Results 191 to 200 of about 143,834 (286)

Building a precision therapeutics program at a tertiary care children's hospital

Pediatric Investigation, EarlyView.
Luke Hamilton, Heidi Trinkman, Diana Carrasco, Kaitlin Smith, Kelly Vallance, Alejandra Dominguez, Danielle Miller, Megan Gibbs, Anish Ray   +8 more
wiley   +1 more source

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

British Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi, Nandakumar Natarajan, Kathryn E. Dickerson, Richard A. Voit   +3 more
wiley   +1 more source

Antibody-oligonucleotide conjugates: design principles, intracellular delivery, and translational opportunities. [PDF]

J Hematol Oncol
Li Y, Liu X, Zhang F, Fu R, Zhao D, Ye L, Zhu YZ.   +6 more
europepmc   +1 more source

Peripheral targets for neuropathic pain

British Journal of Pharmacology, EarlyView.
Neuropathic pain represents a significant clinical challenge, with still limited pharmacological approaches to symptomatic relief. This review focuses on molecular targets implicated in neuropathic pain, particularly those involved in peripheral mechanisms. Using the IUPHAR/BPS database of biological targets, their occurrence together with ‘neuropathic
Amirhossein Afsharipour, Cosmin I. Ciotu, Felix J. Resch, Michael J. M. Fischer   +3 more
wiley   +1 more source

Transient Cyclic Structured Oligonucleotide Designs for Therapeutic Applications. [PDF]

Curr Protoc
Agrawal S.
europepmc   +1 more source

Extracellular vesicles in pharmacology: Innovations in drug delivery and therapeutic applications in cancer

British Journal of Pharmacology, EarlyView.
Extracellular vesicles (EVs) are a diverse population of membrane nanoparticles secreted by nearly all cell types, playing a key role in intercellular communication by transferring bioactive macromolecular cargo. In cancer, EVs shape both the local tumour microenvironment and distant premetastatic niches.
Evangelia Pantazaka, Georgios Sivvas, Evangelia Papadimitriou, Constantinos M. Mikelis   +3 more
wiley   +1 more source

Survivin-Targeting Antisense Oligonucleotides in Cancer Therapy. [PDF]

Molecules
Poudel BH, Chen S, Veedu RN.
europepmc   +1 more source

Selective modulation of NaV channel gating counteracts aberrant hyperexcitability and rescues motor function and survival in a model of spinal muscular atrophy

British Journal of Pharmacology, EarlyView.
Background and Purpose Spinal muscular atrophy (SMA) is a motor neuron disease caused by SMN1 gene loss, leading to reduced survival motor neuron (SMN) protein and progressive motor neuron degeneration. Although SMN‐restoring therapies improve outcomes, residual disease burden and non‐curative efficacy underscore the need for complementary treatments ...
Fernanda C. Cardoso, Jessica A. I. Muller, Yougang Zhang, Jean Giacomotto   +3 more
wiley   +1 more source

The case for targeting enhancer regions in high-risk TCF3::HLF-positive B-cell acute lymphoblastic leukemia. [PDF]

Hemasphere
Sheybani Z, de Bock CE.
europepmc   +1 more source

Long non‐coding RNAs at the crossroads of inflammation, cancer and angiogenesis: Molecular mechanisms and their potential as therapeutic targets

British Journal of Pharmacology, EarlyView.
Long non‐coding RNAs (lncRNAs), a broad class of non‐protein‐coding RNAs, are characterized as new regulators of gene expression at the epigenetic, transcriptional, and post‐transcriptional level. Thus, lncRNAs are involved in the regulation of physiological processes and the development of human diseases and cancer by modulating proinflammatory ...
Charlie Leboff, Pamela Calle‐Mendoza, Maria Hatziapostolou, Christos Polytarchou   +3 more
wiley   +1 more source