /
    • v.42 no.12
    • /
    • pp.828-835
    • /
    • 2019
  • PIWI Argonaute proteins and Piwi-interacting RNAs (piRNAs) are expressed in all animal species and play a critical role in cellular defense by inhibiting the activation of transposable elements in the germline. Recently, new evidence suggests that PIWI proteins and piRNAs also play important roles in various somatic tissues, including neurons. This review summarizes the neuronal functions of the PIWI-piRNA pathway in multiple animal species, including their involvement in axon regeneration, behavior, memory formation, and transgenerational epigenetic inheritance of adaptive memory. This review also discusses the consequences of dysregulation of neuronal PIWI-piRNA pathways in certain neurological disorders, including neurodevelopmental and neurodegenerative diseases. A full understanding of neuronal PIWI-piRNA pathways will ultimately provide novel insights into small RNA biology and could potentially provide precise targets for therapeutic applications.

RNA-Binding Proteins in Amyotrophic Lateral Sclerosis

  • Zhao, Melody;Kim, Jihye Rachel;van Bruggen, Rebekah;Park, Jeehye
    • Molecules and Cells
    • /
    • v.41 no.9
    • /
    • pp.818-829
    • /
    • 2018
  • Significant research efforts are ongoing to elucidate the complex molecular mechanisms underlying amyotrophic lateral sclerosis (ALS), which may in turn pinpoint potential therapeutic targets for treatment. The ALS research field has evolved with recent discoveries of numerous genetic mutations in ALS patients, many of which are in genes encoding RNA binding proteins (RBPs), including TDP-43, FUS, ATXN2, TAF15, EWSR1, hnRNPA1, hnRNPA2/B1, MATR3 and TIA1. Accumulating evidence from studies on these ALS-linked RBPs suggests that dysregulation of RNA metabolism, cytoplasmic mislocalization of RBPs, dysfunction in stress granule dynamics of RBPs and increased propensity of mutant RBPs to aggregate may lead to ALS pathogenesis. Here, we review current knowledge of the biological function of these RBPs and the contributions of ALS-linked mutations to disease pathogenesis.

Conjugation of Protein and Peptide Drugs with Hydrophilic Polymers and Their Applications (수용성 고분자물질-단백질 접합체의 합성 및 응용)

  • Yong, Chul-Soon;Sohn, Young-Taek
    • Journal of Pharmaceutical Investigation
    • /
    • v.23 no.4
    • /
    • pp.187-206
    • /
    • 1993
  • Since the advent of recombinant DNA technology coupled with other biotechnology a variety of therapeutically effective proteins and peptides have been extensively invesitigated and many of them are now on clinical trial. They, however, suffer from some problems such as immunogenicity, antigenicity, instability and short half-life in circulation due to their proteinous natures. These drawbacks can be overcome successfully by conjugating proteins and peptides with hydrophilic polymers such as polyethylene glycol (PEG), albumin or dextran. The resulting soluble conjugates showed reduced antigenicity and immunogenicity, increased circulatory half-life, enhanced stability against proteolytic degradation. Comparing with the unmodified proteins and peptides, the therapeutic potential of conjugates is greatly enhanced. Clinical applications of these conjugates have shown promising results for the future use.

  • PDF

The Role of Proprotein Convertases in Upper Airway Remodeling

  • Lee, Sang-Nam;Yoon, Joo-Heon
    • Molecules and Cells
    • /
    • v.45 no.6
    • /
    • pp.353-361
    • /
    • 2022
  • Chronic rhinosinusitis (CRS) is a multifactorial, heterogeneous disease characterized by persistent inflammation of the sinonasal mucosa and tissue remodeling, which can include basal/progenitor cell hyperplasia, goblet cell hyperplasia, squamous cell metaplasia, loss or dysfunction of ciliated cells, and increased matrix deposition. Repeated injuries can stimulate airway epithelial cells to produce inflammatory mediators that activate epithelial cells, immune cells, or the epithelial-mesenchymal trophic unit. This persistent inflammation can consequently induce aberrant tissue remodeling. However, the molecular mechanisms driving disease within the different molecular CRS subtypes remain inadequately characterized. Numerous secreted and cell surface proteins relevant to airway inflammation and remodeling are initially synthesized as inactive precursor proteins, including growth/differentiation factors and their associated receptors, enzymes, adhesion molecules, neuropeptides, and peptide hormones. Therefore, these precursor proteins require post-translational cleavage by proprotein convertases (PCs) to become fully functional. In this review, we summarize the roles of PCs in CRS-associated tissue remodeling and discuss the therapeutic potential of targeting PCs for CRS treatment.

Pharmacological and Adverse Effects of Aloe vera

  • Lee, Kyoung-Rim;Yoo, Sun-Dong;Lee, Byung-Mu
    • Proceedings of the PSK Conference
    • /
    • 2003.04a
    • /
    • pp.149.1-149.1
    • /
    • 2003
  • Active ingredients, therapeutic and adverse effects of Aloe vera were comprehensively investigated. Aloe vera contains active components. including sugars, proteins, lipids, vitamins, minerals, phenolic compounds and other several compounds like phthalate esters, gibberellin, lectin-like substances, lignin, saponins, salicylic acid and uric acid. (omitted)

  • PDF

Production of Recombinant Anti-Cancer Vaccines in Plants

  • Lee, Jeong Hwan;Ko, Kisung
    • Biomolecules & Therapeutics
    • /
    • v.25 no.4
    • /
    • pp.345-353
    • /
    • 2017
  • Plant expression systems have been developed to produce anti-cancer vaccines. Plants have several advantages as bioreactors for the production of subunit vaccines: they are considered safe, and may be used to produce recombinant proteins at low production cost. However, several technical issues hinder large-scale production of anti-cancer vaccines in plants. The present review covers design strategies to enhance the immunogenicity and therapeutic potency of anti-cancer vaccines, methods to increase vaccine-expressing plant biomass, and challenges facing the production of anti-cancer vaccines in plants. Specifically, the issues such as low expression levels and plant-specific glycosylation are described, along with their potential solutions.

Polycystic kidney disease and therapeutic approaches

  • Park, Eun-Young;Woo, Yu-Mi;Park, Jong-Hoon
    • BMB Reports
    • /
    • v.44 no.6
    • /
    • pp.359-368
    • /
    • 2011
  • Polycystic kidney disease (PKD) is a common genetic disorder in which extensive epithelial-lined cysts develop in the kidneys. In previous studies, abnormalities of polycystin protein and its interacting proteins, as well as primary cilia, have been suggested to play critical roles in the development of renal cysts. However, although several therapeutic targets for PKD have been suggested, no early diagnosis or effective treatments are currently available. Current developments are active for treatment of PKD including inhibitors or antagonists of PPAR-${\gamma}$, TNF-${\alpha}$, CDK and VEGF. These drugs are potential therapeutic targets in PKD, and need to be determined about pathological functions in human PKD. It has recently been reported that the alteration of epigenetic regulation, as well as gene mutations, may affect the pathogenesis of PKD. In this review, we will discuss recent approaches to PKD therapy. It provides important information regarding potential targets for PKD.

Engineered adult stem cells: a promising tool for anti-cancer therapy

  • Youngdong Choi;Hong Kyu Lee;Kyung-Chul Choi
    • BMB Reports
    • /
    • v.56 no.2
    • /
    • pp.71-77
    • /
    • 2023
  • Cancers are one of the most dreaded diseases in human history and have been targeted by numerous trials including surgery, chemotherapy, radiation therapy, and anti-cancer drugs. Adult stem cells (ASCs), which can regenerate tissues and repair damage, have emerged as leading therapeutic candidates due to their homing ability toward tumor foci. Stem cells can precisely target malicious tumors, thereby minimizing the toxicity of normal cells and unfavorable side effects. ASCs, such as mesenchymal stem cells (MSCs), neural stem cells (NSCs), and hematopoietic stem cells (HSCs), are powerful tools for delivering therapeutic agents to various primary and metastatic cancers. Engineered ASCs act as a bridge between the tumor sites and tumoricidal reagents, producing therapeutic substances such as exosomes, viruses, and anti-cancer proteins encoded by several suicide genes. This review focuses on various anti-cancer therapies implemented via ASCs and summarizes the recent treatment progress and shortcomings.

Injectable hydrogels delivering therapeutic agents for disease treatment and tissue engineering

  • Lee, Jin Hyun
    • Biomaterials Research
    • /
    • v.22 no.4
    • /
    • pp.235-248
    • /
    • 2018
  • Background: Injectable hydrogels have been extensively researched for the use as scaffolds or as carriers of therapeutic agents such as drugs, cells, proteins, and bioactive molecules in the treatment of diseases and cancers and the repair and regeneration of tissues. It is because they have the injectability with minimal invasiveness and usability for irregularly shaped sites, in addition to typical advantages of conventional hydrogels such as biocompatibility, permeability to oxygen and nutrient, properties similar to the characteristics of the native extracellular matrix, and porous structure allowing therapeutic agents to be loaded. Main body: In this article, recent studies of injectable hydrogel systems applicable for therapeutic agent delivery, disease/cancer therapy, and tissue engineering have reviewed in terms of the various factors physically and chemically contributing to sol-gel transition via which gels have been formed. The various factors are as follows: several different non-covalent interactions resulting in physical crosslinking (the electrostatic interactions (e.g., the ionic and hydrogen bonds), hydrophobic interactions, ${\pi}$-interactions, and van der Waals forces), in-situ chemical reactions inducing chemical crosslinking (the Diels Alder click reactions, Michael reactions, Schiff base reactions, or enzyme-or photo-mediated reactions), and external stimuli (temperatures, pHs, lights, electric/magnetic fields, ultrasounds, or biomolecular species (e.g., enzyme)). Finally, their applications with accompanying therapeutic agents and notable properties used were reviewed as well. Conclusion: Injectable hydrogels, of which network morphology and properties could be tuned, have shown to control the load and release of therapeutic agents, consequently producing significant therapeutic efficacy. Accordingly, they are believed to be successful and promising biomaterials as scaffolds and carriers of therapeutic agents for disease and cancer therapy and tissue engineering.