CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

(AAV)-mediated episomal gene replacement therapy for monogenic liver disorders is currently limited in pediatric settings due to the loss of vector DNA, associated with hepatocyte duplication during liver growth. Genome editing is a promising strategy leading to a permanent and specific genome modification that is transmitted to daughter cells upon proliferation. Using genome targeting, we previously rescued neonatal lethality in mice with Crigler-Najjar syndrome.

Blood cell differential count discretisation modelling to predict survival in adults reporting to the emergency room: a retrospective cohort study.

Objectives: To assess the survival predictivity of baseline blood cell differential count (BCDC), discretised according to two different methods, in adults visiting an emergency room (ER) for illness or trauma over 1 year.

Design: Retrospective cohort study of hospital records.

Setting: Tertiary care public hospital in northern Italy.

Observed and Perceived Pain: Findings of a Cross-Sectional Study in Hospitalized Subjects.

Background: Pain constitutes a serious problem of a health, economic, ethical, and social equity nature affecting negatively quality of life. Its assessment is often subjected to overestimation or underestimation.

Aim: The aim of this study is threefold: (1) to estimate the prevalence of pain in hospitalized patients; (2) to assess the grade of correlation between the level of pain observed by the nurses and the pain perceived by the patients; and (3) to examine the level of scientific knowledge among the healthcare professionals.

Probabilistic graphical modelling of early childhood caries development.

In order to develop a statistical model for complex interactions among factors affecting early childhood caries development (ECC), 234 children from the "XXXX oral growth longitudinal study" were analysed at ages 3, 4 and 5. A questionnaire for children's parents (clinical history, nutritional and oral hygiene habits) and clinical oral examinations were recorded each year. The probabilistic dependence structure on the most significant factors was modelled with an Undirected Graphical Model (UGM or Markov random fields) which provides a probabilistic reasoning approach for the establishment of multi-way associations.

Recruitment Traits Could Influence Species' Geographical Range: A Case Study in the Genus L.

The reasons why some species occur widespread, while related species have restricted geographical ranges have been attributed to habitat specialization or ecological niche breadth. For species in the genus , habitat specialization alone cannot explain the distributional differences observed. We hypothesize that recruitment traits (i.