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Annual review of medicine最新文献

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Adeno-Associated Virus Gene Therapy for Hemophilia. 腺相关病毒基因治疗血友病。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-043021-033013
Benjamin J Samelson-Jones, Lindsey A George
{"title":"Adeno-Associated Virus Gene Therapy for Hemophilia.","authors":"Benjamin J Samelson-Jones,&nbsp;Lindsey A George","doi":"10.1146/annurev-med-043021-033013","DOIUrl":"https://doi.org/10.1146/annurev-med-043021-033013","url":null,"abstract":"<p><p>In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy. This effort is soon to bear fruit with completed pivotal adeno-associated viral (AAV) vector gene addition trials reporting encouraging results and regulatory approval widely anticipated in the near future for the current generation of HA and HB AAV vectors. Here we review the clinical development of AAV gene therapy for HA and HB and examine outstanding questions that have recently emerged from AAV clinical trials for hemophilia and other monogenic disorders.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9892335/pdf/nihms-1864165.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9713785","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 19
Chronic Neuromuscular Respiratory Failure and Home Assisted Ventilation. 慢性神经肌肉呼吸衰竭和家用辅助通气。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-043021-013620
Hugo Carmona, Andrew D Graustein, Joshua O Benditt
{"title":"Chronic Neuromuscular Respiratory Failure and Home Assisted Ventilation.","authors":"Hugo Carmona,&nbsp;Andrew D Graustein,&nbsp;Joshua O Benditt","doi":"10.1146/annurev-med-043021-013620","DOIUrl":"https://doi.org/10.1146/annurev-med-043021-013620","url":null,"abstract":"<p><p>Chronic respiratory failure is a common, important complication of many types of neuromuscular and chest wall disorders. While the pathophysiology of each disease may be different, these disorders can variably affect all muscles involved in breathing, including inspiratory, expiratory, and bulbar muscles, ultimately leading to chronic respiratory failure and hypoventilation. The use of home assisted ventilation through noninvasive interfaces aims to improve the symptoms of hypoventilation, improve sleep quality, and, when possible, improve mortality. An increasing variety of interfaces has allowed for improved comfort and compliance. In a minority of scenarios, noninvasive ventilation is either not appropriate or no longer effective due to disease progression, and a transition to tracheal ventilation should be considered.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9228094","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Club Cell Secretory Protein in Lung Disease: Emerging Concepts and Potential Therapeutics. 肺部疾病中的俱乐部细胞分泌蛋白：新概念和潜在疗法》（Emerging Concepts and Potential Therapeutics）。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 Epub Date: 2022-11-30 DOI: 10.1146/annurev-med-042921-123443
Tereza Martinu, Jamie L Todd, Andrew E Gelman, Stefano Guerra, Scott M Palmer
{"title":"Club Cell Secretory Protein in Lung Disease: Emerging Concepts and Potential Therapeutics.","authors":"Tereza Martinu, Jamie L Todd, Andrew E Gelman, Stefano Guerra, Scott M Palmer","doi":"10.1146/annurev-med-042921-123443","DOIUrl":"10.1146/annurev-med-042921-123443","url":null,"abstract":"<p><p>Club cell secretory protein (CCSP), also known as secretoglobin 1A1 (gene name <i>SCGB1A1</i>), is one of the most abundant proteins in the lung, primarily produced by club cells of the distal airway epithelium. At baseline, CCSP is found in large concentrations in lung fluid specimens and can also be detected in the blood and urine. Obstructive lung diseases are generally associated with reduced CCSP levels, thought to be due to decreased CCSP production or club cell depletion. Conversely, several restrictive lung diseases have been found to have increased CCSP levels both in the lung and in the circulation, likely related to club cell dysregulation as well as increasedlung permeability. Recent studies demonstrate multiple mechanisms by which CCSP dampens acute and chronic lung inflammation. Given these anti-inflammatory effects, CCSP represents a novel potential therapeutic modality in lung disease.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10472444/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10141068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Maternal Mortality in the United States: Trends and Opportunities for Prevention. 美国孕产妇死亡率:趋势和预防机会。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-042921-123851
Siwen Wang, Kathryn M Rexrode, Andrea A Florio, Janet W Rich-Edwards, Jorge E Chavarro
{"title":"Maternal Mortality in the United States: Trends and Opportunities for Prevention.","authors":"Siwen Wang,&nbsp;Kathryn M Rexrode,&nbsp;Andrea A Florio,&nbsp;Janet W Rich-Edwards,&nbsp;Jorge E Chavarro","doi":"10.1146/annurev-med-042921-123851","DOIUrl":"https://doi.org/10.1146/annurev-med-042921-123851","url":null,"abstract":"<p><p>Maternal mortality is unusually high in the United States compared to other wealthy nations and is characterized by major disparities in race/ethnicity, geography, and socioeconomic factors. Similar to other developed nations, the United States has seen a shift in the underlying causes of pregnancy-related death, with a relative increase in mortality resulting from diseases of the cardiovascular system and preexisting medical conditions. Improved continuity of care aimed at identifying reproductive-age women with preexisting conditions that may heighten the risk of maternal death, preconception management of risk factors for major adverse pregnancy outcomes, and primary care visits within the first year after delivery may offer opportunities to address gaps in medical care contributing to the unacceptable rates of maternal mortality in the United States.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10732006","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 8
New Frontiers in Obesity Treatment: GLP-1 and Nascent Nutrient-Stimulated Hormone-Based Therapeutics. 肥胖症治疗的新领域:GLP-1和新生的营养刺激激素疗法。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-043021-014919
Ania M Jastreboff, Robert F Kushner
{"title":"New Frontiers in Obesity Treatment: GLP-1 and Nascent Nutrient-Stimulated Hormone-Based Therapeutics.","authors":"Ania M Jastreboff,&nbsp;Robert F Kushner","doi":"10.1146/annurev-med-043021-014919","DOIUrl":"https://doi.org/10.1146/annurev-med-043021-014919","url":null,"abstract":"<p><p>Nearly half of Americans are projected to have obesity by 2030, underscoring the pressing need for effective treatments. Glucagon-like peptide 1 receptor agonists (GLP-1 RAs) represent the first agents in a rapidly evolving, highly promising landscape of nascent hormone-based obesity therapeutics. With the understanding of the neurobiology of obesity rapidly expanding, these emerging entero-endocrine and endo-pancreatic agents combined or coformulated with GLP-1 RAs herald a new era of targeted, mechanism-based treatment of obesity. This article reviews GLP-1 RAs in the treatment of obesity and previews the imminent future of nutrient-stimulated hormone-based anti-obesity therapeutics.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10732008","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 8
Genetics of Kidney Disease: The Unexpected Role of Rare Disorders. 肾脏疾病的遗传学：罕见疾病的意外作用。
IF 15.1 1区 医学
Annual review of medicine Pub Date : 2023-01-27 Epub Date: 2022-11-14 DOI: 10.1146/annurev-med-042921-101813
Mark D Elliott, Hila Milo Rasouly, Ali G Gharavi
{"title":"Genetics of Kidney Disease: The Unexpected Role of Rare Disorders.","authors":"Mark D Elliott, Hila Milo Rasouly, Ali G Gharavi","doi":"10.1146/annurev-med-042921-101813","DOIUrl":"10.1146/annurev-med-042921-101813","url":null,"abstract":"<p><p>Hundreds of different genetic causes of chronic kidney disease are now recognized, and while individually rare, taken together they are significant contributors to both adult and pediatric diseases. Traditional genetics approaches relied heavily on the identification of large families with multiple affected members and have been fundamental to the identification of genetic kidney diseases. With the increased utilization of massively parallel sequencing and improvements to genotype imputation, we can analyze rare variants in large cohorts of unrelated individuals, leading to personalized care for patients and significant research advancements. This review evaluates the contribution of rare disorders to patient care and the study of genetic kidney diseases and highlights key advancements that utilize new techniques to improve our ability to identify new gene-disease associations.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":15.1,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10144255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cystic Fibrosis Modulator Therapies. 囊性纤维化调节剂疗法。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-042921-021447
Shijing Jia, Jennifer L Taylor-Cousar
{"title":"Cystic Fibrosis Modulator Therapies.","authors":"Shijing Jia,&nbsp;Jennifer L Taylor-Cousar","doi":"10.1146/annurev-med-042921-021447","DOIUrl":"https://doi.org/10.1146/annurev-med-042921-021447","url":null,"abstract":"<p><p>Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction syndrome, liver dysfunction, and other disorders. Traditional therapies focused on the treatment or prevention of damage to each organ system with incremental modalities such as nebulized medications for the lungs, insulin for diabetes, and supplementation with pancreatic enzymes. However, the advent of highly effective modulator therapies that target specific cystic fibrosis transmembrane conductance regulator protein malformations resulting from individual genetic mutations has transformed the lives and prognosis for persons with CF.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10663302","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 10
Post-COVID-19 Condition. Post-COVID-19条件。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-043021-030635
Ani Nalbandian, Amar D Desai, Elaine Y Wan
{"title":"Post-COVID-19 Condition.","authors":"Ani Nalbandian,&nbsp;Amar D Desai,&nbsp;Elaine Y Wan","doi":"10.1146/annurev-med-043021-030635","DOIUrl":"https://doi.org/10.1146/annurev-med-043021-030635","url":null,"abstract":"<p><p>An estimated 10-15% of those infected with SARS-CoV-2 may have post-COVID-19 condition. Common lingering signs and symptoms include shortness of breath, fatigue, high heart rate, and memory and cognitive dysfunction even several months after infection, often impacting survivors' quality of life. The prevalence and duration of individual symptoms remain difficult to ascertain due to the lack of standardized research methods across various studies and limited patient follow-up in clinical studies. Nonetheless, data indicate post-COVID-19 condition may occur independent of acuity of initial infection, hospitalization status, age, or pre-existing comorbidities. Risk factors may include female sex and underlying respiratory or psychiatric disease. Supportive therapies to mitigate symptoms remain the mainstay of treatment. Reassuringly, most patients experience a reduction in symptoms by 1 year. The use of a universal case definition and shared research methods will allow for further clarity regarding the pervasiveness of this entity and its long-term health consequences.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10671591","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 62
Diverse Approaches to Gene Therapy of Sickle Cell Disease. 镰状细胞病基因治疗的多种方法。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 Epub Date: 2022-09-06 DOI: 10.1146/annurev-med-042921-021707
Shanna L White, Kevyn Hart, Donald B Kohn
{"title":"Diverse Approaches to Gene Therapy of Sickle Cell Disease.","authors":"Shanna L White, Kevyn Hart, Donald B Kohn","doi":"10.1146/annurev-med-042921-021707","DOIUrl":"10.1146/annurev-med-042921-021707","url":null,"abstract":"<p><p>Sickle cell disease (SCD) results from a single base pair change in the sixth codon of the β-globin chain of hemoglobin, which promotes aggregation of deoxyhemoglobin, increasing rigidity of red blood cells and causing vaso-occlusive and hemolytic complications. Allogeneic transplant of hematopoietic stem cells (HSCs) can eliminate SCD manifestations but is limited by absence of well-matched donors and immune complications. Gene therapy with transplantation of autologous HSCs that are gene-modified may provide similar benefits without the immune complications. Much progress has been made, and patients are realizing significant clinical improvements in multiple trials using different approaches with lentiviral vector-mediated gene addition to inhibit hemoglobin aggregation. Gene editing approaches are under development to provide additional therapeutic opportunities. Gene therapy for SCD has advanced from an attractive concept to clinical reality.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11001465/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10672107","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
SGLT2 Inhibitors: The Sweet Success for Kidneys. SGLT2抑制剂:肾脏的甜蜜成功。
IF 10.5 1区 医学
Annual review of medicine Pub Date : 2023-01-27 DOI: 10.1146/annurev-med-042921-102135
Atit Dharia, Abid Khan, Vikas S Sridhar, David Z I Cherney
{"title":"SGLT2 Inhibitors: The Sweet Success for Kidneys.","authors":"Atit Dharia,&nbsp;Abid Khan,&nbsp;Vikas S Sridhar,&nbsp;David Z I Cherney","doi":"10.1146/annurev-med-042921-102135","DOIUrl":"https://doi.org/10.1146/annurev-med-042921-102135","url":null,"abstract":"<p><p>Sodium-glucose cotransporter-2 inhibitors (SGLT2 inhibitors) were originally developed as antidiabetic agents, with cardiovascular (CV) outcome trials demonstrating improved CV outcomes in patients with type 2 diabetes mellitus (T2D). Secondary analyses of CV outcome trials and later dedicated kidney outcome trials consistently reported improved kidney-related outcomes independent of T2D status and across a range of kidney function and albuminuria. Importantly, SGLT2 inhibitors are generally safe and well tolerated, with clinical trials and real-world analyses demonstrating a decrease in the risk of acute kidney injury. The kidney protective effects of SGLT2 inhibitors generally extend across different members of the class, possibly on the basis of hemodynamic, metabolic, anti-inflammatory, and antifibrotic mechanisms. In this review, we summarize the effects of SGLT2 inhibitors on kidney outcomes in diverse patient populations.</p>","PeriodicalId":8056,"journal":{"name":"Annual review of medicine","volume":null,"pages":null},"PeriodicalIF":10.5,"publicationDate":"2023-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10732007","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 4
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