Canadian Journal of Cardiology最新文献_第2页

A Primer on Cardiovascular Gene Therapy for the Practitioner: What Clinicians Need to Know 对从业者心血管基因治疗的入门：什么临床医生需要知道。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2025-12-01 DOI: 10.1016/j.cjca.2025.11.034

Cindy S. Argueta Portillo BS , Andrew P. Landstrom MD, PhD

引用次数: 0

Gene Replacement Therapy in Patients With Cardiac Disease: Challenges in Trial Design and Management of Adverse Events 心脏疾病的基因替代疗法：试验设计和不良事件管理的挑战。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2025-08-26 DOI: 10.1016/j.cjca.2025.08.340

Niccolò Maurizi MD , Kimberly N. Hong MD , Elizabeth Silver MD , Umang Patel BS , Eric D. Adler MD

{"title":"Gene Replacement Therapy in Patients With Cardiac Disease: Challenges in Trial Design and Management of Adverse Events","authors":"Niccolò Maurizi MD , Kimberly N. Hong MD , Elizabeth Silver MD , Umang Patel BS , Eric D. Adler MD","doi":"10.1016/j.cjca.2025.08.340","DOIUrl":"10.1016/j.cjca.2025.08.340","url":null,"abstract":"<div><div>Gene replacement therapy has emerged as a promising strategy to address the underlying molecular defects in inherited and acquired cardiomyopathies, shifting treatment from symptom palliation to potentially disease-modifying interventions. Most clinical programs use adeno-associated viral vectors to deliver functional DNAs, demonstrating safety, durable myocardial transduction, and early improvements in biomarkers or imaging end points. However, the rarity and heterogeneity of target populations constrain trial size and duration, making traditional morbidity and mortality outcomes infeasible. Central to overcoming these challenges has been the concurrent establishment of rigourous natural history cohorts. They serve as external controls, allowing for the capture of exact disease trajectories to define the optimal effective therapeutic windows. Natural history studies are critical to identifying clinically meaningful surrogate end points, ranging from circulating biomarkers and quantitative imaging measures to composite functional ranks that integrate exercise capacity with patient-reported symptoms. Collaborating with regulatory authorities to identify composite outcomes that combine surrogate outcomes predictive of morbidity and mortality with innovative patient-reported outcomes, the obstacles of statistical power and hard outcomes can be overcome. Last, a comprehensive understanding of the immune response to viral capsids, together with optimized and validated immunosuppressive regimens, is much needed to deliver durable, disease-modifying therapies to patients with genetic cardiac diseases. Continued collaboration among investigators, regulators, and patient communities, including rigourous natural history study design, surrogate qualification, and innovative trial frameworks, will be essential to realize the full potential of gene replacement therapies in cardiology.</div></div>","PeriodicalId":9555,"journal":{"name":"Canadian Journal of Cardiology","volume":"42 5","pages":"Pages 933-948"},"PeriodicalIF":5.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144943869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

New Approaches to Cardiac Gene Therapy Delivery 心脏基因治疗递送的新方法。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2026-02-27 DOI: 10.1016/j.cjca.2026.02.031

Samta Veera MS, Ni-Hsuan Lin BS, Spyros A. Mavropoulos MD, PhD, Kiyotake Ishikawa MD, PhD

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Call for Original Papers: Focus Issue on “New Insights into Tricuspid Valve Disease Management” to be published in January 2027 论文征集：重点议题为“三尖瓣疾病管理的新见解”，将于2027年1月发表

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2026-05-08 DOI: 10.1016/S0828-282X(26)00311-9

引用次数: 0

Emerging Gene Therapies for Inherited Arrhythmia Syndromes 遗传性心律失常综合征的新基因疗法。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2026-01-12 DOI: 10.1016/j.cjca.2026.01.003

Luca Grisorio MD , Jason D. Roberts MD, MAS

{"title":"Emerging Gene Therapies for Inherited Arrhythmia Syndromes","authors":"Luca Grisorio MD , Jason D. Roberts MD, MAS","doi":"10.1016/j.cjca.2026.01.003","DOIUrl":"10.1016/j.cjca.2026.01.003","url":null,"abstract":"<div><div>Inherited arrhythmia syndromes predispose to malignant arrhythmic events, including cardiac arrest and sudden cardiac death, which can be particularly catastrophic in young and otherwise healthy individuals. Despite considerable progress in clinical management in recent decades, the therapeutic options for many conditions remain limited and often expose patients to significant side effects that impair quality of life. There is therefore a pressing need for more effective and better-tolerated therapies. Owing to the predominantly monogenic nature of these diseases, and in light of the remarkable advances in molecular biology over the past two decades, gene therapies offer unprecedented potential by directly targeting the molecular determinants of arrhythmogenesis. In this review, we discuss advances in nucleic acid–based therapies for inherited cardiac arrhythmias, highlighting preclinical successes—some already progressing to first-in-class clinical trials—as well as the existing limitations. In light of accumulating preclinical evidence, we also discuss the impact of heterogeneous cardiac transduction/transfection by gene therapy vectors for arrhythmia treatment, a critical yet underexplored concept that carries theoretical concerns for pro-arrhythmia, but may also offer therapeutic opportunities. Finally, we briefly review future directions for the field, including the development of nucleic acid–based therapeutics for more prevalent arrhythmic disorders, such as post–myocardial infarction ventricular tachycardia and atrial fibrillation.</div></div>","PeriodicalId":9555,"journal":{"name":"Canadian Journal of Cardiology","volume":"42 5","pages":"Pages 982-1003"},"PeriodicalIF":5.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145984516","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Contemporary Controversies Around Medical Ethics of Gene Therapy for Cardiovascular Disease 当代心血管疾病基因治疗的医学伦理争议。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2025-12-13 DOI: 10.1016/j.cjca.2025.12.007

Antonio Greco MD, PhD, Luigi Cutore MD, Davide Capodanno MD, PhD

引用次数: 0

In Memoriam: Glen F. Tibbits, PhD 纪念：格伦·f·蒂比茨博士。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2026-03-06 DOI: 10.1016/j.cjca.2026.03.002

Saif F. Dababneh BSc , Tom W. Claydon PhD , Sanam Shafaattalab PhD , Maksymillian Prondzynski PhD , Haruyo Kashihara BSc , Leif Hove-Madsen PhD , Shubhayan Sanatani MD

引用次数: 0

Nucleic Acid–Based Therapies — The Next Frontier in Treating Dyslipidemias 基于核酸的疗法——治疗血脂异常的下一个前沿。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2026-01-23 DOI: 10.1016/j.cjca.2026.01.026

Robert A. Hegele MD, FRCPC

{"title":"Nucleic Acid–Based Therapies — The Next Frontier in Treating Dyslipidemias","authors":"Robert A. Hegele MD, FRCPC","doi":"10.1016/j.cjca.2026.01.026","DOIUrl":"10.1016/j.cjca.2026.01.026","url":null,"abstract":"<div><div>Genetic determinants play a central role in the development of dyslipidemias, which are major contributors to atherosclerotic cardiovascular disease (ASCVD), aortic valve disease, and acute pancreatitis. With conventional lipid-lowering therapies, such as statins, many patients with genetic dyslipidemias remain inadequately controlled while other patients are unable to tolerate them, necessitating ongoing research and development of innovative therapies. Several therapeutic lipid targets—including proprotein convertase subtilisin/kexin type 9 (PCSK9), lipoprotein(a) [Lp(a)], apolipoprotein (apo) C-III (APOC3), and angiopoietin-like protein 3 (ANGPTL3)—have been identified and validated through human genetic and epidemiologic studies. Emerging ribonucleic acid (RNA) targeting and gene-editing therapies now offer the potential for durable correction of these metabolic disturbances. Small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs) targeting PCSK9, Lp(a), apo C-III and ANGPTL3 have shown marked efficacy in lowering atherogenic lipoproteins and triglycerides, while DNA base-editing approaches including clustered regularly interspaced short palindromic repeats (CRISPR), CRISPR associated protein 9 (Cas9) and related base-editing techniques, aim to provide long-term or even permanent gene silencing to control atherogenic lipids. Together, these innovations mark a paradigm shift toward precision, gene-based lipid management, expanding therapeutic options, improving clinical outcomes, and addressing unmet medical needs in patients with severe or refractory dyslipidemias or in those unable to tolerate standard therapies.</div></div>","PeriodicalId":9555,"journal":{"name":"Canadian Journal of Cardiology","volume":"42 5","pages":"Pages 1004-1017"},"PeriodicalIF":5.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146046188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Safeguards or Roadblocks? Overhauling Research Ethics in Canada 保护措施还是路障？改革加拿大的研究伦理。

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2025-12-20 DOI: 10.1016/j.cjca.2025.12.031

Ashish H. Shah MD, MD-Research

引用次数: 0

Information for Readers 读者资讯

IF 5.3 2区医学

Canadian Journal of Cardiology Pub Date : 2026-05-01 Epub Date: 2026-05-08 DOI: 10.1016/S0828-282X(26)00306-5

引用次数: 0