Results 71 to 80 of about 1,551 (185)

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

Heme Biosynthetic Gene Expression Analysis With dPCR in Erythropoietic Protoporphyria Patients [PDF]

, 2022
Background: The heme biosynthesis (HB) involves eight subsequent enzymatic steps. Erythropoietic protoporphyria (EPP) is caused by loss-of-function mutations in the ferrochelatase (FECH) gene, which in the last HB step inserts ferrous iron into ...
Barman-Aksozen J., Brancaleoni V., De Luca G., Di Pierro E., Fustinoni S., Granata F., Graziadei G., Motta I., Scopetti M.   +8 more
core   +1 more source

Tyrosine-Modification of Polypropylenimine (PPI) and Polyethylenimine (PEI) Strongly Improves Efficacy of siRNA-Mediated Gene Knockdown [PDF]

, 2023
The delivery of small interfering RNAs (siRNA) is an efficient method for gene silencing through the induction of RNA interference (RNAi). It critically relies, however, on efficient vehicles for siRNA formulation, for transfection in vitro as well as ...
Aigner, Achim, Ewe, Alexander, Karimov, Michael, Noske, Sandra   +3 more
core   +1 more source

Hydrophilic Hyperbranched Polymer-Coated siRNA/Polyamidoamine Dendron-Bearing Lipid Complexes Preparation for High Colloidal Stability and Efficient RNAi [PDF]

, 2021
application/pdfBioconjugate Chemistry.
28250, 28251, 28252, Harada, Atsushi, Korenaga, Takashi, Yuba, Eiji, 原田, 敦史, 弓場, 英司   +7 more
core   +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

Acute porphyrias – A neurological perspective [PDF]

, 2021
Acute hepatic porphyrias (AHP) can cause severe neurological symptoms involving the central, autonomic, and peripheral nervous system. Due to their relative rarity and their chameleon-like presentation, delayed diagnosis and misdiagnosis are common. AHPs
Gerischer, Lea M., Köhnlein, Martin, Meisel, Andreas, Nümann, Astrid, Scheibe, Franziska, Stölzel, Ulrich   +5 more
core   +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

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

Clinical Features and Outcomes of Acute Intermittent Porphyria Presenting With Acute Quadriparesis: A Case Series and Follow‐Up Study

European Journal of Neurology, Volume 32, Issue 6, June 2025.
ABSTRACT Introduction A retrospective case series of acute intermittent porphyria (AIP) presenting with acute quadriparesis is described with a focus on patterns of neuropathy and nerve conduction study findings. Methods Six patients with acute polyneuropathy were diagnosed with AIP on the basis of characteristic clinical findings, urine ...
Reem M. Alhammad, Rema A. Almutawa, Abdulrahman S. Ali, Nuha M. Alkhawajah, Hana M. Albulaihe, Afag M. Osman, Mohammed A. AlSheef, Alaa A. Alsayed, Raashda A. Sulaiman, Khalid J. Alharbi   +9 more
wiley   +1 more source