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Genetic muscular disease\Myotonic disorders
SCIE.05.17
GENE EDITING IN MYOTONIC DYSTROPHY TYPE 1: ASSESSMENT OF EFFICIENCY, SAFETY AND THERAPEUTIC EFFECT OF CTG-REPEAT DELETION IN A MOUSE MODEL OF DISEASE

Izzo M. * [1] , Battistini J. [1] , Cardinali B. [1] , Provenzano C. [1] , Mandillo S. [1] , Golini E. [1] , Strimpakos G. [1] , Scavizzi F. [1] , Raspa M. [1] , Voellenkle C. [2] , Perfetti A. [2] , Baci D. [2] , Martelli F. [2] , Lazarevic D. [3] , Garcia-Manteiga J.M. [3] , Gourdon G. [4] , Falcone G. [1]

Monterotondo (RM) [1] , San Donato Milanese, Milan [2] , Milan [3] , Paris [4]
SCIE.16.19
ROLE OF POLYAMINES-EIF5A-AUTOPHAGY AXIS IN THE PATHOGENESIS OF MYOTONIC DYSTROPHY TYPE 2

Bordone R. , Coni S. , Marzullo M. , D'Amico R. , Ciapponi L. , Canettieri G. *

Roma
SCIE.05.18
TRIAL READINESS AND ENDPOINT ASSESSMENT IN CONGENITAL AND CHILDHOOD MYOTONIC DYSTROPHY: OUTCOME MEASURES AND ENDPOINT ASSESSMENTS (GUP19002)

Albamonte E. [1] , Trucco F. [1] , Di Bari A. [1] , Salmin F. [1] , Fiorillo C. [2] , D'Amico A. [3] , Ricci F. [4] , Pini A. [5] , Astrea G. [6] , Moroni I. [1] , Berardinelli A. [7] , Mercuri E. [3] , Sansone V. * [1]

Milano [1] , Genova [2] , Roma [3] , Torino [4] , Bologna [5] , Pisa [6] , Pavia [7]