MiRTOSMA - Exploring the potential on miRNA-based therapeutics to target mitochondria in spinal muscle atrophy

National Center for Gene Therapy and Drugs based on RNA Technology (CN RNA & Gene Therapy). Bando a cascata CN3SP4

Abstract

WP 1 Recruitment of SMA patients and collection of blood samples, as well as maintenance of the generated iPSCs in the BioBank.
WP 1 Generation of iPSCs from peripheral blood mononuclear cells PBMCs of SMA patients.Differentiation of iPSCs in skeletal muscle cells or neuromuscular organoids.
WP 1 Evaluation of miRNAs expression in iPSCs differentiated into skeletal muscle cells or neuromuscular organoids.
WP 2 Recruitment of SMA patients and collection of blood samples for iPSCs from SMA patients, as well as maintenance of the generated iPSCs in the BioBank.
WP 2 Cell culture maintenance and differentiation of iPSCs from SMA patients.
WP 2 Treatment of iPSCs differentiated into skeletal muscle cells or neuromuscular organoids with AntagomiRs to determine if it leads to a reduction in the levels of the corresponding microRNAs.
WP 2 RNA-sequencing to study the effect on global transcriptional reprogramming by antagomir.
WP 2 Identification of mitochondrial and other subcellular organelles shape changes in AntagoMirtreated iPSCs of SMA patients by transmission electron microscopy (TEM) imaging.
WP 2 Measurement of critical bioenergetic and functional parameters of mitochondria in AntagoMirtreated iPSCs from SMA patients using the Operetta (PerkinElmer) high-content imaging system,
WP 2 Measurement of metabolic reprogramming in AntagoMir-treated iPSCs from SMA patients using MALDI.

Partenariato

Importo del progetto

Importo del progetto Uniud Euro 245.745,00

Finanziamento Uniud Euro 0,00

Durata

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