Progress in molecular biology and translational science最新文献

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Advances in CRISPR-Cas systems for blood cancer. CRISPR-Cas 系统在治疗血癌方面的进展。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-08-26 DOI: 10.1016/bs.pmbts.2024.07.004
Bernice Monchusi, Phumuzile Dube, Mutsa Monica Takundwa, Vanelle Larissa Kenmogne, Deepak Balaji Thimiri Govinda Raj
{"title":"Advances in CRISPR-Cas systems for blood cancer.","authors":"Bernice Monchusi, Phumuzile Dube, Mutsa Monica Takundwa, Vanelle Larissa Kenmogne, Deepak Balaji Thimiri Govinda Raj","doi":"10.1016/bs.pmbts.2024.07.004","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.07.004","url":null,"abstract":"<p><p>CRISPR-Cas systems have revolutionised precision medicine by enabling personalised treatments tailored to an individual's genetic profile. Various CRISPR technologies have been developed to target specific disease-causing genes in blood cancers, and some have advanced to clinical trials. Although some studies have explored the in vivo applications of CRISPR-Cas systems, several challenges continue to impede their widespread use. Furthermore, CRISPR-Cas technology has shown promise in improving the response of immunotherapies to blood cancers. The emergence of CAR-T cell therapy has shown considerable success in the targeting and correcting of disease-causing genes in blood cancers. Despite the promising potential of CRISPR-Cas in the treatment of blood cancers, issues related to safety, ethics, and regulatory approval remain significant hurdles. This comprehensive review highlights the transformative potential of CRISPR-Cas technology to revolutionise blood cancer therapy.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"208 ","pages":"261-284"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142294132","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Amyloid fibril cytotoxicity and associated disorders. 淀粉样纤维细胞毒性及相关疾病。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-03-30 DOI: 10.1016/bs.pmbts.2024.03.016
Sabereh Saremi, Khosro Khajeh
{"title":"Amyloid fibril cytotoxicity and associated disorders.","authors":"Sabereh Saremi, Khosro Khajeh","doi":"10.1016/bs.pmbts.2024.03.016","DOIUrl":"10.1016/bs.pmbts.2024.03.016","url":null,"abstract":"<p><p>Misfolded proteins assemble into fibril structures that are called amyloids. Unlike usually folded proteins, misfolded fibrils are insoluble and deposit extracellularly or intracellularly. Misfolded proteins interrupt the function and structure of cells and cause amyloid disease. There is increasing evidence that the most pernicious species are oligomers. Misfolded proteins disrupt cell function and cause cytotoxicity by calcium imbalance, mitochondrial dysfunction, and intracellular reactive oxygen species. Despite profound impacts on health, social, and economic factors, amyloid diseases remain untreatable. To develop new therapeutics and to understand the pathological manifestations of amyloidosis, research into the origin and pathology of amyloidosis is urgently needed. This chapter describes the basic concept of amyloid disease and the function of atypical amyloid deposits in them.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"206 ","pages":"265-290"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141176231","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Drug repurposing for respiratory infections. 针对呼吸道感染的药物再利用。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-05-23 DOI: 10.1016/bs.pmbts.2024.03.033
Juveriya Israr, Shabroz Alam, Ajay Kumar
{"title":"Drug repurposing for respiratory infections.","authors":"Juveriya Israr, Shabroz Alam, Ajay Kumar","doi":"10.1016/bs.pmbts.2024.03.033","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.03.033","url":null,"abstract":"<p><p>Respiratory infections such as Coronavirus disease 2019 are a substantial worldwide health challenge, frequently resulting in severe sickness and death, especially in susceptible groups. Conventional drug development for respiratory infections faces obstacles such as extended timescales, substantial expenses, and the rise of resistance to current treatments. Drug repurposing is a potential method that has evolved to quickly find and reuse existing medications for treating respiratory infections. Drug repurposing utilizes medications previously approved for different purposes, providing a cost-effective and time-efficient method to tackle pressing medical needs. This chapter summarizes current progress and obstacles in repurposing medications for respiratory infections, focusing on notable examples of repurposed pharmaceuticals and their probable modes of action. The text also explores the significance of computational approaches, high-throughput screening, and preclinical investigations in identifying potential candidates for repurposing. The text delves into the significance of regulatory factors, clinical trial structure, and actual data in confirming the effectiveness and safety of repurposed medications for respiratory infections. Drug repurposing is a valuable technique for quickly increasing the range of treatments for respiratory infections, leading to better patient outcomes and decreasing the worldwide disease burden.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"207 ","pages":"207-230"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141470524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Exploring the potential of drug repurposing for treating depression. 探索药物再利用治疗抑郁症的潜力。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-05-13 DOI: 10.1016/bs.pmbts.2024.03.037
Chaitenya Verma, Kritika Jain, Ashok Saini, Indra Mani, Vijai Singh
{"title":"Exploring the potential of drug repurposing for treating depression.","authors":"Chaitenya Verma, Kritika Jain, Ashok Saini, Indra Mani, Vijai Singh","doi":"10.1016/bs.pmbts.2024.03.037","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.03.037","url":null,"abstract":"<p><p>Researchers are interested in drug repurposing or drug repositioning of existing pharmaceuticals because of rising costs and slower rates of new medication development. Other investigations that authorized these treatments used data from experimental research and off-label drug use. More research into the causes of depression could lead to more effective pharmaceutical repurposing efforts. In addition to the loss of neurotransmitters like serotonin and adrenaline, inflammation, inadequate blood flow, and neurotoxins are now thought to be plausible mechanisms. Because of these other mechanisms, repurposing drugs has resulted for treatment-resistant depression. This chapter focuses on therapeutic alternatives and their effectiveness in drug repositioning. Atypical antipsychotics, central nervous system stimulants, and neurotransmitter antagonists have investigated for possible repurposing. Nonetheless, extensive research is required to ensure their formulation, effectiveness, and regulatory compliance.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"207 ","pages":"79-105"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141470528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Inhibitors of amyloid fibril formation. 淀粉样蛋白纤维形成抑制剂。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-04-22 DOI: 10.1016/bs.pmbts.2024.03.012
Elaheh Tavili, Fatemeh Aziziyan, Khosro Khajeh
{"title":"Inhibitors of amyloid fibril formation.","authors":"Elaheh Tavili, Fatemeh Aziziyan, Khosro Khajeh","doi":"10.1016/bs.pmbts.2024.03.012","DOIUrl":"10.1016/bs.pmbts.2024.03.012","url":null,"abstract":"<p><p>Many diseases are caused by misfolded and denatured proteins, leading to neurodegenerative diseases. In recent decades researchers have developed a variety of compounds, including polymeric inhibitors and natural compounds, antibodies, and chaperones, to inhibit protein aggregation, decrease the toxic effects of amyloid fibrils, and facilitate refolding proteins. The causes and mechanisms of amyloid formation are still unclear, and there are no effective treatments for Amyloid diseases. This section describes research and achievements in the field of inhibiting amyloid accumulation and also discusses the importance of various strategies in facilitating the removal of aggregates species (refolding) in the treatment of neurological diseases such as chemical methods like as, small molecules, metal chelators, polymeric inhibitors, and nanomaterials, as well as the use of biomolecules (peptide and, protein, nucleic acid, and saccharide) as amyloid inhibitors, are also highlighted.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"206 ","pages":"291-340"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141176241","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CRISPR-Cas based genome editing for eradication of human viruses. 基于 CRISPR-Cas 的基因组编辑技术,用于根除人类病毒。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-08-17 DOI: 10.1016/bs.pmbts.2024.07.012
Dharmisha Solanki, Karan Murjani, Vijai Singh
{"title":"CRISPR-Cas based genome editing for eradication of human viruses.","authors":"Dharmisha Solanki, Karan Murjani, Vijai Singh","doi":"10.1016/bs.pmbts.2024.07.012","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.07.012","url":null,"abstract":"<p><p>Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system possess a broad range of applications for genetic modification, diagnosis and treatment of infectious as well as non-infectious disease. The CRISPR-Cas system is found in bacteria and archaea that possess the Cas protein and guide RNA (gRNA). Cas9 and gRNA forms a complex to target and cleave the desired gene, providing defense against viral infections. Human immunodeficiency virus (HIV), hepatitis B virus (HBV), herpesviruses, human papillomavirus (HPV), and severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) cause major life threatening diseases which cannot cure completely by drugs. This chapter describes the present strategy of CRISPR-Cas systems for altering the genomes of viruses, mostly human ones, in order to control infections.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"208 ","pages":"43-58"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142294150","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Recent development in CRISPR-Cas systems for human protozoan diseases. 针对人类原生动物疾病的 CRISPR-Cas 系统的最新发展。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-08-17 DOI: 10.1016/bs.pmbts.2024.07.010
Utkarsh Gangwar, Himashree Choudhury, Risha Shameem, Yashi Singh, Abhisheka Bansal
{"title":"Recent development in CRISPR-Cas systems for human protozoan diseases.","authors":"Utkarsh Gangwar, Himashree Choudhury, Risha Shameem, Yashi Singh, Abhisheka Bansal","doi":"10.1016/bs.pmbts.2024.07.010","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.07.010","url":null,"abstract":"<p><p>Protozoan parasitic diseases pose a substantial global health burden. Understanding the pathogenesis of these diseases is crucial for developing intervention strategies in the form of vaccine and drugs. Manipulating the parasite's genome is essential for gaining insights into its fundamental biology. Traditional genomic manipulation methods rely on stochastic homologous recombination events, which necessitates months of maintaining the cultured parasites under drug pressure to generate desired transgenics. The introduction of mega-nucleases (MNs), zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs) greatly reduced the time required for obtaining a desired modification. However, there is a complexity associated with the design of these nucleases. CRISPR (Clustered regularly interspaced short palindromic repeats)/Cas (CRISPR associated proteins) is the latest gene editing tool that provides an efficient and convenient method for precise genomic manipulations in protozoan parasites. In this chapter, we have elaborated various strategies that have been adopted for the use of CRISPR-Cas9 system in Plasmodium, Leishmania and Trypanosoma. We have also discussed various applications of CRISPR-Cas9 pertaining to understanding of the parasite biology, development of drug resistance mechanism, gene drive and diagnosis of the infection.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"208 ","pages":"109-160"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142294154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Breakthroughs in synthetic controlling strategies for precision in CAR-T therapy. 为实现 CAR-T 疗法的精确性而制定的合成控制策略取得突破性进展。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-02-22 DOI: 10.1016/bs.pmbts.2024.02.002
Wang Tik Tang, Ryohichi Sugimura
{"title":"Breakthroughs in synthetic controlling strategies for precision in CAR-T therapy.","authors":"Wang Tik Tang, Ryohichi Sugimura","doi":"10.1016/bs.pmbts.2024.02.002","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.02.002","url":null,"abstract":"<p><p>Chimeric antigen receptors (CAR) are synthetic receptors engineered to target a user-defined antigen. They comprise an extracellular single-chain variable fragment for target recognition and intracellular signalling domains commonly derived from immune cells. CAR-T cells have proven to be successful in therapy of some cancers. CAR-T cells are activated upon antigen-priming and subsequent intracellular signalling. However, tonic signalling in CAR-T cells remains a challenge in developing CAR-T therapeutics of high efficacy as it causes early T-cell exhaustion, limiting therapeutic persistence. Moreover, a poor choice of target antigen leads to off-target cytotoxicity, often hampering the host's survival. In addition, conventional methods of delivering CAR gene circuits utilise viral vectors, such as lentiviruses and retroviruses, which insert the CAR gene circuits into transcriptionally active sites in the genome. This increases the risks of malignant transformation due to improper genome integration. Optimisation in CAR-T engineering, from the architecture of CAR gene circuits to the structure of CAR and the behaviour of CAR-T cells, is paramount to ensure high efficacy, persistence, and precision in CAR-T therapy. This review provides insights into engineering CAR-T cells for precision in cancer therapy by highlighting the key strategies recently developed to optimise the function and efficiency of CARs. The delivery method of CAR gene circuits, circuit and structural modification of CAR, T-cell phenotype manipulation and T-cell arming will be discussed to accentuate their interplay in regulating CAR-T therapy's safety, precision, and efficacy.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"209 ","pages":"61-100"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142506942","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Immunotherapy outcomes in non-small cell lung cancer according to a gender perspective. 从性别角度看非小细胞肺癌的免疫疗法效果。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-10-11 DOI: 10.1016/bs.pmbts.2024.09.004
Tiziana Vavalà
{"title":"Immunotherapy outcomes in non-small cell lung cancer according to a gender perspective.","authors":"Tiziana Vavalà","doi":"10.1016/bs.pmbts.2024.09.004","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.09.004","url":null,"abstract":"<p><p>In the last few years, immune checkpoint inhibitors (ICIs) improved treatment strategies for advanced non-small cell lung cancer (NSCLC) with no targetable driver mutations. Empirical evidence strongly suggests that males and females differ in outcomes following the use of ICIs for treatments of solid cancers. Women in fact exhibit greater humoral and cell-mediated immune responses and an even more advanced immune editing which plays an important role in controlling cancer rising and evolution. However, at present, no conclusive studies have addressed differences in response to ICIs regarding sex and, to note, reproductive status in women or autoimmune diseases in both sexes are often not recorded in clinical trials. Consequently, it can be argued that to assess cancer responses and study cancer spread, results of published studies in men may not unconditionally be applied on female patients treated with ICIs, and vice versa. In this chapter have been discussed recent data about gender differences in the immune system and in NSCLC patients treated with ICIs, highlighting sex as a key factor in evaluating different responses in the two sexes.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"209 ","pages":"241-258"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142506945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advances in RNA therapeutics for modulation of 'undruggable' targets. 调节 "不可药用 "靶点的 RNA 疗法的进展。
3区 生物学
Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-01-17 DOI: 10.1016/bs.pmbts.2023.12.003
Emily Martinsen, Tasmia Jinnurine, Saranya Subramani, Marie Rogne
{"title":"Advances in RNA therapeutics for modulation of 'undruggable' targets.","authors":"Emily Martinsen, Tasmia Jinnurine, Saranya Subramani, Marie Rogne","doi":"10.1016/bs.pmbts.2023.12.003","DOIUrl":"10.1016/bs.pmbts.2023.12.003","url":null,"abstract":"<p><p>Over the past decades, drug discovery utilizing small pharmacological compounds, fragment-based therapeutics, and antibody therapy have significantly advanced treatment options for many human diseases. However, a major bottleneck has been that>70% of human proteins/genomic regions are 'undruggable' by the above-mentioned approaches. Many of these proteins constitute essential drug targets against complex multifactorial diseases like cancer, immunological disorders, and neurological diseases. Therefore, alternative approaches are required to target these proteins or genomic regions in human cells. RNA therapeutics is a promising approach for many of the traditionally 'undruggable' targets by utilizing methods such as antisense oligonucleotides, RNA interference, CRISPR/Cas-based genome editing, aptamers, and the development of mRNA therapeutics. In the following chapter, we will put emphasis on recent advancements utilizing these approaches against challenging drug targets, such as intranuclear proteins, intrinsically disordered proteins, untranslated genomic regions, and targets expressed in inaccessible tissues.</p>","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"204 ","pages":"249-294"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140065803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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