Therapeutic activity of modified U1 core spliceosomal particles

Malgorzata Ewa Rogalska; Mojca Tajnik; Danilo Licastro; Erica Bussani; Luca Camparini; Chiara Mattioli; Franco Pagani

doi:10.1038/ncomms11168

Nature Communications (Apr 2016)

Therapeutic activity of modified U1 core spliceosomal particles

Malgorzata Ewa Rogalska,
Mojca Tajnik,
Danilo Licastro,
Erica Bussani,
Luca Camparini,
Chiara Mattioli,
Franco Pagani

Affiliations

Malgorzata Ewa Rogalska: Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology
Mojca Tajnik: Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology
Danilo Licastro: CBM S.c.r.l., Area Science Park
Erica Bussani: Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology
Luca Camparini: Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology
Chiara Mattioli: Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology
Franco Pagani: Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology

DOI: https://doi.org/10.1038/ncomms11168
Journal volume & issue: Vol. 7, no. 1
pp. 1 – 13

Abstract

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Modification of the spliceosome is being tested as a potential therapy for exon-skipping diseases, such as spinal muscular atrophy (SMA). Here the authors show that 70K and stem loop IV structural elements of a modified U1 particle are essential for splicing enhancement and effective treatment of SMA mice.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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