N. Bresolin - E. Medea

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DMD genotypes for exon- skipping of AC133+ stem cells Exon phasing around exon 51 : DMD genotypes selected
Lentivirus U7exon51 map : Promoteur U7 (267 Pb) GGGUCUAGAUAACAACAUAGGAGCUGUGAU UGGCUGUUUUCAGCCAAUCAGCACUGACUC AUUUGCAUAGCCUUUACAAGCGGUCACAAAC UCAAGAAACGAGCGGUUUUAAUAGUCUUUUA GAAUAUUGUUUAUCGAACCGAAUAAGGAACU GUGCUUUGUGAUUCACAUAUCAGUGGAGGG GUGUGGAAAUGGCACCUUGAUCUCACCCUC AUCGAAAGUGGAGUUGAUGUCCUUCCCUGG CUCGCUACAGACGCACUUCCGCAA Lentivirus-mediated exon-skipping U7SmOPT (85 pb) Downstream Sequences (116 pb) CCCAAUUUCACUGGU CUACAAUGAAAGCAA AACAGUUCUCUUCCC CGCUCCCCGGUGUG UGAGAGGGGCUUUG AUCCUUCUCUGGUUU CCUAGGAAACGCGUA UGUGGCUAGCUUU U C G A U G C U A C G U A U A U A U A G C 5’ CCUCUGUGAUUUUAUAACUUGAU/UCAAGGAAGAUGGCAUUUCUAAUUUUUGGAGCAG 3’ UC h51AON2 h51AON1 Site de liaison aux protéines SM (OPT) U G C G A U G C U A C G U A U A U A U A G C 5’ CCUCUGUGAUUUUAUAACUUGAU/UCAAGGAAGAUGGCAUUUCUAAUUUUUGGAGCAG CCCU 3’ UC U G C G A U G C U A C G U A U A U A U A G C 5’ CCUCUGUGAUUUUAUAACUUGAU/UCAAGGAAGAUGGCAUUUCUAAUUUUUGGAGCAG CCCU 3’ UC U G C G A U G C U A C G U A U A U A U A G C CCUCUGUGAUUUUAUAACUUGAU/UCAAGGAAGAUGGCAUUUCUAAUUUUUGGAGCAG CCCU UC U G C G A U G C U A C G U A U A U A U A G C CCUCUGUGAUUUUAUAACUUGAU/UCAAGGAAGAUGGCAUUUCUAAUUUUUGGAGCAG CCCU UC h51AON2 h51AON1 Site de liaison aux protéines SM (OPT) G CCCU Skipped band A Characteristics : . Pseudotype : VSV-G . Title : 2.10 9 ip/ml . Transduction : 10 6 to 10 8 ip/ml Exon-skipping efficiency in vitro : tested on human myoblasts Δ52 1 2 3 4 1 2 3 4 T 30 50 B Ex50 Ex53 Legend : 1 : Myob Δ52 no transduced 2 : Transduction (10 6 ip/ml) 3 : Transduction (10 7 ip/ml) 4 : H 2O
Page 1 and 2: L’impiego delle cellule staminali
Page 3 and 4: Cellule staminali • Capacità di
Page 5: Blood blood cells Vessels endotheli
Page 8: In vitro differentiation of iPS. In
Page 11 and 12: SVZ Isolation Astrocytes SVZ MATURE
Page 13 and 14: Multipotentiali Multipotentiality,
Page 15 and 16: Il trapianto di cellule staminali L
Page 17 and 18: Distrofia Muscolare di Duchenne E
Page 19 and 20: Il nostro obiettivo: trapiantare le
Page 21 and 22: Muscle homing of the Sca-1+/CD34-MD
Page 23 and 24: Myogenic differentiation of Sca-1+/
Page 25 and 26: TRAP assay Clonogenic, self-renewal
Page 27 and 28: Double-blinded randomized clinical
Page 29 and 30: Local side effects after intramuscu
Page 31 and 32: Intra-arterial delivery of wild-typ
Page 33 and 34: Expression of α-SG and dystrophin
Page 35: Three-dimensional visualization of
Page 39: Human dystrophin expression in scid
Page 43 and 44: Sclerosi Laterale Amiotrofica
Page 45 and 46: LeX+CX+ cells share the properties
Page 47 and 48: Time to fall (s) 250 200 150 100 50
Page 49 and 50: Il trapianto di cellule LeX+CXCR4+
Page 51 and 52: Cellule staminali nelle malattie de
Page 53 and 54: Atrofia Muscolare Spinale (SMA) SMN
Page 56: Neuroni ALDH proiettano lunghi asso
Page 60 and 61: GFP/Nestina Predifferenziamento GFP
Page 62 and 63: Midollo spinale di topo trapiantato
Page 64 and 65: Effetti del trapianto sui motoneuro
Page 66 and 67: Utilizzo di sostanze per promuovere
Page 68: Stem Cell Research Institute DIBIT-

N. Bresolin - E. Medea

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