Search results for "PEDIATRIA"

Development of a new S/MAR containing Minicircle DNA vector: a new promise for Gene Therapy.

2009

A barrier limiting the use of nonviral vectors for gene therapy is related to the short duration of transgene expression in vivo. Development, evaluation, and optimisation of a long term transgene expression using a non viral vector is currently the primary aim in our research field. Recently, we have demonstrate that a nonviral episomal plasmid (pDNA) vector combined with a scaffold/matrix attachment region (S/MAR) is able to sustain long-term expression in murine liver for at least six months following hydrodynamic injection. However, plasmids contain sequences, which are essential for propagation in bacteria but are unnecessary for expression in mammalian cells. These bacterial component…

Sindrome da duplicazione 1q21: variabile penetranza ed espressività clinica intrafamiliare di un riarrangimento genomico di circa 6 Mb non precedente…

2016

Sindrome da duplicazione 1q21, riarrangimento genomico

Impact of Distressing Media Imagery on Children

2016

Children are particularly fragile and vulnerable to the impact of traumatic events or their mediated representation because they lack the skills and experience in the management of difficult information. Children have different concepts of health and disaster than adults and institutions, depending mainly on their cognitive, emotional, social, psychological, and physical development. If left alone with threatening messages, children are less able to fully understand the information to which they had been exposed. Therefore, they may fail to integrate the external data into their psychological schema of coping strategies. Children usually count on caregivers to deal with stressors, and they …

Traps o non Traps? Questo è il dilemma! Dati real life di una casistica con mutazione R92Q

2021

Thyroid dysfunction in pediatric patients with mild-moderate chronic renal insufficiency

2010

Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector

2008

An ideal gene therapy vector should enable persistent transgene expression without limitations of safety and reproducibility. Here we report the development of a non-viral episomal plasmid DNA (pDNA) vector that appears to fulfil these criteria. This pDNA vector combines a scaffold/matrix attachment region (S/MAR) with a human liver-specific promoter (alpha1-antitrypsin (AAT)) in such a way that long-term expression is enabled in murine liver following hydrodynamic injection. Long-term expression is demonstrated by monitoring the longitudinal luciferase expression profile for up to 6 months by means of in situ bioluminescent imaging. All relevant control pDNA constructs expressing luciferas…

Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver.

2010

We have previously described the development of a scaffold/matrix attachment region (S/MAR) episomal vector system for in vivo application and demonstrated its utility to sustain transgene expression in the mouse liver for at least 6 months following a single administration. Subsequently, we observed that transgene expression is sustained for the lifetime of the animal. The level of expression, however, does drop appreciably over time. We hypothesised that by eliminating the bacterial components in our vectors, we could improve their performance since bacterial sequences have been shown to be responsible for the immunotoxicity of the vector and the silencing of its expression when applied i…

MTHFR C677T homozygous as risk factor for complications after OLT for cryptogenic cirrhosis

2006

TROMBOSI VENOSA PROFONDA DA ESTROPROGESTINICI IN TETRASOMIA X

2009

TERATOMA IMMATURO EXTRACRANICO: Risultati dello Studio Cooperativo AIEOP "TCG-91".

1998

I Teratomi Immatuti sono tumori costituiti da tessuto non maligno ma con potenzialità evolutiva maligna. Gli Autori presentano i dati dello Studio Cooperativo "TCG-91".