المؤلفون: Cesana M., Tufano G., Panariello F., Zampelli N., Soldati C., Mutarelli M., Montefusco S., Grieco G., Sepe L. V., Rossi B., Nusco E., Rossignoli G., Panebianco G., Merciai F., Salviati E., Sommella E. M., Campiglia P., Martello G., Cacchiarelli D., Medina D. L., Ballabio A.

المساهمون: Cesana, M., Tufano, G., Panariello, F., Zampelli, N., Soldati, C., Mutarelli, M., Montefusco, S., Grieco, G., Sepe, L. V., Rossi, B., Nusco, E., Rossignoli, G., Panebianco, G., Merciai, F., Salviati, E., Sommella, E. M., Campiglia, P., Martello, G., Cacchiarelli, D., Medina, D. L., Ballabio, A.

Relation: info:eu-repo/semantics/altIdentifier/pmid/38965447; info:eu-repo/semantics/altIdentifier/wos/WOS:001262224900001; journal:CELL DEATH AND DIFFERENTIATION; https://hdl.handle.net/11577/3528761

الاتاحة: https://hdl.handle.net/11577/3528761
https://doi.org/10.1038/s41418-024-01337-y

المؤلفون: Di Malta C, Zampelli A, Granieri L, Vilardo C, De Cegli R, Cinque L, Nusco E, Pece S, Tosoni D, Sanguedolce F, Sorrentino NC, Merino MJ, Nielsen D, Srinivasan R, Ball MW, Ricketts CJ, Vocke CD, Lang M, Karim B, Lanfrancone L, Schmidt LS, Linehan WM, Ballabio A

المساهمون: C. Di Malta, A. Zampelli, L. Granieri, C. Vilardo, R. De Cegli, L. Cinque, E. Nusco, S. Pece, D. Tosoni, F. Sanguedolce, N. Sorrentino, M. Merino, D. Nielsen, R. Srinivasan, M. Ball, C. Rickett, C. Vocke, M. Lang, B. Karim, L. Lanfrancone, L. Schmidt, W. Linehan, A. Ballabio

مصطلحات موضوعية: BHD, cyst, kidney cancer, TFE3, TFEB, Settore BIOS-10/A - Biologia cellulare e applicata, Settore MEDS-02/A - Patologia generale

Relation: info:eu-repo/semantics/altIdentifier/pmid/36987696; info:eu-repo/semantics/altIdentifier/wos/WOS:000958635200001; volume:15; issue:5; firstpage:1; lastpage:12; numberofpages:12; journal:EMBO MOLECULAR MEDICINE; https://hdl.handle.net/2434/1124317

الاتاحة: https://hdl.handle.net/2434/1124317
https://doi.org/10.15252/emmm.202216877

المؤلفون: Esposito F., Lyubenova H., Tornabene P., Auricchio S., Iuliano A., Nusco E., Merlin S., Olgasi C., Manni G., Gargaro M., Fallarino F., Follenzi A., Auricchio A.

المساهمون: Esposito, F., Lyubenova, H., Tornabene, P., Auricchio, S., Iuliano, A., Nusco, E., Merlin, S., Olgasi, C., Manni, G., Gargaro, M., Fallarino, F., Follenzi, A., Auricchio, A.

مصطلحات موضوعية: AAV vector, haemophilia A, liver gene therapy, protein trans-splicing, Animal, Dependoviru, Genetic Therapy, Genetic Vector, Liver, Mice, Trans-Splicing, Hemophilia A, Inteins

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000789218100001; volume:14; issue:6; journal:EMBO MOLECULAR MEDICINE; https://hdl.handle.net/11563/160587; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85129209306

الاتاحة: https://hdl.handle.net/11563/160587
https://doi.org/10.15252/emmm.202115199

المؤلفون: Sorrentino N. C., Cacace V., De Risi M., Maffia V., Strollo S., Tedesco N., Nusco E., Romagnoli N., Ventrella D., Huang Y., Liu N., Kalled S. L., Choi V. W., De Leonibus E., Fraldi A.

المساهمون: Sorrentino N.C., Cacace V., De Risi M., Maffia V., Strollo S., Tedesco N., Nusco E., Romagnoli N., Ventrella D., Huang Y., Liu N., Kalled S.L., Choi V.W., De Leonibus E., Fraldi A.

مصطلحات موضوعية: Mucopolysaccharidosis IIIA, Sulfamidase, Gene Therapy

وصف الملف: ELETTRONICO

Relation: info:eu-repo/semantics/altIdentifier/pmid/31788497; info:eu-repo/semantics/altIdentifier/wos/WOS:000502573100031; volume:15; firstpage:333; lastpage:342; numberofpages:10; journal:MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT; http://hdl.handle.net/11585/718865; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85075264742; https://www.journals.elsevier.com/molecular-therapy-methods-and-clinical-development/; https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(19)30117-2

الاتاحة: http://hdl.handle.net/11585/718865
https://doi.org/10.1016/j.omtm.2019.10.009
https://www.journals.elsevier.com/molecular-therapy-methods-and-clinical-development/
https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(19)30117-2

المؤلفون: Tornabene P., Ferla R., Llado-Santaeularia M., Centrulo M., Dell'Anno M., Esposito F., Marrocco E., Pone E., Minopoli R., Iodice C., Nusco E., Rossi S., Lyubenova H., Manfredi A., Di Filippo L., Iuliano A., Torella A., Piluso G., Musacchia F., Surace E. M., Cacchiarelli D., Nigro V., Auricchio A.

المساهمون: Tornabene, P., Ferla, R., Llado-Santaeularia, M., Centrulo, M., Dell'Anno, M., Esposito, F., Marrocco, E., Pone, E., Minopoli, R., Iodice, C., Nusco, E., Rossi, S., Lyubenova, H., Manfredi, A., Di Filippo, L., Iuliano, A., Torella, A., Piluso, G., Musacchia, F., Surace, E. M., Cacchiarelli, D., Nigro, V., Auricchio, A.

مصطلحات موضوعية: Animal, CRISPR-Cas System, Genetic Vector, Liver, Mice, Retina, Swine, Dependoviru, Gene Editing

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000782204100014; volume:13; issue:1; firstpage:1963; journal:NATURE COMMUNICATIONS; http://hdl.handle.net/11588/883837; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85128149400

الاتاحة: http://hdl.handle.net/11588/883837
https://doi.org/10.1038/s41467-022-29550-8

المؤلفون: Capuozzo A., Montefusco S., Cacace V., Sofia M., Esposito A., Napolitano G., Nusco E., Polishchuk E., Pizzo M. T., De Risi M., De Leonibus E., Sorrentino N. C., Medina Diego Luis

المساهمون: Capuozzo, A., Montefusco, S., Cacace, V., Sofia, M., Esposito, A., Napolitano, G., Nusco, E., Polishchuk, E., Pizzo, M. T., De Risi, M., De Leonibus, E., Sorrentino, N. C., MEDINA SANABRIA, Diego Luis

مصطلحات موضوعية: autophagy, drug repurposing, fluoxetine, high content imaging, lysosomal exocytosi, lysosomal storage disorder, MPS-IIIA, TFEB, Animal, Disease Models, Fibroblast, Heparitin Sulfate, Hydrolase, Mice, Mucopolysaccharidosis III

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000784369100011; volume:30; issue:4; firstpage:1432; lastpage:1450; numberofpages:19; journal:MOLECULAR THERAPY; http://hdl.handle.net/11588/888656; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85124586011

الاتاحة: http://hdl.handle.net/11588/888656
https://doi.org/10.1016/j.ymthe.2022.01.037

المؤلفون: Calcagni A, Kors L, Verschuren E, De Cegli R, Zampelli N, Nusco E, Confalonieri S, Bertalot G, Pece S, Settembre C, Malouf GG, Leemans JC, de Heer E, Salvatore M, Peters DJM, Di Fiore PP, Ballabio A

المساهمون: Calcagni, A, Kors, L, Verschuren, E, De Cegli, R, Zampelli, N, Nusco, E, Confalonieri, S, Bertalot, G, Pece, S, Settembre, C, Malouf, Gg, Leemans, Jc, de Heer, E, Salvatore, M, Peters, Djm, Di Fiore, Pp, Ballabio, A

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000385162200001; volume:5; firstpage:e1704701; lastpage:e1704726; numberofpages:26; journal:ELIFE; https://hdl.handle.net/11572/361842; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-84988850778; https://elifesciences.org/articles/17047

الاتاحة: https://hdl.handle.net/11572/361842
https://doi.org/10.7554/eLife.17047
https://elifesciences.org/articles/17047

المؤلفون: Tarallo A., Damiano C., Strollo S., Minopoli N., Indrieri A., Polishchuk E., Zappa F., Nusco E., Fecarotta S., Porto C., Coletta M., Iacono R., Moracci M., Polishchuk R., Medina D. L., Imbimbo P., Monti D. M., De Matteis M. A., Parenti G.

المساهمون: Tarallo, A., Damiano, C., Strollo, S., Minopoli, N., Indrieri, A., Polishchuk, E., Zappa, F., Nusco, E., Fecarotta, S., Porto, C., Coletta, M., Iacono, R., Moracci, M., Polishchuk, R., Medina, D. L., Imbimbo, P., Monti, D. M., De Matteis, M. A., Parenti, G.

مصطلحات موضوعية: alpha-glucosidase, enzyme replacement therapy, N-acetylcysteine, oxidative stre, Pompe disease

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000703158100001; volume:13; issue:11; journal:EMBO MOLECULAR MEDICINE; http://hdl.handle.net/11588/861128; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85116291717

الاتاحة: http://hdl.handle.net/11588/861128
https://doi.org/10.15252/emmm.202114434

المؤلفون: Ferla R., Alliegro M., Dell'Anno M., Nusco E., Cullen J. M., Smith S. N., Wolfsberg T. G., O'Donnell P., Wang P., Nguyen A. -D., Chandler R. J., Chen Z., Burgess S. M., Vite C. H., Haskins M. E., Venditti C. P., Auricchio A.

المساهمون: Ferla, R., Alliegro, M., Dell'Anno, M., Nusco, E., Cullen, J. M., Smith, S. N., Wolfsberg, T. G., O'Donnell, P., Wang, P., Nguyen, A. -D., Chandler, R. J., Chen, Z., Burgess, S. M., Vite, C. H., Haskins, M. E., Venditti, C. P., Auricchio, A.

مصطلحات موضوعية: AAV8, age, cancer, gene therapy, insertional mutagenesi, liver, lysosomal storage disease, mouse, MPS VI

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000629246400021; volume:20; firstpage:247; lastpage:257; numberofpages:11; journal:MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT; https://hdl.handle.net/11588/854859; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85098952143

الاتاحة: https://hdl.handle.net/11588/854859
https://doi.org/10.1016/j.omtm.2020.11.015

المؤلفون: Ferriero R, Manco G, Lamantea E, Nusco E, Ferrante MI, Sordino P, Stacpoole PW, Lee B, Zeviani M, BRUNETTI PIERRI, NICOLA

المساهمون: Ferriero, R, Manco, G, Lamantea, E, Nusco, E, Ferrante, Mi, Sordino, P, Stacpoole, Pw, Lee, B, Zeviani, M, BRUNETTI PIERRI, Nicola

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000315922500008; volume:5; firstpage:175ra31; lastpage:175ra31; journal:SCIENCE TRANSLATIONAL MEDICINE; http://hdl.handle.net/11588/575375; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-84875126013

الاتاحة: http://hdl.handle.net/11588/575375
https://doi.org/10.1126/scitranslmed.3004986

المؤلفون: Napolitano G., Di Malta C., Esposito A., de Araujo M. E. G., Pece S., Bertalot G., Matarese M., Benedetti V., Zampelli A., Stasyk T., Siciliano D., Venuta A., Cesana M., Vilardo C., Nusco E., Monfregola J., Calcagni A., Di Fiore P. P., Huber L. A., Ballabio A.

المساهمون: Napolitano, G., Di Malta, C., Esposito, A., de Araujo, M. E. G., Pece, S., Bertalot, G., Matarese, M., Benedetti, V., Zampelli, A., Stasyk, T., Siciliano, D., Venuta, A., Cesana, M., Vilardo, C., Nusco, E., Monfregola, J., Calcagni, A., Di Fiore, P. P., Huber, L. A., Ballabio, A.

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000547610500001; journal:NATURE; http://hdl.handle.net/11588/814557; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85087318149

الاتاحة: http://hdl.handle.net/11588/814557
https://doi.org/10.1038/s41586-020-2444-0

المؤلفون: Monaco A., Maffia V., Sorrentino N. C., Sambri I., Ezhova Y., Giuliano T., Cacace V., Nusco E., De Risi M., De Leonibus E., Schrader T., Klarner F. -G., Bitan G., Fraldi A.

المساهمون: Monaco, A., Maffia, V., Sorrentino, N. C., Sambri, I., Ezhova, Y., Giuliano, T., Cacace, V., Nusco, E., De Risi, M., De Leonibus, E., Schrader, T., Klarner, F. -G., Bitan, G., Fraldi, A.

مصطلحات موضوعية: amyloid aggregation, autophagy, lysosomal storage disease, molecular tweezer, mucopolysaccharidosis type IIIA

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000525341800015; journal:MOLECULAR THERAPY; http://hdl.handle.net/11588/795949; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85079858182

الاتاحة: http://hdl.handle.net/11588/795949
https://doi.org/10.1016/j.ymthe.2020.02.005

المؤلفون: Naso F., Intartaglia D., Falanga D., Soldati C., Polishchuk E., Giamundo G., Tiberi P., Marrocco E., Scudieri P., Di Malta C., Trapani I., Nusco E., Salierno F. G., Surace E. M., Galietta L. J. V., Banfi S., Auricchio A., Ballabio A., Medina D. L., Conte I.

المساهمون: Naso, F., Intartaglia, D., Falanga, D., Soldati, C., Polishchuk, E., Giamundo, G., Tiberi, P., Marrocco, E., Scudieri, P., Di Malta, C., Trapani, I., Nusco, E., Salierno, F. G., Surace, E. M., Galietta, L. J. V., Banfi, S., Auricchio, A., Ballabio, A., Medina, D. L., Conte, I.

مصطلحات موضوعية: autophagy, Ezrin, miR-211, RPE, TFEB

وصف الملف: ELETTRONICO

Relation: info:eu-repo/semantics/altIdentifier/pmid/32154600; info:eu-repo/semantics/altIdentifier/wos/WOS:000526932700009; volume:39; firstpage:e102468; lastpage:e102468; numberofpages:21; journal:EMBO JOURNAL; http://hdl.handle.net/11567/1021645; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85081224643

الاتاحة: http://hdl.handle.net/11567/1021645
https://doi.org/10.15252/embj.2019102468

المؤلفون: Tarallo A., Carissimo A., Gatto F., Nusco E., Toscano A., Musumeci O., Coletta M., Karali M., Acampora E., Damiano C., Minopoli N., Fecarotta S., Della Casa R., Mongini T., Vercelli L., Santoro L., Ruggiero L., Deodato F., Taurisano R., Bembi B., Dardis A., Banfi S., Pijnappel W. P., van der Ploeg A. T., Parenti G.

المساهمون: Tarallo A., Carissimo A., Gatto F., Nusco E., Toscano A., Musumeci O., Coletta M., Karali M., Acampora E., Damiano C., Minopoli N., Fecarotta S., Della Casa R., Mongini T., Vercelli L., Santoro L., Ruggiero L., Deodato F., Taurisano R., Bembi B., Dardis A., Banfi S., Pijnappel W.P., van der Ploeg A.T., Parenti G.

مصطلحات موضوعية: Enzyme replacement therapy, microRNA, miR-133a, Next-generation sequencing, Pompe disease, Adult, Animal, Biomarker, Disease Models, Female, Glycogen Storage Disease Type II, Human, Male, Mice, Knockout, Middle Aged

Relation: info:eu-repo/semantics/altIdentifier/pmid/29997386; info:eu-repo/semantics/altIdentifier/wos/WOS:000460274400014; volume:21; issue:3; firstpage:591; lastpage:600; numberofpages:10; journal:GENETICS IN MEDICINE; http://hdl.handle.net/2318/1786345; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85049685959

الاتاحة: http://hdl.handle.net/2318/1786345
https://doi.org/10.1038/s41436-018-0103-8

المؤلفون: Bartolomeo, R, Cinque, L, De Leonibus, C, Forrester, A, Salzano, A, Monfregola, J, De Gennaro, E, Nusco, E, Azario, I, Lanzara, C, Serafini, M, Levine, B, Ballabio, A, Settembre, C, Bartolomeo R, Cinque L, De Leonibus C, Forrester A, Salzano AC, Monfregola J, De Gennaro E, Nusco E, Azario I, Lanzara C, Serafini M, Levine B, Ballabio A, Settembre C

مصطلحات الفهرس: Lysosomal storage disorder, mTORC1, info:eu-repo/semantics/article

URL: https://hdl.handle.net/10281/459446
info:eu-repo/semantics/altIdentifier/pmid/28872463
info:eu-repo/semantics/altIdentifier/wos/WOS:000412017800019
volume:127
issue:10
firstpage:3717
lastpage:3729
numberofpages:13
journal:THE JOURNAL OF CLINICAL INVESTIGATION

المؤلفون: Bartolomeo R, Cinque L, De Leonibus C, Forrester A, Salzano AC, Monfregola J, De Gennaro E, Nusco E, Azario I, Lanzara C, Serafini M, Levine B, Ballabio A, Settembre C

المساهمون: Bartolomeo, R, Cinque, L, De Leonibus, C, Forrester, A, Salzano, A, Monfregola, J, De Gennaro, E, Nusco, E, Azario, I, Lanzara, C, Serafini, M, Levine, B, Ballabio, A, Settembre, C

مصطلحات موضوعية: Lysosomal storage disorder, mTORC1

وصف الملف: STAMPA

Relation: info:eu-repo/semantics/altIdentifier/pmid/28872463; info:eu-repo/semantics/altIdentifier/wos/WOS:000412017800019; volume:127; issue:10; firstpage:3717; lastpage:3729; numberofpages:13; journal:THE JOURNAL OF CLINICAL INVESTIGATION; https://hdl.handle.net/10281/459446; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85030530713

الاتاحة: https://hdl.handle.net/10281/459446
https://doi.org/10.1172/JCI94130

المؤلفون: Ferla, R, Alliegro, M, Marteau, J, Dell'Anno, M, Nusco, E, Pouillot, S, Zuliani, V, Auricchio, A., GALIMBERTI, STEFANIA, VALSECCHI, MARIA GRAZIA

المساهمون: Ferla, R, Alliegro, M, Marteau, J, Dell'Anno, M, Nusco, E, Pouillot, S, Galimberti, S, Valsecchi, M, Zuliani, V, Auricchio, A

مصطلحات موضوعية: AAV, AAV8, enabling studie, gene therapy, IND, MPSVI, non-clinical safety, Genetic, Molecular Biology, Molecular Medicine

Relation: info:eu-repo/semantics/altIdentifier/pmid/28932756; info:eu-repo/semantics/altIdentifier/wos/WOS:000410660600016; volume:6; firstpage:143; lastpage:158; numberofpages:16; journal:MOLECULAR THERAPY. METHODS & CLINICAL DEVELOPMENT; http://hdl.handle.net/10281/171566; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85029312399; https://www.journals.elsevier.com/molecular-therapy-methods-and-clinical-development/

الاتاحة: http://hdl.handle.net/10281/171566
https://doi.org/10.1016/j.omtm.2017.07.004
https://www.journals.elsevier.com/molecular-therapy-methods-and-clinical-development/

المؤلفون: Di Malta C., Siciliano D., Calcagni A., Monfregola J., Punzi S., Pastore N., Eastes A. N., Davis O., De Cegli R., Zampelli A., Di Giovannantonio L. G., Nusco E., Platt N., Guida A., Ogmundsdottir M. H., Lanfrancone L., Perera R. M., Zoncu R., Pelicci P. G., Settembre C., Ballabio A.

المساهمون: Di Malta, C., Siciliano, D., Calcagni, A., Monfregola, J., Punzi, S., Pastore, N., Eastes, A. N., Davis, O., De Cegli, R., Zampelli, A., Di Giovannantonio, L. G., Nusco, E., Platt, N., Guida, A., Ogmundsdottir, M. H., Lanfrancone, L., Perera, R. M., Zoncu, R., Pelicci, P. G., Settembre, C., Ballabio, A.

Relation: info:eu-repo/semantics/altIdentifier/pmid/28619945; info:eu-repo/semantics/altIdentifier/wos/WOS:000403327400042; volume:356; issue:6343; firstpage:1188; lastpage:1193; numberofpages:6; journal:SCIENCE; https://hdl.handle.net/20.500.11768/164358

الاتاحة: https://hdl.handle.net/20.500.11768/164358
https://doi.org/10.1126/science.aag2553
https://www.science.org/doi/10.1126/science.aag2553

المؤلفون: Soria, L., Allegri, G., Melck, D., Pastore, N., Annunziata, P., Paris, D., Polishchuk, E., Nusco, E., Thöny, B., Motta, A., Häberle, J., Ballabio, A., Brunetti-Pierri, N.

المصدر: Journal of Hepatology ; volume 68, page S413 ; ISSN 0168-8278

الاتاحة: http://dx.doi.org/10.1016/s0168-8278(18)31064-x
https://api.elsevier.com/content/article/PII:S016882781831064X?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S016882781831064X?httpAccept=text/plain

المؤلفون: Ferriero, R., Nusco, E., Cegli, R.D., Carissimo, A., Manco, G., Brunetti-Pierri, N.

المصدر: Journal of Hepatology ; volume 68, page S595-S596 ; ISSN 0168-8278

الاتاحة: http://dx.doi.org/10.1016/s0168-8278(18)31453-3
https://api.elsevier.com/content/article/PII:S0168827818314533?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0168827818314533?httpAccept=text/plain