Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.

Introduction: Gene therapy for spinal muscular atrophy (SMA) represents a significant milestone in the treatment of neurologic diseases. SMA is a neurodegenerative disease that results in motor neuron loss because of mutations of the gene, which directs survival motor neuron (SMN) protein production. Onasemnogene abeparvovec, a one-time gene replacement therapy, delivers a functional transgene to restore SMN protein expression.

Topography of Bone Erosions at the Metatarsophalangeal Joints in Rheumatoid Arthritis: Bilateral Mapping by Computed Tomography.

Objectives: To describe the bilateral anatomical location of bone erosions (BE) at the metatarsophalangeal joints in patients with rheumatoid arthritis using computed tomography.

Materials And Methods: Eighteen consecutive patients with established rheumatoid arthritis prospectively underwent computed tomography of both forefeet. Each joint surface of the metatarsal heads (MTH) and the proximal phalangeal bases were divided into four quadrants: superior, plantar, tibial, and fibular.

The splicing FK506-binding protein-51 isoform plays a role in glioblastoma resistance through programmed cell death ligand-1 expression regulation.

Gliomas aberrantly express programmed cell death ligand-1 (PD-L1), which has a pivotal role in immunoevasion. The splicing isoform of , termed FKBP51s, is a PD-L1 foldase, assisting the immune checkpoint molecule in maturation and expression on the plasma membrane. The concept that PD-L1 supports tumor-intrinsic properties is increasingly emerging.

Seno-suppressive molecules as new therapeutic perspectives in rheumatic diseases.

Over the past years, through in vitro studies and unique animal models, biologists and clinicians have demonstrated that cellular senescence is at the root of numerous age-related chronic diseases including osteoarthritis and osteoporosis. This non-proliferative cellular syndrome can modify other surrounding tissue-resident cells through the establishment of a deleterious catabolic and inflammatory microenvironment. Targeting these deleterious cells through local or systemic seno-therapeutic agent delivery in pre-clinical models improves dramatically clinical signs and extends health span.