Publication

DMD-TREATMENT: EP. 147 2.5-years of vamorolone treatment in Duchenne muscular dystrophy: Results of an open label long-term extension

Vamorolone is a first-in-class steroidal anti-inflammatory drug with novel structure/activity relationships with glucocorticoid and mineralocorticoid receptor targets compared to deflazacort or prednisone. Published open-label dose-finding studies (0.25-6.0 mg/kg/day) in DMD showed significant motor function improvement over 24 weeks for 2.0 and 6.0 mg/kg/day dose groups (n=48; age 4 to 6-month delay and maintained a higher dose through the remaining study period. Analyses of disease trajectories showed a strong effect of age at initiation of treatment.

LRG1 destabilizes tumor vessels and restricts immunotherapeutic potency

Background A poorly functioning tumor vasculature is pro-oncogenic and may impede the delivery of therapeutics. Normalizing the vasculature, therefore, may be beneficial. We previously reported that the secreted glycoprotein leucine-rich α-2-glycoprotein 1 (LRG1) contributes to pathogenic neovascularization. Here, we investigate whether LRG1 in tumors is vasculopathic and whether its inhibition has therapeutic utility. Methods Tumor growth and vascular structure were analyzed in subcutaneous and genetically engineered mouse models in wild-type and Lrg1 knockout mice.

Non-clinical assessment of safety, biodistribution and tumorigenicity of human mesenchymal stromal cells

Guidelines regulating the development of advanced therapy medicinal products (ATMPs) request nonclinical data for toxicity, biodistribution and tumorigenicity before mesenchymal stromal cell (MSC) products can be administered in large clinical trials.

TNF-α-producing macrophages determine subtype identity and prognosis via AP1 enhancer reprogramming in pancreatic cancer

A,B, Expression correlation analysis in 78 PDAC patient tumors12 [/articles/s43018-021-00258-w#ref-CR12] (E-MTAB-6134 [http://www.ebi.ac.uk/arrayexpress/experiments/E-MTAB-6134/]) with high tumor cellularity between cJUN and VIM (A) as well as GATA6 and VIM (B). RMA-normalized probe intensity values were plotted. A linear regression with 95% CI is shown in orange. Pearson’s correlation (_R_) and corresponding two-tailed _P_ value are indicated.

Abstract PO-117: The role of Hippo signaling in stromal-epithelial interactions in acinar-to-ductal metaplasia and pancreatic cancer initiation

Background: Pancreatic ductal adenocarcinoma (PDAC) is the fourth leading cause of cancer deaths with a 5-year survival rate of approximately 7%. PDAC may originate from acinar cell trans-differentiation into ductal-like cells, termed acinar-to-ductal metaplasia (ADM), triggered by chronic pancreatitis and/or mutations in K-Ras. The progression to PDAC is associated with a dense fibrotic stroma, including cancer-associated fibroblasts (CAFs). YAP is a tension-stimulated CAF activator that promotes ECM stiffening, creating a permissive microenvironment for cancer progression.

Advances in mass cytometry and its applicability to digital pathology in clinical-translational cancer research

The development and subsequent adaptation of mass cytometry for the histological analysis of tissue sections has allowed the simultaneous spatial characterization of multiple components. This is useful to find the correlation between the genotypic and phenotypic profile of tumor cells and their environment in clinical-translational studies. In this revision, we provide an overview of the most relevant hallmarks in the development, implementation and application of multiplexed imaging in the study of cancer and other conditions.

The Male Reproductive Organs in African Swine Fever-Implications for Transmission

African swine fever (ASF) has evolved from an exotic animal disease to a threat to global pig production. An important avenue for wide-spread transmission of animal diseases is the dissemination of viruses through boar semen used for artificial insemination (AI). In this context, we investigated the role of male reproductive organs in ASF. Mature domestic boars and adolescent wild boar inoculated with different ASF virus strains were investigated by means of virological and pathological methods. Additionally, electron microscopy was employed to investigate in vitro inoculated sperm.

Wholemount in situ Hybridization for Spatial-temporal Visualization of Gene Expression in Early Post-implantation Mouse Embryos

Wholemount _in situ_ hybridization has been widely used to explore gene expression distribution in both tissues and sections (Hauptmann and Gerster, 1994; Nieto _et al._, 1996). In the field of developmental biology, information on the spatial and temporal distribution of gene expression revealed by _in situ_ hybridization has facilitated the identification of master regulators of embryogenesis.

COVID-19 induces neuroinflammation and loss of hippocampal neurogenesis

Infection with the Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) is associated with onset of neurological and psychiatric symptoms during and after the acute phase of illness 1-4 . Acute SARS-CoV-2 disease (COVID-19) presents with deficits of memory, attention, movement coordination, and mood.

Circular Rims2 Deficiency Causes Retinal Degeneration

Circular RNAs (circRNAs) refer to a newly recognized family of non-coding RNA with single-stranded RNAs. Despite emerging evidence indicating that circRNAs are abundantly expressed in various tissues, especially in the brain and retina, the role of circRNAs in retinal function and diseases is still largely unknown. Circular Rims2 (circRims2) is highly expressed and conserved in both the human and mouse brains. However, little is known about the expression and function of circRims2 in the retina.

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