Results 51 to 60 of about 1,115 (145)

Progress in RNA‐Targeted Therapeutics for Human Diseases

MedComm, Volume 7, Issue 2, February 2026.
RNA‐targeted therapies are revolutionizing molecular medicine by transitioning from a “protein‐centric” focus to an “RNA‐regulatory network” approach. Leveraging RNA's diverse roles in gene regulation, signaling, and epigenetic modifications, advanced platforms such as ASOs, siRNA, miRNA, mRNA, aptamers, shRNA, and CRISPR/Cas systems are enabling ...
Wangzheqi Zhang, Aimin Jiang, Bin‐Kui Jia, Yuming Jin, Yinghu Chen, Zhaoyu Li, Yan Liao, Haoling Zhang, Zhiheng Lin, Xiao Fang, Linhui Wang   +10 more
wiley   +1 more source

New cases of δ‐aminolevulinic acid dehydratase deficiency: Functional insights into gene variants using an innovative mouse liver model

Journal of Internal Medicine, Volume 299, Issue 1, Page 126-142, January 2026.
Abstract Background Dysfunction of δ‐aminolevulinic acid dehydratase (ALAD), the second enzyme involved in heme biosynthesis, leads to two pathologies: genetic and acquired. The genetic form is an ultrarare, severe childhood‐onset disease inherited in an autosomal recessive manner, whereas the acquired form usually affects adults due to enzyme ...
Elena Di Pierro, Isabel Solares, Daniel Jericó, Francisco J. Castelbón, Javier Tomás Solera, Antoni Riera‐Mestre, María Barreda‐Sánchez, Carlo Poci, Annamaria Nicolli, Francesco Urigo, Ana Sampedro, Rafael Enríquez de Salamanca, Matteo Marcacci, Matías A. Ávila, Pauline Harper, Marta G. Fanlo‐Maresma, Encarna Guillén‐Navarro, Giovanna Graziadei, Andrea Wenzel, Bodo B. Beck, Paolo Ventura, Montserrat Morales‐Conejo, Antonio Fontanellas   +22 more
wiley   +1 more source

The Immune Microenvironment in Liver Cancer: From Analysis to Targeting

Advanced Science, Volume 12, Issue 47, December 18, 2025.
This review explores how smart nanomedicines overcome the immunosuppressive tumor microenvironment in liver cancer. It details targeted delivery strategies and immune reprogramming mechanisms, including remodeling abnormal physiology, modulating metabolism, and inducing immunogenic cell death. The article highlights the paradigm shift toward multimodal
Jiaming Lan, Hao Li, Jian Xue, Yourong Duan, Jin Sun, Meng Niu   +5 more
wiley   +1 more source

NIR II‐Guided Photoactivatable Silencing Polyplex Boosts Cancer Immunotherapy

Exploration, Volume 5, Issue 5, October 2025.
Due to limitation of penetration therapy with first and second‐generation photodynamic therapy (PDT) photosensitizers, an NIR II‐guided photoactivatable complex was proposed via the self‐assembly of the photosensitive polymer PTSQ and the electrostatic adsorption of siPD‐L1 to enhance cancer immunotherapy.
Yuquan Zhang, Jie Wang, Tian Zhang, Dongsheng Tang, Haiyin Yang, Shuai Guo, Yuchuan Fan, Caixia Sun, Haihua Xiao, Yuanyu Huang, Yuhua Weng   +10 more
wiley   +1 more source

Amphipathic Octenyl‐Alanine Modified Peptides Mediate Effective siRNA Delivery

Journal of Peptide Science, Volume 31, Issue 10, October 2025.
This study demonstrates that novel octenyl‐alanine–modified hPep peptides efficiently encapsulate siRNA into well‐defined nanoparticles. Our findings highlight the potential of these hPep/siRNA nanoparticles to induce RNA interference, effectively silencing therapeutically important CD45 expression.
Tõnis Lehto, Marit Isakannu, Helena Sork, Annely Lorents, Safa Bazaz, Oscar P. B. Wiklander, Samir EL Andaloussi, Taavi Lehto   +7 more
wiley   +1 more source

Data Sharing Experience, Guidance, and Resources From the Rare Diseases Clinical Research Network (RDCRN)

Clinical and Translational Science, Volume 18, Issue 9, September 2025.
ABSTRACT The Rare Diseases Clinical Research Network (RDCRN) comprises research consortia and other partners focused on the study of rare diseases. Its goals include sharing de‐identified data with the scientific community and other stakeholders to advance rare disease research.
Elaine Schwendeman, Henry J. Kaminski, Adeline Vanderver, Michael Wagner, Eileen King, The RDCRN Data Use & Data Sharing Committee, Michael Benatar, Lori E. Crosby, Eileen King, Matthias Kretzler, Joanne Lumsden, Peter A. Merkel, Jill Morris, Susan Ruediger, Elaine Schwendeman, Tiina Urv, Adeline Vanderver, Maurizio Macaluso   +17 more
wiley   +1 more source

Adverse events of givosiran in the treatment of acute hepatic porphyria: a pharmacovigilance study using the FAERS and VigiAccess databases

Orphanet Journal of Rare Diseases
Background Acute hepatic porphyria is a rare metabolic disorder characterized by life-threatening acute attacks and chronic neurological symptoms. Givosiran is an RNA interference therapeutic approved by the FDA in 2019 to treat AHP by inhibiting hepatic
Yinpeng Xu, Fang Li, Li Huang, Mei Zhang, Pengqiang Du   +4 more
doaj   +1 more source

A Prospective, Blinded Study of Symptom Prevalence and Specificity of Porphyrin Precursors in Carriers of Acute Hepatic Porphyria

Liver International, Volume 45, Issue 7, July 2025.
ABSTRACT Background and Aims This study aimed to characterise symptoms and assess the prevalence of elevated urine porphyrin precursors in first‐degree relatives of acute hepatic porphyria (AHP) patients who have never experienced acute attacks and had no previous AHP genetic or biochemical testing.
Mohsen Merati, Nanditha Jayakumar, Yuvraaj Kapoor, Hetanshi Naik, Manisha Balwani, Karl E. Anderson, Herbert L. Bonkovsky, Robert J. Desnick, Brendan McGuire, John Phillips, D. Montgomery Bissell, Bruce Wang   +11 more
wiley   +1 more source

Acute intermittent porphyria: A case report with an unlisted HMBS gene variant (c.345–2A>C)

Brain Disorders
We report a case of acute intermittent porphyria in a 19-year-old patient, linked to an unlisted variant of the gene encoding hydroxymethylbilane synthase c.345–2A>C.
Julien Lerusse, Dominique Dive, François Charles Wang   +2 more
doaj   +1 more source

Sporadic Porphyria Cutanea Tarda, Cutaneous Sarcoidosis, and Compound Heterozygosity of HFE Mutations Cys282Tyr and His63Asp—A Case Report

eJHaem, Volume 6, Issue 3, June 2025.
ABSTRACT Porphyria cutanea tarda (PCT) is caused by inherited or acquired defects of uroporphyrinogen decarboxylase (UROD) in the heme biosynthetic pathway. Altered iron homeostasis via hemochromatosis gene (HFE) mutations is one of many susceptibility factors associated with the sporadic form of PCT.
Jowon L. Kim, Richard Crawford, Ian Marie Lano, Hayley Merkeley   +3 more
wiley   +1 more source