المؤلفون: Barreca M., Renda M., Spano' V., Montalbano A., Raimondi M. V., Giuffrida S., Bivacqua R., Bandiera T., Galietta L. J. V., Barraja P.

المساهمون: Barreca M., Renda M., Spano' V., Montalbano A., Raimondi M.V., Giuffrida S., Bivacqua R., Bandiera T., Galietta L.J.V., Barraja P.

مصطلحات موضوعية: CFTR correctors, Cystic fibrosis, F508del-CFTR, pyrrolo[3,2-h]quinazolines

Relation: info:eu-repo/semantics/altIdentifier/pmid/39089001; info:eu-repo/semantics/altIdentifier/wos/WOS:001286092500001; volume:276; numberofpages:14; journal:EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY; https://hdl.handle.net/10447/652034

الاتاحة: https://hdl.handle.net/10447/652034
https://doi.org/10.1016/j.ejmech.2024.116691

المؤلفون: Scano M., Benetollo A., Dalla Barba F., Sandona D.

المساهمون: Scano, M., Benetollo, A., Dalla Barba, F., Sandona, D.

مصطلحات موضوعية: Gene therapy, AAV, Small molecules, CFTR correctors, ERAD, LGMDR3, LGMDR4, LGMDR5, LGMDR6, Clinical trials, pre-clinical studies, muscular dystrophy

Relation: info:eu-repo/semantics/altIdentifier/pmid/38713975; info:eu-repo/semantics/altIdentifier/wos/WOS:001238663400001; volume:76; journal:CURRENT OPINION IN PHARMACOLOGY; https://hdl.handle.net/11577/3514122; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85192084419

الاتاحة: https://hdl.handle.net/11577/3514122
https://doi.org/10.1016/j.coph.2024.102459

المؤلفون: Debora Baroni, Naomi Scarano, Alessandra Ludovico, Chiara Brandas, Alice Parodi, Dario Lunaccio, Paola Fossa, Oscar Moran, Elena Cichero, Enrico Millo

المصدر: Pharmaceuticals, Vol 16, Iss 12, p 1702 (2023)

مصطلحات موضوعية: CFTR, CFTR correctors, VX445, VX661, molecular docking, YFP functional assay, Medicine, Pharmacy and materia medica, RS1-441

وصف الملف: electronic resource

Relation: https://www.mdpi.com/1424-8247/16/12/1702; https://doaj.org/toc/1424-8247

URL الوصول: https://doaj.org/article/e98237455bcb4b93b338fd4789aa0e3d

المؤلفون: Roberta Bongiorno, Alessandra Ludovico, Oscar Moran, Debora Baroni

المصدر: International Journal of Molecular Sciences; Volume 24; Issue 16; Pages: 12838

مصطلحات موضوعية: cystic fibrosis transmembrane conductance regulator (CFTR), cystic fibrosis (CF), F508del CFTR, CFTR correctors, CFTR domains

جغرافية الموضوع: agris

وصف الملف: application/pdf

Relation: Molecular Biology; https://dx.doi.org/10.3390/ijms241612838

الاتاحة: https://doi.org/10.3390/ijms241612838

المؤلفون: Xinxiu Fang, Jiunn-Tyng Yeh, Tzyh-Chang Hwang

المصدر: Frontiers in Molecular Biosciences, Vol 9 (2022)

مصطلحات موضوعية: CFTR, premature termination codon, nonsense mutations, CFTR correctors, read-through reagent, Biology (General), QH301-705.5

وصف الملف: electronic resource

Relation: https://www.frontiersin.org/articles/10.3389/fmolb.2022.921680/full; https://doaj.org/toc/2296-889X

URL الوصول: https://doaj.org/article/4e430ee905ab43e39072ecb308e8adb4

المؤلفون: Martina Scano, Alberto Benetollo, Leonardo Nogara, Michela Bondì, Francesco Dalla Barba, Michela Soardi, Sandra Furlan, Akyurek Eylem Emek, Paola Caccin, Marcello Carotti, Roberta Sacchetto, Bert Blaauw, Dorianna Sandonà

المساهمون: Scano, Martina, Benetollo, Alberto, Nogara, Leonardo, Bondì, Michela, DALLA BARBA, Francesco, Soardi, Michela, Furlan, Sandra, Akyurek, EYLEM EMEK, Caccin, Paola, Carotti, Marcello, Sacchetto, Roberta, Blaauw, Bert, Sandonà, Dorianna

مصطلحات موضوعية: folding defective protein, humanized mice, muscle force, sarcoglycanopathy, /small molecules, CFTR correctors, AAV

وصف الملف: ELETTRONICO

Relation: info:eu-repo/semantics/altIdentifier/pmid/34505136; info:eu-repo/semantics/altIdentifier/wos/WOS:000776693700002; volume:31; issue:4; firstpage:499; lastpage:509; numberofpages:11; journal:HUMAN MOLECULAR GENETICS; http://hdl.handle.net/11577/3400586; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85123972583

الاتاحة: http://hdl.handle.net/11577/3400586
https://doi.org/10.1093/hmg/ddab260

المؤلفون: Alyssa Bowling, Alice Eastman, Christian Merlo, Gabrielle Lin, Natalie West, Shivani Patel, Garry Cutting, Neeraj Sharma

المصدر: Journal of Personalized Medicine; Volume 12; Issue 9; Pages: 1448

مصطلحات موضوعية: alternate start, NMD, readthrough, CFTR correctors, CFTR potentiators, transcript stability

وصف الملف: application/pdf

Relation: Pharmacogenetics; https://dx.doi.org/10.3390/jpm12091448

الاتاحة: https://doi.org/10.3390/jpm12091448

المؤلفون: Saint-Criq, Vinciane, Wang, Yiting, Delpiano, Livia, Lin, JinHeng, Sheppard, David N, Gray, Michael A

المصدر: Saint-Criq , V , Wang , Y , Delpiano , L , Lin , J , Sheppard , D N & Gray , M A 2021 , ' Extracellular phosphate enhances the function of F508del-CFTR rescued by CFTR correctors ' , Journal of Cystic Fibrosis , vol. 20 , no. 5 , pp. 843-850 . https://doi.org/10.1016/j.jcf.2021.04.013

مصطلحات موضوعية: F508del-CFTR, CFTR correctors, elexacaftor-tezacaftor-ivacaftor (Trikafta), airway epithelia, phosphate, SLC34A2

وصف الملف: application/pdf

الاتاحة: https://hdl.handle.net/1983/bd6dae53-d035-4504-ae79-38f303b8080c
https://research-information.bris.ac.uk/en/publications/bd6dae53-d035-4504-ae79-38f303b8080c
https://doi.org/10.1016/j.jcf.2021.04.013
https://research-information.bris.ac.uk/ws/files/309543945/PIIS1569199321001235.pdf

المؤلفون: Chiara Brandas, Alessandra Ludovico, Alice Parodi, Oscar Moran, Enrico Millo, Elena Cichero, Debora Baroni

المصدر: Biomolecules; Volume 11; Issue 10; Pages: 1417

مصطلحات موضوعية: cystic fibrosis transmembrane conductance regulator (CFTR), cystic fibrosis, F508del CFTR, CFTR correctors, CFTR domains, aminoarylthiazoles

جغرافية الموضوع: agris

وصف الملف: application/pdf

Relation: Chemical Biology; https://dx.doi.org/10.3390/biom11101417

الاتاحة: https://doi.org/10.3390/biom11101417

المؤلفون: Carotti, Marcello, Scano, Martina, Fancello, Irene, Richard, Isabelle, Risato, Giovanni, Bensalah, Mona, Soardi, Michela, Sandonà, Dorianna

المساهمون: Università degli Studi di Padova = University of Padua (Unipd), Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris-Saclay-Généthon, Centre de recherche en Myologie – U974 SU-INSERM, Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)

المصدر: ISSN: 1661-6596.

مصطلحات موضوعية: protein folding correctors, sarcoglycanopathy, myogenic cells, myotubes, folding-defective proteins, proteostasis regulators, CFTR correctors, [SDV.SP.PHARMA]Life Sciences [q-bio]/Pharmaceutical sciences/Pharmacology

Relation: info:eu-repo/semantics/altIdentifier/pmid/32155735; PUBMED: 32155735; PUBMEDCENTRAL: PMC7084537

الاتاحة: https://hal.sorbonne-universite.fr/hal-02556539
https://hal.sorbonne-universite.fr/hal-02556539v1/document
https://hal.sorbonne-universite.fr/hal-02556539v1/file/ijms-21-01813.pdf
https://doi.org/10.3390/ijms21051813

المؤلفون: Anna Esposito, Daniele D’Alonzo, Maria De Fenza, Eliana De Gregorio, Anna Tamanini, Giuseppe Lippi, Maria Cristina Dechecchi, Annalisa Guaragna

المصدر: International Journal of Molecular Sciences; Volume 21; Issue 9; Pages: 3353

مصطلحات موضوعية: cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR), iminosugars, miglustat, glycomimetics, glycosidase inhibitors, CFTR correctors, anti-inflammatory agents

جغرافية الموضوع: agris

وصف الملف: application/pdf

Relation: Molecular Pathology, Diagnostics, and Therapeutics; https://dx.doi.org/10.3390/ijms21093353

الاتاحة: https://doi.org/10.3390/ijms21093353

المؤلفون: Giulia Amico, Chiara Brandas, Oscar Moran, Debora Baroni

المصدر: International Journal of Molecular Sciences; Volume 20; Issue 21; Pages: 5463

مصطلحات موضوعية: cystic fibrosis transmembrane conductance regulator (CFTR), cystic fibrosis, F508del-CFTR, CFTR-correctors, CFTR halves, transmembrane-nucleotide binding domains

جغرافية الموضوع: agris

وصف الملف: application/pdf

Relation: Molecular Pathology, Diagnostics, and Therapeutics; https://dx.doi.org/10.3390/ijms20215463

الاتاحة: https://doi.org/10.3390/ijms20215463

المؤلفون: Christian Borgo, Claudio D'Amore, Mauro Salvi

المصدر: Journal of Cystic Fibrosis. 20:891-894

مصطلحات موضوعية: CFTR correctors, F508del-CFTR, mutagenesis, Post-translational modifications, Regulatory insertion region, 0301 basic medicine, Pulmonary and Respiratory Medicine, Functional role, congenital, hereditary, and neonatal diseases and abnormalities, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Mutagenesis (molecular biology technique), Endoplasmic Reticulum, medicine.disease_cause, Cystic fibrosis, Cell Line, 03 medical and health sciences, 0302 clinical medicine, Ubiquitin, medicine, Humans, Phosphorylation, Mutation, biology, business.industry, Endoplasmic reticulum, Methylation, respiratory system, medicine.disease, digestive system diseases, respiratory tract diseases, Cell biology, 030104 developmental biology, 030228 respiratory system, Pediatrics, Perinatology and Child Health, biology.protein, business, Protein Processing, Post-Translational

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::24adef1af32b21814d9e199a1de6e25b
https://doi.org/10.1016/j.jcf.2021.03.010

المؤلفون: Westphal, Anna Philine

المساهمون: Banac, Srđan

مصطلحات موضوعية: BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti, BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences, CFTR modulators, Cystic Fibrosis, Ivacaftor, G551D mutation, F508del mutation, CFTR correctors, CFTR potentiators

وصف الملف: application/pdf

Relation: https://www.unirepository.svkri.uniri.hr/islandora/object/medri:7816; https://urn.nsk.hr/urn:nbn:hr:184:380526; https://www.unirepository.svkri.uniri.hr/islandora/object/medri:7816/datastream/PDF

الاتاحة: https://www.unirepository.svkri.uniri.hr/islandora/object/medri:7816
https://urn.nsk.hr/urn:nbn:hr:184:380526
https://www.unirepository.svkri.uniri.hr/islandora/object/medri:7816/datastream/PDF

المؤلفون: Li, Hongyu, Pesce, Emanuela, Sheppard, David N., Singh, Ashvani K., Pedemonte, Nicoletta

المصدر: Li , H , Pesce , E , Sheppard , D N , Singh , A K & Pedemonte , N 2018 , ' Therapeutic approaches to CFTR dysfunction : from discovery to drug development ' , Journal of Cystic Fibrosis , vol. 17 , no. 2 , pp. S14-S21 . https://doi.org/10.1016/j.jcf.2017.08.013

مصطلحات موضوعية: CFTR CI-channel, F508del-CFTR, CFTR correctors, Pharmacological chaperones, Proteostasis regulators, Artificial anion transporters (anionophores)

وصف الملف: application/pdf

Relation: https://research-information.bris.ac.uk/en/publications/26d0819c-4f21-4931-91b6-df23c6af5b4b

الاتاحة: https://hdl.handle.net/1983/26d0819c-4f21-4931-91b6-df23c6af5b4b
https://research-information.bris.ac.uk/en/publications/26d0819c-4f21-4931-91b6-df23c6af5b4b
https://doi.org/10.1016/j.jcf.2017.08.013
https://research-information.bris.ac.uk/ws/files/127424547/Li_et_al_J_Cyst_Fibros_2017_accepted.pdf
http://www.scopus.com/inward/record.url?scp=85029173090&partnerID=8YFLogxK

المؤلفون: Michela Bondì, Roberta Sacchetto, Marcello Carotti, Alberto Benetollo, Dorianna Sandonà, Bert Blaauw, Martina Scano, Eylem Emek Akyurek, Michela Soardi, Sandra Furlan, Paola Caccin, Leonardo Nogara, Francesco Dalla Barba

مصطلحات موضوعية: folding defective protein, humanized mice, muscle force, sarcoglycanopathy, /small molecules, CFTR correctors, AAV, Cystic Fibrosis, Protein subunit, small molecules, Cystic Fibrosis Transmembrane Conductance Regulator, Biology, Muscular Dystrophies, Mice, Dystrophin-associated protein complex, Sarcoglycans, Genetics, medicine, Animals, sarcoglycanopathy, Muscular dystrophy, Muscle, Skeletal, Molecular Biology, Genetics (clinical), Loss function, SGCA, Sarcolemma, CFTR correctors, AAV, General Medicine, medicine.disease, Transmembrane protein, Cell biology, humanized mice, Muscular Dystrophies, Limb-Girdle, muscle force, medicine.symptom, Muscle contraction

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5db33b7e45a43a1f7e1b4fbfecbf0adb
http://hdl.handle.net/11577/3400586

المؤلفون: Xinxiu Fang, Jiunn-Tyng Yeh, Tzyh-Chang Hwang

مصطلحات موضوعية: Biochemistry, Molecular Biology, Structural Biology, Enzymes, Protein Trafficking, Proteomics and Intermolecular Interactions (excl. Medical Proteomics), Receptors and Membrane Biology, Signal Transduction, Structural Biology (incl. Macromolecular Modelling), Synthetic Biology, CFTR, premature termination codon, nonsense mutations, CFTR correctors, read-through reagent

Relation: https://figshare.com/articles/figure/Image2_Pharmacological_Responses_of_the_G542X-CFTR_to_CFTR_Modulators_tiff/20140454

الاتاحة: https://doi.org/10.3389/fmolb.2022.921680.s002
https://figshare.com/articles/figure/Image2_Pharmacological_Responses_of_the_G542X-CFTR_to_CFTR_Modulators_tiff/20140454

المؤلفون: Anna Carbone, Virginia Spanò, Alessandra Montalbano, Paola Barraja, Luis J. V. Galietta, Paolo Scudieri

المساهمون: Spanò, Virginia, Montalbano, Alessandra, Carbone, Anna, Scudieri, Paolo, Galietta, Luis J V, Barraja, Paola, Spano, V., Montalbano, A., Carbone, A., Scudieri, P., Galietta, L. J. V., Barraja, P.

المصدر: European Journal of Medicinal Chemistry. 180:430-448

مصطلحات موضوعية: Protein Folding, Cystic Fibrosis, CFTR corrector, Mutant, Cystic Fibrosis Transmembrane Conductance Regulator, Pyrimidinones, medicine.disease_cause, 01 natural sciences, F508del-CFTR, 03 medical and health sciences, Mutant protein, Drug Discovery, medicine, Animals, Humans, CFTR, 030304 developmental biology, Pharmacology, 0303 health sciences, Mutation, CFTR correctors, biology, 010405 organic chemistry, Chemistry, Organic Chemistry, Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, Thiazoles, General Medicine, Settore CHIM/08 - Chimica Farmaceutica, Small molecule, 0104 chemical sciences, Cell biology, Mechanism of action, Cystic fibrosi, biology.protein, medicine.symptom, Protein A, δf508 cftr

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fc8a48cdfdc7f00633304d59ca348553
https://doi.org/10.1016/j.ejmech.2019.07.037

المؤلفون: Ugo, Carraro, Zipora, Yablonka-Reuveni

المصدر: European Journal of Translational Myology

مصطلحات موضوعية: Simvastatin, muscle, sarcoplasmic reticulum (SR), dynamic MRI, muscle oxygen saturation, Euganean Thermal District, collagen type III, EMG, Essential, motor unit, immunodeficient mouse hosts, pericyte, CD82, satellite cell, caspase 3, complementary medicine, IUR, FOP, Padua Muscle Days, motoneuron, mono-galactosyldiacylglycerols, resting force, Motor Units, exopolysaccharides, novel mouse models, resident cells, mitochondrial adaptations, live imaging, instrumental evaluation, endothelial cells, macrophages, loading, high-fat diet, spa therapy, diaphragm, CRISPR, ichthyosis, fiber aligned strains, muscle growth, hypertrophy, HDEMG, biological interpretation of miRNA dysregulation, cranial myogenesis, Article, molecular regulation, hyaluronan, host gene, proteomics, Histone Deacetylase, GHSR-1a, autocrine signaling, Machine learning, unloading, Mobility Medicine, mechanomyography, muscle hypertrophy, skeletal muscle, intramuscular connective tissue, dynamic contractions, nestin-GFP, mechanotransduction, Decomposition, denervation, Muscle strength, mitochondrial control of cytosolic calcium, Physical exercise, Convalescence, T1 mapping, firing rate, Parkinson’s disease, cell therapy, muscle spindle, myofibers, collagen, muscle atrophy, tendon, dysphagia, Balneotherapy, 2-deoxy-ATP, hemoglobin amount, mitochondrial Ca2+ uptake, Pax3/7, respiratory function, motor control, oxidative stress, compressed sensing, finite element model, endurance, satellite cells, HMGB1, sarcomere length, training, CFTR correctors, micro-dystrophin, aggregation, health, Tetraspanin, Nuclear misplacement, Mitochondria, cell tracking, Differentiation, biomarker, sarcoglycanopathies, musculoskeletal disorders, ageing research, CT, muscular dystrophy, Therapeutic Physical Agent, tumor, pool size, systolic function, Pink1-Parkin, holding isometric muscle function, facioscapulohumeral muscular dystrophy, contracture, Cyanobacteria, cachexia, uremic myopathy, HDsEMG decomposition, Translational Myology and Mobility Medicine, stem cell proliferation, Functional Electrical Stimulation, aging, fibrosis, COVID-19, hypo-gravity, neuromuscualr junction, muscle regeneration and repair, stem cell, inflammation, ageing, plasticity, eccentric exercise, stereology, activin A, sarcomere, hyper-gravity, Duchenne muscular dystrophy cardiomyopathy, sarcomere operating length, recruitment threshold, chronic kidney disease, Aging muscle, Follistatin, isometric muscle action, macromolecular fraction content, Lipid disorder, adaptation, Neural Drive, Chanarin-Dorfman Syndrome, 2021 PDM3 Program & Abstracts, calcium entry unit (CEU), transcriptomics, growth hormone secretagogues, Rhabdomyosarcoma, Glycogenosis type 2, dystrophic mouse model of MΦ depletion, Adaptive Force, dental practice, muscle biomechanics, reactive oxygen species, excitation-contraction (EC) coupling, exercise, Historical, ultrasound, PABPN1, Myogenesis, Parvalbumin knock out mice, aerobic metabolism, symmetric/asymmetric cell division, Iontophoresis, proteomic analysis, Sertoli cell, gene therapy, ERK signaling, TCM, Cholesterol, nutrition, Myogenin-Tomato KI mouse, SRF, prevention and rehabilitation, strength, Pulsed-Shortwave Diathermy, force, cancer cachexia, Duchenne muscular dystrophy, Novel mechanisms, long-term denervated human muscles, masticatory muscles, nuclear positioning, apical, fiber death, muscle stem cell heterogeneity, mud therapy, doxorubicin, atrophy, Transcutaneous auricular-nerve stimulation, Galvanic Current, rehabilitation exercisetraining, quiescence, human, FoxP1, anti-inflammatory bioactive molecules, cerebral palsy, oxidative modifications, skeletal muscle ultrastructure, Pax3, Health resort medicine, muscle quality tissue, Ca2+ entry unit (CEU), mitochondrial permeability transition pore, Electron Microscopy (E.M.), muscle stem cells, balneology, fibro/ adipogenic progenitors, modulus, regeneration, Padua Days, caveolae, Crosstalk between MΦs, muscular dystrophies, SREBP2, permeability, senescence, TNPO3, PGC-1α, 3D strain imaging, Physical Agents, chemotherapy, network physiology, nutritional intervention, muscle damage, high density electromyography, basal, lipid metabolism, blood flow, connective tissue, mass spectrometry, deltoid muscle, muscle regeneration, active force, alkaline Glauber's salt, AAV, ultrastructure, IGF-I, SRSF1, E3 ubiquitin ligase, liver involvement, olfaction, myopathy, sampling, extracellular matrix, peloidotherapy, collagen type I, histological study, sarcomerogenesis, muscle differentiation, extraocular muscle, fascicle length, heat-stroke, TGFβ, Oculopharyngeal Muscular Dystrophy, store-operated Ca2+ entry (SOCE), external ear, cancer, myosin activator, corticomuscular coherence, ASB2, AAV-micro-dystrophin, Contractile Speed, skeletal muscle fibers, epigenetics, conventional Western medicine, muscle wasting, fibro/adipocyte, bilateral motor task, calcium deregulation, elastic fiber, drop-jump, heterotopic ossification, dissector, neural connectivity, soft tissues, perimysium

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid________::bd70c8062b0ec8cff97f6ccfd24aaed9
https://pubmed.ncbi.nlm.nih.gov/33733717

المؤلفون: HATIPOGLU, BESTE

المساهمون: SANDONA', DORIANNA

مصطلحات موضوعية: ERAD-pathway, CFTR correctors, Target validation, Mass spectrometry, Click chemistry

وصف الملف: application/pdf

Relation: Dipartimento di Scienze del Farmaco - DSF; PHARMACEUTICAL BIOTECHNOLOGIES - BIOTECNOLOGIE FARMACEUTICHE Laurea Magistrale (D.M. 270/2004); 2023; https://hdl.handle.net/20.500.12608/80650

الاتاحة: https://hdl.handle.net/20.500.12608/80650