Results 91 to 100 of about 39,421 (210)

Challenges in Gene‐Directed Therapies for Rare Neurodevelopmental Disorders Using HNRNPU as an Exemplar

Clinical Genetics, EarlyView.
Beyond diagnosing rare neurodevelopmental disorders like HNRNPU‐RNDD, developing gene‐directed therapies requires overcoming complex hurdles, including spatial, developmental, and cell‐type constraints. Addressing these requires understanding the molecular effects of dosage and chromatin dynamics to deliver precise, effective treatments.
Daniel Aiyedun, Susan F. Rogers, Doaa M. Taha, Meena Balasubramanian   +3 more
wiley   +1 more source

Human Embryonic Stem Cell‐Derived Immunity‐And‐Matrix‐Regulatory Cells Attenuate Pulmonary Fibrosis via MMP1‐Mediated Collagen Degradation

Cell Proliferation, EarlyView.
Human embryonic stem cell‐derived Immunity‐and‐Matrix‐Regulatory Cells (IMRCs) effectively attenuate pulmonary fibrosis by secreting Matrix Metalloproteinase‐1 (MMP1), which directly degrades excess collagen I in the extracellular matrix. By directly degrading collagen I, IMRCs reverse ECM stiffness, suppress myofibroblast activation, and promote a ...
Zhongwen Li, Dingyun Song, Faguo Sun, Licheng Song, Bin An, Tingting Gao, Jingjing Liu, Kan Zhang, Kaidi Liu, Kaiwei Wu, Yajing Li, Yi Yang, Yaru Liu, Ruofan Su, Zai Wang, Tiemei Zhao, Huaiyong Chen, Li Xiao, Liang Peng, Liu Wang, Wei Li, Lixin Xie, Jie Hao, Huaping Dai, Jun Wu   +24 more
wiley   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Interleukin‐12: Central Roles in Immune Regulation and Disease Therapy

Immunology &Cell Biology, EarlyView.
IL‐12 levels are tightly regulated to balance effective protective immunity against infections and cancer with the risk of excessive inflammation and autoimmune disease. Both insufficient and excessive IL‐12 activity can therefore be detrimental, highlighting the importance of precisely controlling its production and signaling. ABSTRACT Interleukin‐12 (
Anthi Psoma, Geert van den Bogaart
wiley   +1 more source

BCG enables protection against malaria through adjuvanting an adenovirus-vectored vaccine

npj Vaccines
BCG vaccination against TB may also reduce malaria incidence through non-specific innate immune activation. We investigated whether giving BCG alongside an adenoviral-vectored malaria vaccine improves protection against malaria in mice.
Sören Reinke, Marta Ulaszewska, Qi Su, Lee Sims, Romain Guyon, Adam Truby, Gundra Raissar, Jelena S. Bezbradica, Joannah R. Fergusson, Anita Milicic   +9 more
doaj   +1 more source

Proteostasis in the regulation of pancreatic islet cell plasticity

Journal of Diabetes Investigation, EarlyView.
Adaptive proteostasis integrates protein synthesis, folding, trafficking, and degradation through ISR, UPR, ER stress responses, autophagy, and proteasomal degradation. In pancreatic islet cells, this coordinated network preserves protein quality, secretory function, proliferation, and survival, enabling adaptation to metabolic stress and aging while ...
Jun Shirakawa
wiley   +1 more source

Selective epithelial expression of KRASG12D in the Oncopig pancreas drives ductal proliferation and desmoplasia that is accompanied by an immune response

Scientific Reports
Pancreatic ductal adenocarcinoma (PDAC) remains a formidable challenge in oncology, characterized by a high mortality rate, largely attributable to delayed diagnosis and the intricacies of its tumor microenvironment.
Carlos P. Jara, Al-Murtadha Al-Gahmi, Audrey Lazenby, Michael A. Hollingsworth, Mark A. Carlson   +4 more
doaj   +1 more source

Development of adenoviral vectors that transduce Purkinje cells and other cerebellar cell-types in the cerebellum of a humanized mouse model

Molecular Therapy: Methods & Clinical Development
Viral vector gene therapy has immense promise for treating central nervous system (CNS) disorders. Although adeno-associated virus vectors (AAVs) have had success, their small packaging capacity limits their utility to treat the root cause of many CNS ...
Emre Kul, Uchechi Okoroafor, Amanda Dougherty, Lauren Palkovic, Hao Li, Paula Valiño-Ramos, Leah Aberman, Samuel M. Young, Jr.   +7 more
doaj   +1 more source

Treatment strategies for interstitial granulomatous dermatitis: Systematic review and expert opinion

Journal of the European Academy of Dermatology and Venereology, EarlyView.
Systematic review of 108 studies with a total of 182 patients with interstitial granulomatous dermatitis (IGD). IGD was frequently associated with systemic conditions or drug exposure. Management includes careful medication review and prompt withdrawal of potential triggers. Corticosteroids (systemic and topical) were commonly effective, while biologic
Francesco Bellinato, Sara Salvagno, Chiara Colato, Paolo Gisondi, Giampiero Girolomoni   +4 more
wiley   +1 more source

AQP1 Corrects Acinar Cells’ Secretion Deficits in Sjögren's Disease

Oral Diseases, EarlyView.
ABSTRACT Introduction Sjögren's disease (SjD) is a systemic autoimmune disease characterized by dry mouth, dry eyes, and profound fatigue. Preclinical studies suggest that AQP1 gene expression in salivary glands corrects salivary hypofunction in preclinical and clinical studies of radiation‐induced xerostomia and improves salivary and lacrimal function
Paola Perez, Blake M. Warner, Youngmi Ji, Sandra Afione, Thomas J. F. Pranzatelli, Margaret E. Beach, Zohreh Khavandgar, Margie Grisius, Eileen Pelayo, Daniel Martin, NIDCD/NIDCR Genomics and Computational Biology Core, Alan N. Baer, John A. Chiorini   +12 more
wiley   +1 more source