Characterization of a novel conditional knockout mouse model to assess efficacy of mRNA therapy in the context of severe ornithine transcarbamylase deficiency.

Ornithine transcarbamylase deficiency (OTCD) is the most common urea cycle disorder, characterized by hyperammonemia and accompanied by a high unmet patient need. mRNA therapies have been shown to be efficacious in hypomorphic Sparse-fur abnormal skin and hair (Spf-ash) mice, a model of late-onset disease. However, studying the efficacy of ornithine transcarbamylase (OTC) mRNA therapy in traditional knockout mice, a model for severe early-onset OTCD, is hampered by the rapid lethality of the model, and poor lipid nanoparticle (LNP) uptake into neonatal mouse liver.

Multiple Free Flaps and Second Toe Transfer to Salvage Grasp Function in Bilateral Complete Degloved Hands.

A 21-year-old male laborer sustained bilateral degloving injury of the hands with multiple digital amputations and devascularized digits. After X-rays, preliminary debridement was done, when digital amputations were completed, including index ray amputation on both sides. The next day, two anterolateral thigh (ALT) flaps and one second toe transfer were done to restore coverage in the palm and the web and reconstruct the lost thumb.

Meta-regression of optic nerve imaging and visual outcome in myelin oligodendrocyte glycoprotein antibody optic neuritis.

Background: Few predictors of visual outcome after myelin oligodendrocyte glycoprotein (MOG) auto-antibody disease optic neuritis (ON) have been reliably elucidated. We evaluate whether between-study differences in ON neuroimaging regional enhancement features may underlie heterogeneity in reported visual prognosis.

Methods: PROSPERO (CRD42024580123).