Blastic plasmacytoid dendritic cell neoplasm: Genomics mark epigenetic dysregulation as a primary therapeutic target [PDF]
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematologic malignancy for which there is still no effective B therapy.
Abate F.+30 more
core +2 more sources
This study reveals how prime editing guide RNA (pegRNA) secondary structure and reverse transcriptase template length affect prime editing efficiency in correcting the phospholamban R14del cardiomyopathy‐associated mutation. Insights support the design of structurally optimized enhanced pegRNAs for precise gene therapy.
Bing Yao+7 more
wiley +1 more source
Intracellular localisation of mTHPC and effect of photodynamic therapy in cells of the mammalian peripheral nervous system [PDF]
Fewer nerve-related side effects have been noted after treating head and neck cancer with photodynamic therapy (PDT) compared to conventional cancer therapy. Our aim is to investigate the biological basis for any such nerve-sparing effect.
MacRobert, A. J.+2 more
core
A bioprinted cardiac patch composed of cardiac-specific extracellular matrix and progenitor cells for heart repair [PDF]
Congenital heart defects are present in 8 of 1000 newborns and palliative surgical therapy has increased survival. Despite improved outcomes, many children develop reduced cardiac function and heart failure requiring transplantation.
Bejleri, Donald+6 more
core +1 more source
Single‐cell insights into the role of T cells in B‐cell malignancies
Single‐cell technologies have transformed our understanding of T cell–tumor cell interactions in B‐cell malignancies, revealing new T‐cell subsets, functional states, and immune evasion mechanisms. This Review synthesizes these findings, highlighting the roles of T cells in pathogenesis, progression, and therapy response, and underscoring their ...
Laura Llaó‐Cid
wiley +1 more source
Functional roles of the membrane-associated AAV protein MAAP
With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved over-lapping genes to maximise the usage of its genome. An example is the recently found ORF in the cap gene, encoding membrane-associated accessory protein (MAAP),
Lionel Galibert+13 more
doaj +1 more source
Generation and characterization of a mitotane-resistant adrenocortical cell line [PDF]
Mitotane is the only drug approved for the therapy of adrenocortical carcinoma (ACC). Its clinical use is limited by the occurrence of relapse during therapy.
Bachmann, Sebastian+14 more
core +2 more sources
Transplantation of islet allografts into type 1 diabetic recipients usually requires multiple pancreas donors to achieve insulin independence. This adds to the challenges of immunological monitoring of islet transplantation currently relying on surrogate
Cornelis R. Van Der Torren+12 more
doaj +1 more source
The natural dietary genistein boosts bacteriophage-mediated cancer cell killing by improving phage-targeted tumor cell transduction [PDF]
Gene therapy has long been regarded as a promising treatment for cancer. However, cancer gene therapy is still facing the challenge of targeting gene delivery vectors specifically to tumors when administered via clinically acceptable non-invasive ...
Albahrani, M+5 more
core +2 more sources
Cyclic nucleotide signaling as a drug target in retinitis pigmentosa
Disruptions in cGMP and cAMP signaling can contribute to retinal dysfunction and photoreceptor loss in retinitis pigmentosa. This perspective examines the mechanisms and evaluates emerging evidence on targeting these pathways as a potential therapeutic strategy to slow or prevent retinal degeneration.
Katri Vainionpää+2 more
wiley +1 more source