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2015

Publications 2015

Auricchio A, Trapani I, Allikmets R (2015). Gene Therapy of ABCA4-Associated Diseases. Cold Spring Harb Perspect Med[Epub ahead of print]

Caiazzo M, Giannelli S, Valente P, Lignani G, Carissimo A, Sessa A, Colasante G, Bartolomeo R, Massimino L, Ferroni S, Settembre C, Benfenati F, Broccoli V (2015). Direct conversion of fibroblasts into functional astrocytes by defined transcription factors. Stem Cell Reports. 4 (1):25-36.

Fanin M, Savarese M, Nascimbeni AC, Di Fruscio G, Pastorello E, Tasca E, Trevisan CP, Nigro V, Angelini C (2015). Dominant muscular dystrophy with a novel SYNE1 gene mutation. Muscle Nerve. 51 (1):145-147.

Ferriero R, Iannuzzi C, Manco G, Brunetti-Pierri N (2015). Differential inhibition of PDKs by phenylbutyrate and enhancement of pyruvate dehydrogenase complex activity by combination with dichloroacetate. J Inherit Metab Dis[Epub ahead of print]

Parenti G, Andria G, Ballabio A (2015). Lysosomal storage diseases: from pathophysiology to therapy. Annu Rev Med. 66 471-486.

Roosing S, van den Born LI, Sangermano R, Banfi S, Koenekoop RK, Zonneveld-Vrieling MN, Klaver CC, van Lith-Verhoeven JJ, Cremers FP, den Hollander AI, Hoyng CB (2015). Mutations in MFSD8, Encoding a Lysosomal Membrane Protein, Are Associated with Nonsyndromic Autosomal Recessive Macular Dystrophy. Ophthalmology. 122 (1):170-179.

Strating JR, van der Linden L, Albulescu L, Bigay J, Arita M, Delang L, Leyssen P, van der Schaar HM, Lanke KH, Thibaut HJ, Ulferts R, Drin G, Schlinck N, Wubbolts RW, Sever N, Head SA, Liu JO, Beachy PA, De Matteis MA, Shair MD, Olkkonen VM, Neyts J, van Kuppeveld FJ (2015). Itraconazole Inhibits Enterovirus Replication by Targeting the Oxysterol-Binding Protein. Cell Rep. [Epub ahead of print]