{"title":"Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives.","authors":"Chao Huang, Qing Li, Jinsong Li","doi":"10.1515/mr-2022-0029","DOIUrl":"10.1515/mr-2022-0029","url":null,"abstract":"<p><p>Advancements in genome editing enable permanent changes of DNA sequences in a site-specific manner, providing promising approaches for treating human genetic disorders caused by gene mutations. Recently, genome editing has been applied and achieved significant progress in treating inherited genetic disorders that remain incurable by conventional therapy. Here, we present a review of various programmable genome editing systems with their principles, advantages, and limitations. We introduce their recent applications for treating inherited diseases in the clinic, including sickle cell disease (SCD), β-thalassemia, Leber congenital amaurosis (LCA), heterozygous familial hypercholesterolemia (HeFH), etc. We also discuss the paradigm of <i>ex vivo</i> and <i>in vivo</i> editing and highlight the promise of somatic editing and the challenge of germline editing. Finally, we propose future directions in delivery, cutting, and repairing to improve the scope of clinical applications.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 5","pages":"471-500"},"PeriodicalIF":0.0,"publicationDate":"2022-11-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b4/9e/mr-2-5-mr-2022-0029.PMC10388762.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10657919","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Developmental origins of adult diseases.","authors":"Jiaying Mo, Xuanqi Liu, Yutong Huang, Renke He, Yu Zhang, Hefeng Huang","doi":"10.1515/mr-2022-0027","DOIUrl":"10.1515/mr-2022-0027","url":null,"abstract":"<p><p>The occurrence and mechanisms of developmental adult diseases have gradually attracted attention in recent years. Exposure of gametes and embryos to adverse environments, especially during plastic development, can alter the expression of certain tissue-specific genes, leading to increased susceptibility to certain diseases in adulthood, such as diabetes, cardiovascular disease, neuropsychiatric, and reproductive system diseases, etc. The occurrence of chronic disease in adulthood is partly due to genetic factors, and the remaining risk is partly due to environmental-dependent epigenetic information alteration, including DNA methylation, histone modifications, and noncoding RNAs. Changes in this epigenetic information potentially damage our health, which has also been supported by numerous epidemiological and animal studies in recent years. Environmental factors functionally affect embryo development through epimutation, transmitting diseases to offspring and even later generations. This review mainly elaborated on the concept of developmental origins of adult diseases, and revealed the epigenetic mechanisms underlying these events, discussed the theoretical basis for the prevention and treatment of related diseases.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 5","pages":"450-470"},"PeriodicalIF":0.0,"publicationDate":"2022-11-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/80/62/mr-2-5-mr-2022-0027.PMC10388800.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10658303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Organoid research on human early development and beyond.","authors":"Lu Wen, Fuchou Tang","doi":"10.1515/mr-2022-0028","DOIUrl":"10.1515/mr-2022-0028","url":null,"abstract":"<p><p>The organoid field has been developing rapidly during the last decade. Organoids for human pre-, peri- and post-implantation development have opened an avenue to study these biological processes <i>in vitro</i>, which have been hampered by lack of accessible research models for long term. The technologies of four fields, single cell omics sequencing, genome editing and lineage tracing, microfluidics and tissue engineering, have fueled the rapid development of the organoid field. In this review, we will discuss the organoid research on human early development as well as future directions of the organoid field combining with other powerful technologies.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 5","pages":"512-523"},"PeriodicalIF":0.0,"publicationDate":"2022-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/53/26/mr-2-5-mr-2022-0028.PMC10471100.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10657922","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Ultrasound contrast agents from microbubbles to biogenic gas vesicles.","authors":"Wenlong Zeng, Xiuli Yue, Zhifei Dai","doi":"10.1515/mr-2022-0020","DOIUrl":"10.1515/mr-2022-0020","url":null,"abstract":"<p><p>Microbubbles have been the earliest and most widely used ultrasound contrast agents by virtue of their unique features: such as non-toxicity, intravenous injectability, ability to cross the pulmonary capillary bed, and significant enhancement of echo signals for the duration of the examination, resulting in essential preclinical and clinical applications. The use of microbubbles functionalized with targeting ligands to bind to specific targets in the bloodstream has further enabled ultrasound molecular imaging. Nevertheless, it is very challenging to utilize targeted microbubbles for molecular imaging of extravascular targets due to their size. A series of acoustic nanomaterials have been developed for breaking free from this constraint. Especially, biogenic gas vesicles, gas-filled protein nanostructures from microorganisms, were engineered as the first biomolecular ultrasound contrast agents, opening the door for more direct visualization of cellular and molecular function by ultrasound imaging. The ordered protein shell structure and unique gas filling mechanism of biogenic gas vesicles endow them with excellent stability and attractive acoustic responses. What's more, their genetic encodability enables them to act as acoustic reporter genes. This article reviews the upgrading progresses of ultrasound contrast agents from microbubbles to biogenic gas vesicles, and the opportunities and challenges for the commercial and clinical translation of the nascent field of biomolecular ultrasound.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"3 1","pages":"31-48"},"PeriodicalIF":0.0,"publicationDate":"2022-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/78/00/mr-3-1-mr-2022-0020.PMC10471104.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10363192","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Trimethylamine-N-oxide is an important target for heart and brain diseases.","authors":"Shusi Ding, Jing Xue, Qi Zhang, Lemin Zheng","doi":"10.1515/mr-2022-0026","DOIUrl":"10.1515/mr-2022-0026","url":null,"abstract":"Trimethylamine-N-oxide (TMAO) is a metabolite produced","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 4","pages":"321-323"},"PeriodicalIF":0.0,"publicationDate":"2022-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/c3/cc/mr-2-4-mr-2022-0026.PMC10388736.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10311415","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The polarizable and reprogrammable identity of Kupffer cells in Nonalcoholic Steatohepatitis.","authors":"Tarik Zahr, Kevin Sun, Li Qiang","doi":"10.1515/mr-2022-0023","DOIUrl":"10.1515/mr-2022-0023","url":null,"abstract":"<p><p>Kupffer cells (KCs) are the resident macrophages of the liver with similar origins to myeloid-derived macrophages. Once differentiated, KCs exhibit distinct cellular machinery capable of longevity and self-renewal, making them a crucial player in promoting effective intrahepatic communication. However, this gets compromised in disease states like Nonalcoholic Steatohepatitis (NASH), where the loss of embryo-derived KCs (EmKCs) is observed. Despite this, other KC-like and KC-derived populations start to form and contribute to a variety of roles in NASH pathogenesis, often adopting a NASH-associated molecular signature. Here we offer a brief overview of recent reports describing KC polarization and reprogramming in the liver. We describe the complexities of KC cellular identity, their proposed ability to reprogram to fibroblast-like and endothelial-like cells, and the potential implications in NASH.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 4","pages":"324-327"},"PeriodicalIF":0.0,"publicationDate":"2022-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/13/c7/mr-2-4-mr-2022-0023.PMC10388795.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10311416","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Crosstalk between bone and other organs.","authors":"Wanqiong Yuan, Chunli Song","doi":"10.1515/mr-2022-0018","DOIUrl":"10.1515/mr-2022-0018","url":null,"abstract":"<p><p>Bone has long been considered as a silent organ that provides a reservoir of calcium and phosphorus, traditionally. Recently, further study of bone has revealed additional functions as an endocrine organ connecting systemic organs of the whole body. Communication between bone and other organs participates in most physiological and pathological events and is responsible for the maintenance of homeostasis. Here, we present an overview of the crosstalk between bone and other organs. Furthermore, we describe the factors mediating the crosstalk and review the mechanisms in the development of potential associated diseases. These connections shed new light on the pathogenesis of systemic diseases and provide novel potential targets for the treatment of systemic diseases.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 4","pages":"331-348"},"PeriodicalIF":0.0,"publicationDate":"2022-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ae/24/mr-2-4-mr-2022-0018.PMC10471111.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10311417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Proteomics research of SARS-CoV-2 and COVID-19 disease.","authors":"Nan Zhang, Siyuan Wang, Catherine C L Wong","doi":"10.1515/mr-2022-0016","DOIUrl":"10.1515/mr-2022-0016","url":null,"abstract":"<p><p>Currently, coronavirus disease 2019 (COVID-19) is still spreading in a global scale, exerting a massive health and socioeconomic crisis. Deep insights into the molecular functions of the viral proteins and the pathogenesis of this infectious disease are urgently needed. In this review, we comprehensively describe the proteome of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and summarize their protein interaction map with host cells. In the protein interaction network between the virus and the host, a total of 787 host prey proteins that appeared in at least two studies or were verified by co-immunoprecipitation experiments. Together with 29 viral proteins, a network of 1762 proximal interactions were observed. We also review the proteomics results of COVID-19 patients and proved that SARS-CoV-2 hijacked the host's translation system, post-translation modification system, and energy supply system via viral proteins, resulting in various immune disorders, multiple cardiomyopathies, and cholesterol metabolism diseases.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 4","pages":"427-445"},"PeriodicalIF":0.0,"publicationDate":"2022-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/57/66/mr-2-4-mr-2022-0016.PMC10388787.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10311418","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Hypothalamic GABAergic neurocircuitry in the regulation of energy homeostasis and sleep/wake control.","authors":"Hong Jiang","doi":"10.1515/mr-2022-0022","DOIUrl":"10.1515/mr-2022-0022","url":null,"abstract":"<p><p>Gamma-aminobutyric acid (GABAergic) neuron, as one of important cell types in synaptic transmission, has been widely involved in central nervous system (CNS) regulation of organismal physiologies including cognition, emotion, arousal and reward. However, upon their distribution in various brain regions, effects of GABAergic neurons in the brain are very diverse. In current report, we will present an overview of the role of GABAergic mediated inhibitory neurocircuitry in the hypothalamus, underlying mechanism of feeding and sleep homeostasis as well as the characteristics of latest transcriptome profile in order to call attention to the GABAergic system as potentially a promising pharmaceutical intervention or a deep brain stimulation target in eating and sleep disorders.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 5","pages":"531-540"},"PeriodicalIF":0.0,"publicationDate":"2022-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6e/4d/mr-2-5-mr-2022-0022.PMC10388747.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10675081","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Fibroblast growth factor 21 and dietary interventions: what we know and what we need to know next.","authors":"Tianru Jin","doi":"10.1515/mr-2022-0019","DOIUrl":"10.1515/mr-2022-0019","url":null,"abstract":"<p><p>Dietary interventions include the change of dietary styles, such as fasting and dietary or nutrient restrictions; or the addition of plant-derived compounds (such as polyphenols known as curcumin, resveratrol, or anthocyanin, or other nutraceuticals) into the diet. During the past a few decades, large number of studies have demonstrated therapeutic activities of these dietary interventions on metabolic and other diseases in human subjects or various animal models. Mechanisms underlying those versatile therapeutic activities, however, remain largely unclear. Interestingly, recent studies have shown that fibroblast growth factor 21 (FGF21), a liver-derived hormone or hepatokine, mediates metabolic beneficial effects of certain dietary polyphenols as well as protein restriction. Here I have briefly summarized functions of FGF21, highlighted related dietary interventions, and presented literature discussions on role of FGF21 in mediating function of dietary polyphenol intervention and protein restriction. This is followed by presenting my perspective view, with the involvement of gut microbiota. It is anticipated that further breakthroughs in this field in the near future will facilitate conceptual merge of classical medicine and modern medicine.</p>","PeriodicalId":74151,"journal":{"name":"Medical review (Berlin, Germany)","volume":"2 5","pages":"524-530"},"PeriodicalIF":0.0,"publicationDate":"2022-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/c9/49/mr-2-5-mr-2022-0019.PMC10388781.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10657921","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}