Results 181 to 190 of about 144,032 (247)

Chronic Non‐Progressive Neurophysiology in an ALS Patient Treated With Tofersen for 6 Years

Muscle &Nerve, EarlyView.
Ivar Winroth, Christian Krarup, Henrik Berntsson, Lisette Salvesen, Kirsten Svenstrup, Erik Nordh, Peter M. Andersen   +6 more
wiley   +1 more source

Integrating novel therapeutic strategies in myeloproliferative neoplasms: Driving disease‐directed progress

British Journal of Haematology, EarlyView.
Summary Over the past decade, there has been a substantial increase in the diversity and number of therapeutic options for myeloproliferative neoplasms (MPNs). While many remain within the clinical trial arena, the clinician and patient community have seen more approvals reaching the clinic and a rethink on how best we should be approaching these ...
Trung Q. Ngo, Maria Georgiou, Donal P. McLornan, Claire N. Harrison   +3 more
wiley   +1 more source

Organ-specific redox imbalances in spinal muscular atrophy mice are partially rescued by SMN antisense oligonucleotides. [PDF]

FEBS Lett
Vrettou S, Wirth B.
europepmc   +1 more source

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

Characterisation of Antisense Oligonucleotides by Ion-Pair Reversed-Phase UHPLC-HRMS: Method development using Design of Experiments. [PDF]

J Mass Spectrom
Triolo A, Tavani F, Barnini P, Furlanetto S, Orlandini S.   +4 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

Glycyrrhetinic Acid-Grafted Glycogen Nanoparticles Mediated Hepatic Delivery of Survivin Antisense Oligonucleotides for Liver Cancer-Targeted Gene Therapy. [PDF]

ACS Omega
Zhang H, Li S, Cui R, He X, Liu S, Yin F, Zhang Y.   +6 more
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

Chemically inducible antisense oligonucleotides for cell-specific gene silencing. [PDF]

RSC Chem Biol
Xun Z, Hai Y, Tang LJ, Jiang JH, Wu Z.
europepmc   +1 more source