Progress in molecular biology and translational science最新文献_第4页

RNA therapeutics for β-thalassemia. 治疗β地中海贫血症的 RNA 疗法。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-01-24 DOI: 10.1016/bs.pmbts.2023.12.006

Hong-Quan Duong, Thi-Hue Nguyen, Minh-Cong Hoang, Van-Lang Ngo, Van-Thu Le

{"title":"RNA therapeutics for β-thalassemia.","authors":"Hong-Quan Duong, Thi-Hue Nguyen, Minh-Cong Hoang, Van-Lang Ngo, Van-Thu Le","doi":"10.1016/bs.pmbts.2023.12.006","DOIUrl":"10.1016/bs.pmbts.2023.12.006","url":null,"abstract":"β-thalassemia is an autosomal recessive disease, caused by one or more mutations in the β-globin gene that reduces or abolishes β-globin chain synthesis causing an imbalance in the ratio of α- and β-globin chain. Therefore, the ability to target mutations will provide a good result in the treatment of β-thalassemia. RNA therapeutics represents a promising class of drugs inclusive antisense oligonucleotides (ASO), small interfering RNA (siRNA), microRNA (miRNA) and APTAMER have investigated in clinical trials for treatment of human diseases as β-thalassemia; Especially, ASO therapeutics can completely treat β-thalassemia patients by the way of making ASO infiltrating through erythrocyte progenitor cells, migrating to the nucleus and hybridizing with abnormal splicing sites to suppress an abnormal splicing pattern of β-globin pre-mRNA. As a result, the exactly splicing process is restored to increase the expression of β-globin which increases the amount of mature hemoglobin of red blood cells of β-thalassemia patients. Furthermore, current study demonstrates that RNA-based therapeutics get lots of good results for β-thalassemia patients. Then, this chapter focuses on current advances of RNA-based therapeutics and addresses current challenges with their development and application for treatment of β-thalassemia patients.","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"204 ","pages":"97-107"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140065813","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 CRISPR/Cas systems-based cell and gene therapy. 基于 CRISPR/Cas 系统的细胞和基因疗法的进展。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-08-12 DOI: 10.1016/bs.pmbts.2024.07.005

Arpita Poddar, Farah Ahmady, Prashanth Prithviraj, Rodney B Luwor, Ravi Shukla, Shakil Ahmed Polash, Haiyan Li, Suresh Ramakrishna, George Kannourakis, Aparna Jayachandran

引用次数: 0

Emerging drug delivery systems to alter tumor immunosuppressive microenvironment: Overcoming the challenges in immunotherapy for glioblastoma. 改变肿瘤免疫抑制微环境的新兴给药系统：克服胶质母细胞瘤免疫疗法的挑战。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-05-31 DOI: 10.1016/bs.pmbts.2024.04.006

P Soma Yasaswi, Harsh P Nijhawan, Bala Prabhakar, Shilpee Dutt, Khushwant S Yadav

{"title":"Emerging drug delivery systems to alter tumor immunosuppressive microenvironment: Overcoming the challenges in immunotherapy for glioblastoma.","authors":"P Soma Yasaswi, Harsh P Nijhawan, Bala Prabhakar, Shilpee Dutt, Khushwant S Yadav","doi":"10.1016/bs.pmbts.2024.04.006","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.04.006","url":null,"abstract":"Glioblastoma (GBM) is a highly proliferative, lethal cancer of the brain. The median survival at eight months is ca. 6.8%. Resistance towards the anti-glioblastoma drug temozolomide (TMZ), recurrence of cancer cells, blood-tumor brain barrier (BTBB), blood-brain barrier (BBB), and tumor immunosuppression are major challenges in treating GBM. Drug delivery systems employing TMZ and other anti-cancer drugs and combination therapy (temozolomide with immunotherapeutics) are under pre-clinical and clinical studies, respectively. Immunotherapeutics have emerged as a dominant mechanism to silence tumor development and dissemination. Paradoxically, immunotherapy has witnessed failure in treating GBM. This is due to the unique immunosuppressive microenvironment in GBM. Future immunotherapeutics with inherent tumor environment-modulating properties have to be identified. In this review, we discuss recent delivery systems and devices engineered to deliver immunotherapeutics with the ability to alter/silence tumor immune suppression.","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"209 ","pages":"165-182"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142506944","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 liposome-based delivery of RNA therapeutics for cancer treatment. 基于脂质体的 RNA 治疗药物在癌症治疗中的应用进展。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-01-05 DOI: 10.1016/bs.pmbts.2023.12.010

Mehdi Sanati, Amir R Afshari, Seyed Sajad Ahmadi, Prashant Kesharwani, Amirhossein Sahebkar

{"title":"Advances in liposome-based delivery of RNA therapeutics for cancer treatment.","authors":"Mehdi Sanati, Amir R Afshari, Seyed Sajad Ahmadi, Prashant Kesharwani, Amirhossein Sahebkar","doi":"10.1016/bs.pmbts.2023.12.010","DOIUrl":"10.1016/bs.pmbts.2023.12.010","url":null,"abstract":"Liposomal drug delivery systems stand as versatile therapeutic platforms for precisely targeting related elements in cancerous tissues owing to their intrinsic passive and acquired active targeting capabilities and exceptional compatibility with physiologic environments. When the capacity of liposomes as nanocarriers is combined with the revolutionary potential of RNA therapies in affecting undruggable targets, the outcome would be promising drug candidates as game-changers in the cancer treatment arena. However, optimizing liposome composition, physicochemical properties, and surface chemistry is paramount to maximizing their pharmacokinetic and pharmacodynamic attributes. This review highlighted the potential of liposomes as nanovehicles for RNA therapeutics through a literature review and looked at the most recent preclinical and clinical advancements in utilizing liposomal RNA therapeutics for cancer management. Notably, the discovery of novel targets, advancements in liposome engineering, and organizing well-planned clinical trials would help uncover the incredible potential of these nanotherapeutics in cancer patients.","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"204 ","pages":"177-218"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140065802","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

RNA interference-based therapies for atherosclerosis: Recent advances and future prospects. 基于 RNA 干扰的动脉粥样硬化疗法：最新进展与未来展望。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2023-12-29 DOI: 10.1016/bs.pmbts.2023.12.009

Negin Parsamanesh, Mohadeseh Poudineh, Haleh Siami, Alexandra E Butler, Wael Almahmeed, Amirhossein Sahebkar

引用次数: 0

RNA therapeutics for kidney injury. 治疗肾损伤的 RNA 疗法。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-01-24 DOI: 10.1016/bs.pmbts.2023.12.007

Pouya Goleij, Pantea Majma Sanaye, Aryan Rezaee, Mohammad Amin Khazeei Tabari, Reza Arefnezhad, Hossein Motedayyen

引用次数: 0

An overview and potential of CRISPR-Cas systems for genome editing. 概述用于基因组编辑的 CRISPR-Cas 系统及其潜力。

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.2024.07.009

Karan Murjani, Renu Tripathi, Vijai Singh

{"title":"An overview and potential of CRISPR-Cas systems for genome editing.","authors":"Karan Murjani, Renu Tripathi, Vijai Singh","doi":"10.1016/bs.pmbts.2024.07.009","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.07.009","url":null,"abstract":"Genome editing involves altering of the DNA in organisms including bacteria, plants, and animals using molecular scissors that helps in treatment and diagnosis of various diseases. Genome editing technology is exponentially growing and have been developed for enabling precise genomic alterations and the addition, removal, and correction of genes. These modifications begin with the creation of double-stranded breaks (DSBs) that is generated by nucleases and can be joined through homology-directed repair (HDR) or non-homologous end-joining (NHEJ). NHEJ is quick but increases mutation chances due to deletions and insertions of nucleotides at the break site, while HDR uses homologous templates for precise repair and targeted DNA specific to the gene or sequence. Other methods such as zinc-finger protein is a transcription factor that binds with DNA and binds specific to that sequence, which uniquely recognise 3-base pairs of DNA. TALENs consists of two domains: TALE domain, a transcription activator and FokI that is a restriction endonuclease that cuts the DNA at specific sites. CRISPR-Cas systems are clustered regularly interspersed short palindromic repeats present in various bacterial species. These sequences activate RNA-guided DNA cleavage, aiding in the development of an adaptive immune defence against foreign DNA. CRISPR-Cas9 is widely used for genome editing, regulation, diagnostic and many.","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"208 ","pages":"1-17"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142294149","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

Current progress in CRISPR-Cas systems for cancer. CRISPR-Cas 癌症治疗系统的最新进展。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-08-15 DOI: 10.1016/bs.pmbts.2024.07.007

Hunaiza Fatima, Hajra Ali Raja, Rabia Amir, Alvina Gul, Mustafeez Mujtaba Babar, Jayakumar Rajadas

{"title":"Current progress in CRISPR-Cas systems for cancer.","authors":"Hunaiza Fatima, Hajra Ali Raja, Rabia Amir, Alvina Gul, Mustafeez Mujtaba Babar, Jayakumar Rajadas","doi":"10.1016/bs.pmbts.2024.07.007","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.07.007","url":null,"abstract":"Cancer has been a primary contributor to morbidity and mortality worldwide. With an increasing trend of incidence and prevalence of cancer, progress has also been made in its treatment, starting from radiation and chemotherapy to immunotherapy and gene therapy. CRISPR-Cas technique, a promising gene editing tool, has been employed in cancer research for novel treatment regimens, identification of therapeutic targets, and unraveling the genetic mechanisms behind oncogenesis. CRISPR-based genome editing helped in identifying the roles of specific genetic factors linked to treatment resistance, metastasis, and cancer development. CRISPR allows the discovery of genes and treatment options through specifically interrupting tumor activators or activating tumor suppressor genes in cancer cells. Advancements in CRISPR technology, especially the use of immune cells like chimeric antigen receptor (CAR) T cells, has the potential to revolutionize personalized cancer treatment by precisely targeting and killing cancer cells. Furthermore, reactivating tumor suppressor genes makes cancer cells more susceptible to chemotherapy or immunotherapy. CRISPR-mediated genome editing can, hence, help to overcome resistance to traditional cancer treatments. The current manuscript covers that how is the CRISPR technology propelling revolutionary development in the field of cancer research, providing advance perspectives on the molecular causes of the disease and creating new lines for the development of more precise and potent cancer therapies.","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"208 ","pages":"211-229"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142294152","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

Application of RNA-based therapeutics in glioma: A review. 基于 RNA 的疗法在胶质瘤中的应用：综述。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2023-12-29 DOI: 10.1016/bs.pmbts.2023.12.001

Mehdi Sanati, Amir R Afshari, Seyed Sajad Ahmadi, Tannaz Jamialahmadi, Amirhossein Sahebkar

引用次数: 0

Drug repurposing: A multi targetted approach to treat cardiac disease from existing classical drugs to modern drug discovery. 药物再利用：从现有经典药物到现代药物发现，治疗心脏病的多靶点方法。

3区生物学

Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-05-14 DOI: 10.1016/bs.pmbts.2024.02.001

Shyam Tripathi, Kusum Rani, V Samuel Raj, Rashmi K Ambasta

{"title":"Drug repurposing: A multi targetted approach to treat cardiac disease from existing classical drugs to modern drug discovery.","authors":"Shyam Tripathi, Kusum Rani, V Samuel Raj, Rashmi K Ambasta","doi":"10.1016/bs.pmbts.2024.02.001","DOIUrl":"https://doi.org/10.1016/bs.pmbts.2024.02.001","url":null,"abstract":"Cardiovascular diseases (CVDs) are characterized by abnormalities in the heart, blood vessels, and blood flow. CVDs comprise a diverse set of health issues. There are several types of CVDs like stroke, endothelial dysfunction, thrombosis, atherosclerosis, plaque instability and heart failure. Identification of a new drug for heart disease takes longer duration and its safety efficacy test takes even longer duration of research and approval. This chapter explores drug repurposing, nano-therapy, and plant-based treatments for managing CVDs from existing drugs which saves time and safety issues with testing new drugs. Existing drugs like statins, ACE inhibitor, warfarin, beta blockers, aspirin and metformin have been found to be useful in treating cardiac disease. For better drug delivery, nano therapy is opening new avenues for cardiac research by targeting interleukin (IL), TNF and other proteins by proteome interactome analysis. Nanoparticles enable precise delivery to atherosclerotic plaques, inflammation areas, and damaged cardiac tissues. Advancements in nano therapeutic agents, such as drug-eluting stents and drug-loaded nanoparticles are transforming CVDs management. Plant-based treatments, containing phytochemicals from Botanical sources, have potential cardiovascular benefits. These phytochemicals can mitigate risk factors associated with CVDs. The integration of these strategies opens new avenues for personalized, effective, and minimally invasive cardiovascular care. Altogether, traditional drugs, phytochemicals along with nanoparticles can revolutionize the future cardiac health care by identifying their signaling pathway, mechanism and interactome analysis.","PeriodicalId":21157,"journal":{"name":"Progress in molecular biology and translational science","volume":"207 ","pages":"151-192"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141470526","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