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Journal Article
Bello, Luca ORCID: 0000-0002-3075-6525, Morgenroth, Lauren P., Gordish-Dressman, Heather, Hoffman, Eric P., McDonald, Craig M. and Cirak, Sebahattin
(2016).
DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study.
Neurology, 87 (4).
S. 401 - 410.
PHILADELPHIA:
LIPPINCOTT WILLIAMS & WILKINS.
ISSN 1526-632X
Novak, James S., Hogarth, Marshall W., Boehler, Jessica F., Nearing, Marie ORCID: 0000-0002-5261-4544, Vila, Maria C., Heredia, Raul, Fiorillo, Alyson A., Zhang, Aiping, Hathout, Yetrib, Hoffman, Eric P., Jaiswal, Jyoti K., Nagaraju, Kanneboyina, Cirak, Sebahattin and Partridge, Terence A.
(2017).
Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.
Nat. Commun., 8.
LONDON:
NATURE PUBLISHING GROUP.
ISSN 2041-1723
Novak, James S., Hogarth, Marshall W., Boehler, Jessica F., Nearing, Marie, Vila, Maria C., Heredia, Raul, Fiorillo, Alyson A., Zhang, Aiping, Hathout, Yetrib, Hoffman, Eric P., Jaiswal, Jyoti K., Nagaraju, Kanneboyina, Cirak, Sebahattin and Partridge, Terence A. (2018). Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle (vol 8, 2017). Nat. Commun., 9. LONDON: NATURE PUBLISHING GROUP. ISSN 2041-1723
Novak, James S., Hogarth, Marshall W., Boehler, Jessica F., Nearing, Marie, Vila, Maria C., Heredia, Raul, Fiorillo, Alyson A., Zhang, Aiping, Hathout, Yetrib, Hoffman, Eric P., Jaiswal, Jyoti K., Nagaraju, Kanneboyina, Cirak, Sebahattin and Partridge, Terence A. (2018). Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle (vol 8, 941, 2017). Nat. Commun., 9. LONDON: NATURE PUBLISHING GROUP. ISSN 2041-1723
Novak, James S., Nearing, Marie, Hogarth, Marshall W., Vila, Maria C., Boehler, Jessica F., Hoffman, Eric P., Nagaraju, Kanneboyina, Cirak, Sebahattin and Partridge, Terence A. (2016). Myoblast Fusion Mediates Morpholino Entry and Exon Skipping In Dystrophic Muscle. Mol. Ther., 24. S. S232 - 1. NEW YORK: NATURE PUBLISHING GROUP. ISSN 1525-0024
Spitali, Pietro ORCID: 0000-0003-2783-688X, Zaharieva, Irina, Bohringer, Stefan, Hiller, Monika, Chaouch, Amina, Roos, Andreas, Scotton, Chiara, Claustres, Mireille, Bello, Luca
ORCID: 0000-0002-3075-6525, McDonald, Craig M., Hoffman, Eric P., Koeks, Zaida, Suchiman, H. Eka, Cirak, Sebahattin, Scoto, Mariacristina, Reza, Mojgan, 't Hoen, Peter A. C., Niks, Erik H., Tuffery-Giraud, Sylvie, Lochmueller, Hanns, Ferlini, Alessandra, Muntoni, Francesco, Aartsma-Rus, Annemieke, Dubrovsky, Alberto, Kornberg, Andrew, North, Kathryn, Ryan, Monique, Webster, Richard, Biggar, W. Douglas, McAdam, Laura C., Mah, Jean K., Kolski, Hanna, Vishwanathan, V., Chidambaranathan, S., Nevo, Yoram, Gorni, Ksenija, Carlo, Jose, Tulinius, Mar, Lotze, Timothy, Bertorini, Tulio E., Day, John W., Karachunski, Peter, Clemens, Paula R., Abdel-Hamid, Hoda, Teasley, Jean, Kuntz, Nancy, Driscoll, Sherilyn, Bodensteiner, John B., Connolly, Anne M., Pestronk, Alan, Abresch, R. T., Henricson, Erik K., Joyce, Nanette C., Cnaan, Avital, Gordish-Dressmsn, Heather, Morgenroth, Lauren P., Leshner, Robert, Tesi-Rocha, Carolina, Thangarajh, Mathula and Duong, Tina
(2020).
TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy.
Eur. J. Hum. Genet., 28 (6).
S. 815 - 826.
LONDON:
NATURE PUBLISHING GROUP.
ISSN 1476-5438
Willkomm, Lena, Heredia, Raul, Hoffmann, Katrin, Wang, Haicui, Voit, Thomas, Hoffman, Eric P. and Cirak, Sebahattin (2016). Homozygous mutation in Atlastin GTPase 1 causes recessive hereditary spastic paraplegia. J. Hum. Genet., 61 (6). S. 571 - 574. NEW YORK: NATURE PUBLISHING GROUP. ISSN 1435-232X