Advances in Lipid Nanoparticle‐Based Disease Treatment

ChemMedChem, Journal Year: 2025, Volume and Issue: unknown

Published: Feb. 17, 2025

Abstract Lipid nanoparticles (LNPs) have emerged as a transformative platform for the targeted delivery of therapeutic agents, revolutionizing treatment paradigms across spectrum diseases. Since inception liposomes in 1960s, lipid‐based nanotechnology has evolved to address limitations such poor bioavailability, off‐target effects, and instability, thereby enhancing efficacy safety drug administration. This review highlights latest advancements LNPs technology, focusing on their application cancer therapy, gene infectious disease management, glaucoma, other clinical areas. Recent studies underscore potential deliver messenger RNA (mRNA) small interfering (siRNA) precise genetic intervention, exemplified by breakthroughs interference CRISPR‐Cas9 genome editing. Additionally, been successfully employed ameliorate conditions, demonstrating versatility addressing both acute chronic disorders. However, challenges persist concerning large‐scale manufacturing, long‐term stability, comprehensive evaluations. Future research must focus optimizing formulations, exploring synergistic combinations with existing therapies, expanding scope treatable The integration into personalized medicine exploration applications diseases represent promising avenues further investigation. are poised play an increasingly central role development next‐generation therapeutics.

Language: Английский

COVID‐19 Vaccination in Patients With Systemic Lupus Erythematosus: Adverse Events and Rating Agreement of Flares Between Patients and Physicians

International Journal of Rheumatic Diseases, Journal Year: 2024, Volume and Issue: 27(12)

Published: Dec. 1, 2024

ABSTRACT Objectives To compare adverse events and flares among different doses types of COVID‐19 vaccines in patients with systemic lupus erythematosus (SLE). Methods All consecutive SLE a cohort, seen between March October 2022, were invited to join this retrospective study. Inclusion criteria aged ≥ 20 years had received at least one dose vaccine. Data regarding after vaccination, clinical disease activity within 30 days postvaccination reviewed. Results A total 201 524 vaccine doses, 201, 199, 124 1, 2, 3 respectively. The included inactivated virus vaccine, adenovirus‐vectored mRNA 183 (35%), 128 (24%), 213 (41%) Regardless the type occurred 50%–70% patients. Pain swelling injection site common local symptoms, whereas constitutional, neurological, musculoskeletal, mucocutaneous symptoms ones. majority these mild moderate. Patients reported they vaccination 5%–6%, while actual determined by physicians 8%–13% them, giving fair moderate rating agreement (Cohen's kappa: 0.21–0.44). There was no significant difference mean mSLEDAI‐2K pre‐ postvaccination. Conclusions Adverse common, regardless or vaccines, but only small proportion severe symptoms. Flares uncommon. as

Language: Английский

Effective targeting of CD19 positive primary B-ALL cells using CAR-NK cells generated with mRNA-LNPs

bioRxiv (Cold Spring Harbor Laboratory), Journal Year: 2024, Volume and Issue: unknown

Published: Nov. 21, 2024

Chimeric antigen receptor natural killer (CAR-NK) cell therapy is recognized as a promising modality for the treatment of hematologic malignancies, particularly B-cell malignancies. In this study, we developed ''off-the-shelf'' anti-CD19 CAR-NK cells using CAR mRNAs formulated in proprietary ionizable lipid nanoparticles (LNPs). The efficiency mRNA-LNP delivery into umbilical cord blood (UCB)-derived NK and primary T was evaluated an in-vitro setting, demonstrating superior cells. Further investigation showed probable role endocytic mechanism, macropinocytosis, efficient transfection with LNPs. Nevertheless, generated through platform exhibited significantly enhanced cytotoxicity against CD19+ target cells, such EGFP+Raji stable line malignant B derived from refractory/relapsed acute lymphoblastic leukemia (B-ALL) patients. These findings highlight promise advancing therapies

Language: Английский