Inhibition of FcRn with rozanolixizumab in adults with immune thrombocytopenia: Two randomised, double-blind, placebo-controlled phase 3 studies and their open-label extension.

Primary immune thrombocytopenia (ITP) is an antiplatelet-antibody-mediated disorder with accelerated platelet clearance and decreased platelet production. Rozanolixizumab, a monoclonal IgG4 anti-FcRn antibody, blocks IgG recycling and decreases IgG levels. We report efficacy and safety of rozanolixizumab in adults with persistent/chronic ITP in 24-week phase 3 studies (TP0003; TP0006), and their 52-week open-label extension (OLE).

Mummy of a juvenile sabre-toothed cat Homotherium latidens from the Upper Pleistocene of Siberia.

The frozen mummy of the large felid cub was found in the Upper Pleistocene permafrost on the Badyarikha River (Indigirka River basin) in the northeast of Yakutia, Russia. The study of the specimen appearance showed its significant differences from a modern lion cub of similar age (three weeks) in the unusual shape of the muzzle with a large mouth opening and small ears, the very massive neck region, the elongated forelimbs, and the dark coat color. Tomographic analysis of the mummy skull revealed the features characteristic of Machairodontinae and of the genus Homotherium.

Archaeocytes in sponges: simple cells of complicated fate.

Archaeocytes are considered a key cell type in sponges (Porifera). They are believed to be multifunctional cells performing various functions, from nutrient digestion to acting as adult stem cells (ASCs). Thus, archaeocytes are mentioned in discussions on various aspects of sponge biology.

The hydroxycarboxylic acid receptor HCA2 is required for the protective effect of ketogenic diet in epilepsy.

One third of epilepsy patients are resistant to treatment with current anti-seizure medications. The ketogenic diet is used to treat some forms of refractory epilepsy, but the mechanism of its action has not yet been elucidated. In this study, we aimed to investigate whether the hydroxycarboxylic acid receptor 2 (HCA2), a known immunomodulatory receptor, plays a role in mediating the protective effect of this diet.

Etruscan Wolf Canis etruscus (Canidae, Carnivora) from the Early Pleistocene of Crimea (Taurida Cave).

The cranial and mandibular fragments of the medium-sized canid from the Early Pleistocene locality of Taurida cave (about 1.8-1.5 Ma) in Crimea are described and included in the hypodigm of the species Canis etruscus Forsyth Major, 1877, a common member of the Late Villafranchian fauna of the Circum-Mediterranean region.

Synthesis, Structure, and Properties of a Copper(II) Binuclear Complex Based on Trifluoromethyl Containing Bis(pyrazolyl)hydrazone.

A new complex of copper(II) with methyl-5-(trifluoromethyl)pyrazol-3-yl-ketazine (HL) was synthesized with the composition [CuL]∙CHOH (). Recrystallization of the sample from DMSO yielded a single crystal of the composition [CuL((CH)SO)] (). The coordination compounds were studied by single-crystal X-ray diffraction analysis, IR spectroscopy, and static magnetic susceptibility method.

Cheetah Acinonyx pardinensis (Felidae, Carnivora) from the Early Pleistocene of Crimea (Taurida Cave).

A mandible fragment of Acinonyx pardinensis (Croizet et Jobert, 1828) is described from the Early Pleistocene locality in the Taurida cave (Crimea, Late Villafranchian, about 1.8-1.5 Ma).

Airway basal cells from human-induced pluripotent stem cells: a new frontier in cystic fibrosis research.

Human-induced airway basal cells (hiBCs) derived from human-induced pluripotent stem cells (hiPSCs) offer a promising cell model for studying lung diseases, regenerative medicine, and developing new gene therapy methods. We analyzed existing differentiation protocols and proposed our own protocol for obtaining hiBCs, which involves step-by-step differentiation of hiPSCs into definitive endoderm, anterior foregut endoderm, NKX2.1+ lung progenitors, and cultivation on basal cell medium with subsequent cell sorting using the surface marker CD271 (NGFR).

Evolution of Prime Editing Systems: Move Forward to the Treatment of Hereditary Diseases.

The development of gene therapy using genome editing tools recently became relevant. With the invention of programmable nucleases, it became possible to treat hereditary diseases due to introducing targeted double strand break in the genome followed by homology directed repair (HDR) or non-homologous end-joining (NHEJ) reparation. CRISPR-Cas9 is more efficient and easier to use in comparison with other programmable nucleases.

Cell cycle dynamics of food-entrapping cells of sponges: an experimental approach.

Sponges (phylum Porifera) exhibit surprisingly complex tissue dynamics, maintaining constant cell turnover and migration, rearranging internal structures, and regenerating after severe injuries. Such tissue plasticity relies on the activity of proliferating cells represented primarily by the food-entrapping cells, choanocytes. Although there are plenty of studies regarding the dynamics of regeneration and tissue rearrangement in sponges, cell cycle kinetics of choanocytes in intact tissues remains a controversial issue.

Generation of induced pluripotent stem cell line (RCMGi012-A) from fibroblasts of patient with mucopolysaccharidosis type VI.

Skin fibroblasts obtained from a 5-year-old girl with genetically proven (two heterozygous mutations in ARSB gene) and clinically manifested mucopolysaccharidosis type VI were successfully transformed into induced pluripotent stem cells by using Sendai virus-based reprogramming vectors including the four Yamanaka factors namely SOX2, OCT3/4, KLF4, and c-MYC. These iPSCs expressed pluripotency markers, had a normal karyotype and the potential to differentiate into three germ layers in spontaneous differentiation assay. The line may be used for cell differentiation and pharmacological investigations, and also may provide a model for development of a personalized treatment including drug screening and genome editing.

Unexpected extra exon skipping in the DYSF gene during restoring the reading frame by CRISPR/Cas9.

The DYSF gene encoding dysferlin protein is one of the largest and has many transcripts. Pathogenic variants in the gene can lead to various types of myopathies, which makes it a good object for studying the events occurring in it during genome editing by the CRISPR/Cas method. In this study, we evaluated the possibility of permanent skipping of exons 3-4, and 26-27 which deletion does not violate the reading frame and allows to eliminate truncated variants within exons.

Synthesis, Structural Versatility, Magnetic Properties, and I Adsorption in a Series of Cobalt(II) Metal-Organic Frameworks with a Charge-Neutral Aliphatic (O,O)-Donor Bridge.

Four new metal-organic frameworks based on cobalt(II) salts and 1,4-diazabicyclo[2.2.2]octane N,N'-dioxide (odabco) were obtained.

Mustela palerminea (Mustelidae, Carnivora) from the Early Pleistocene of Crimea.

The mandibles of two individuals of Mustela palerminea (Petenyi, 1864) are described  from the Lower Pleistocene deposits of the Taurida cave in Crimea (Late Villafranchian, 1.8-1.5 Ma).

Mustela strandi (Mustelidae, Carnivora) from the Early Pleistocene of Crimea.

The dentary of Mustela strandi Kormos, 1934 is described from the Lower Pleistocene deposits (Late Villafranchian, 1.8-1.5 Ma) of the Taurida cave in Crimea.

Generation of two iPSC lines from patient with Mucopolysaccharidosis IV B type and autosomal recessive non-syndromic hearing loss 12.

We generated two human induced pluripotency stem cell (hiPSC) lines, RCMGi011-A and 11-B, from skin fibroblast from patient with Mucopolysaccharidosis IV B type and autosomal recessive non-syndromic hearing loss 12 using non-integrating, viral CytoTune™-iPS 2.0 Sendai Reprogramming Kit. We verified variant c.

Synthesis, Structure, and Magnetic and Biological Properties of Copper(II) Complexes with 1,3,4-Thiadiazole Derivatives.

New coordination compounds of copper(II) with 2,5-bis(ethylthio)-1,3,4-thiadiazole (L) and 2,5-bis(pyridylmethylthio)-1,3,4-thiadiazole (L) with compositions Cu(L)Br, Cu(L)(CN), Cu(L)Cl, and Cu(L)Br were prepared. The complexes were identified and studied by CHN analysis, infrared (IR) spectroscopy, powder X-Ray diffraction (XRD), and static magnetic susceptibility. The crystal structures of Cu(II) complexes with L were determined.

Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial.

Bulbar function in spinal muscular atrophy has been defined as the ability to meet nutritional needs by mouth while maintaining airway protection and communicate verbally. The effects of disease-modifying treatment on bulbar function are not clear. A multidisciplinary team conducted post-hoc analyses of phase 3 SPR1NT trial data to evaluate bulbar function of infants at risk for spinal muscular atrophy who received one-time gene replacement therapy (onasemnogene abeparvovec) before symptom onset.

Iridium Complexes with BIAN-Type Ligands: Synthesis, Structure and Redox Chemistry.

A series of iridium complexes with bis(diisopropylphenyl)iminoacenaphtene (dpp-bian) ligands, [Ir(cod)(dpp-bian)Cl] (), [Ir(cod)(NO)(dpp-bian)](BF) () and [Ir(cod)(dpp-bian)](BF) (), were prepared and characterized by spectroscopic techniques, elemental analysis, X-ray diffraction analysis and cyclic voltammetry (CV). The structures of - feature a square planar backbone consisting of two C = C π-bonds of 1,5-cyclooctadiene (cod) and two nitrogen atoms of dpp-bian supplemented with a chloride ion (for ) or a NO group (for ) to complete a square-pyramidal geometry. In the nitrosyl complex , the Ir-N-O group has a bent geometry (the angle is 125°).

Generation of induced pluripotent stem cell line (RCMGi009-A) from urine cells of patient with fibrodysplasia ossificans progressiva.

Urine cells obtained from a 14-year-old man with genetically proven (ACVR1: c.6176G > A) and clinically manifested fibrodysplasia ossificans progressiva were successfully transformed into induced pluripotent stem cells by using Sendai virus-based reprogramming vectors including the four Yamanaka factors such as OCT3/4, SOX2, KLF4, and c-MYC. These iPSCs expressed pluripotency markers, exhibited the potential to differentiate into three germ layers in spontaneous differentiation assay and had a normal karyotype.

Semiconducting Electronic Structure of the Ferromagnetic Spinel HgCr_{2}Se_{4} Revealed by Soft-X-Ray Angle-Resolved Photoemission Spectroscopy.

We study the electronic structure of the ferromagnetic spinel HgCr_{2}Se_{4} by soft-x-ray angle-resolved photoemission spectroscopy (SX-ARPES) and first-principles calculations. While a theoretical study has predicted that this material is a magnetic Weyl semimetal, SX-ARPES measurements give direct evidence for a semiconducting state in the ferromagnetic phase. Band calculations based on the density functional theory with hybrid functionals reproduce the experimentally determined band gap value, and the calculated band dispersion matches well with ARPES experiments.

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment.

Background: Improvement and maintenance of bulbar function are goals of disease-modifying treatments for spinal muscular atrophy (SMA). Lack of standardized measures and a widely accepted definition of bulbar function represents a gap in SMA care.

Objective: A multidisciplinary team conducted post-hoc analyses of pooled data from one phase 1 (START) and two phase 3 (STR1VE-US, STR1VE-EU) studies to define and evaluate bulbar function of infants with SMA type 1 after receiving one-time gene replacement therapy, onasemnogene abeparvovec.