Introduction: To date, the treatment option for tachyarrhythmia is classified into drug therapy, catheter ablation, and implantable device therapy. However, the efficacy of the antiarrhythmic drugs is limited. Although the indication of catheter ablation is expanding, several fatal tachyarrhythmias are still refractory to ablation. Implantable cardioverter-defibrillator increases survival, but it is not a curable treatment. Therefore, a novel therapy for tachyarrhythmias refractory to present treatments is desired. Gene therapy is being developed as a promising candidate for this purpose, and basic research and translational research have been accumulated in recent years.
Areas Covered: This paper reviews the current state of gene therapy for arrhythmias, including susceptible arrhythmias, the route of administration to the heart, and the type of vector to use. We also discuss the latest progress in the technology of gene delivery and genome editing.
Expert Opinion: Gene therapy is one of the most promising technologies for arrhythmia treatment. However, additional technological innovation to achieve safe, localized, homogeneous, and long-lasting gene transfer is required for its clinical application.
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http://dx.doi.org/10.1080/14779072.2022.2085686 | DOI Listing |
Exp Hematol Oncol
January 2025
Bone Marrow Transplantation Center of The First Affiliated Hospital Liangzhu Laboratory, Zhejiang University School of Medicine, No. 79 Qingchun Road, Hangzhou, Zhejiang, China.
Background: Sequential CD19 and CD22 chimeric antigen receptor (CAR)-T cell therapy offers a promising approach to antigen-loss relapse in relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL); however, research in adults remains limited.
Methods: This study aimed to evaluate the efficacy and safety of sequential CD19 and CD22 CAR-T cell therapy in adult patients with R/R B-ALL between November 2020 and November 2023 (ChiCTR2100053871). Key endpoints included the adverse event incidence, overall survival (OS), and leukemia-free survival (LFS).
Neurol Ther
January 2025
Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.
Hereditary transthyretin amyloidosis (ATTRv, v for variant) is a genetic disorder characterized by the deposition of misfolded transthyretin (TTR) protein in tissues, resulting in progressive dysfunction of multiple organs, including the nervous system, heart, kidneys, and gastrointestinal (GI) tract. Noninvasive serum biomarkers have become key tools for diagnosing and monitoring ATTRv. This review examines the role of available biomarkers for neurological, cardiac, renal, gastrointestinal, and multisystemic involvement in ATTRv.
View Article and Find Full Text PDFCNS Neurosci Ther
January 2025
Jiujiang Clinical Precision Medicine Research Center, Jiujiang, Jiangxi, China.
Background: Adenosine deaminase action on RNA 1 (ADAR1) can convert the adenosine in double-stranded RNA (dsRNA) molecules into inosine in a process known as A-to-I RNA editing. ADAR1 regulates gene expression output by interacting with RNA and other proteins; plays important roles in development, including growth; and is linked to innate immunity, tumors, and central nervous system (CNS) diseases.
Results: In recent years, the role of ADAR1 in tumors has been widely discussed, but its role in CNS diseases has not been reviewed.
Nat Med
January 2025
Leiden University Center for Infectious Diseases, Leiden University Medical Center, Leiden, The Netherlands.
Malaria vaccines consisting of metabolically active Plasmodium falciparum (Pf) sporozoites can offer improved protection compared with currently deployed subunit vaccines. In a previous study, we demonstrated the superior protective efficacy of a three-dose regimen of late-arresting genetically attenuated parasites administered by mosquito bite (GA2-MB) compared with early-arresting counterparts (GA1-MB) against a homologous controlled human malaria infection. Encouraged by these results, we explored the potency of a single GA2-MB immunization in a placebo-controlled randomized trial.
View Article and Find Full Text PDFAutosomal-recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) is an early-onset neurodegenerative disease caused by mutations in the SACS gene. The first two mutations were identified in French Canadian populations 20 years ago. The disease is now known as one of the most frequent recessive ataxias worldwide.
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