Results 41 to 50 of about 1,115 (145)
Journal of Hepatology, 2023 Acute hepatic porphyria (AHP) is caused by defects in hepatic heme biosynthesis, leading to disabling acute neurovisceral attacks and chronic symptoms. In ENVISION (NCT03338816), givosiran treatment for 6 months reduced attacks and other disease manifestations compared with placebo.
David J. Kuter +11 more
openaire +2 more sources
RNA Biology, 2022 Nucleic acid-based therapeutics that control gene expression have been steadily progressing towards achieving their full clinical potential throughout the last few decades.
Sumit Gangopadhyay, Kiran R Gore
doaj +1 more source
Brazilian registry of patients with porphyria: REBRAPPO study
Orphanet Journal of Rare Diseases, 2023 Background Porphyrias are a rare group of disease due to inherited defects of heme synthesis with important systemic manifestations and great burden of disease for patients and families due to the exceptional course of disease with disabling chronic ...
Paulo Victor Sgobbi Souza +13 more
doaj +1 more source
Design of Nanocarriers for Kidney Targeted Delivery of Nucleic Acid Therapeutics
Macromolecular Bioscience, Volume 26, Issue 7, July 2026.Nucleic acid therapeutics have been investigated to expand their applications to renal genetic disorders. This review summarizes key considerations in the design and fabrication of nanocarriers for the systemic delivery of nucleic acid therapeutics to the kidneys.
Jun Hyuk Lee +3 more
wiley +1 more source
Pain in acute hepatic porphyrias: Updates on pathophysiology and management
Frontiers in Neurology, 2022 Acute hepatic porphyrias (AHPs) typically present with recurrent acute attacks of severe abdominal pain and acute autonomic dysfunction. While chronic symptoms were historically overlooked in the literature, recent studies have reported increased ...
Mohamed Kazamel +2 more
doaj +1 more source
Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives
MedComm, Volume 7, Issue 7, July 2026.Schematic illustration of four emerging CRISPR–Cas delivery platforms defined by distinct design principles and structural features: virus‐mimicking nanosystems (e.g., VLPs), cell‐derived extracellular vesicles, cell‐penetrating peptides, and stimuli‐responsive scaffolds. These platforms enable spatiotemporally controlled delivery of RNPs, mRNA, or DNA
Meijia Yang +9 more
wiley +1 more source
Saúde & Tecnologia, 2022 Oligonucleotídeos sintéticos, como os small interfering RNAs (siRNAs), providenciam uma forma simples e eficiente de modular a expressão de qualquer gene.
Bruno M. D. C. Godinho +1 more
doaj +1 more source
Optimizing Oligonucleotide Therapeutics: A Model‐Informed Drug Development Perspective
Clinical and Translational Science, Volume 19, Issue 5, May 2026.ABSTRACT Oligonucleotide therapies have emerged as a powerful therapeutic class, providing novel solutions for diverse diseases through antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers that specifically target and modulate gene expression or protein function. However, oligonucleotide development faces distinct challenges,
Ye Yuan +7 more
wiley +1 more source
Advancing the Landscape of RNAi Nanotherapeutics for Ischemic Heart Disease
Advanced Materials, Volume 38, Issue 17, 20 March 2026.RNA interference (RNAi) nanomedicine revolutionizes treatment regimens for ischemic heart diseases by enabling tailored, sequence‐anchored gene regulation. This review highlights the recent advances in nanotechnology‐driven RNAi therapeutics for myocardial ischemia and discusses the key design principles that govern efficient delivery, providing ...
Han Gao, Da Pan, Hélder A. Santos
wiley +1 more source
RNA‐Based Therapies for Inherited Metabolic Disorders
Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri +5 more
wiley +1 more source