| Conference poster |
- Rodrigues, Cláudia S.; Pankonien, Ines; Railean, Violeta; Amaral, Margarida. "Does CFTR Play a Role of CFTR in Epithelial
Differentiation, EMT and Cancer?". Paper presented in 20th ECFS Basic Science Conference. Lido di Camaiore (Italy) 2025,
2025.
- Rodrigues, Cláudia S.; Pankonien, Ines; Railean, Violeta; Coelho, Isabel; Amaral, Margarida. "Theranostics for People with
Cystic Fibrosis: p.Ala561Glu-CFTR is rescued by elexacaftor/tezacaftor/ivacaftor.". Paper presented in 20th ECFS Basic
Science Conference. Lido di Camaiore (Italy) 2025, 2025.
- Caleiro, Mariana F.; Daniela Neugebauer; ABDUL, SHAZIA; Michou, Ioanna; Pacheco, Rita; Margarida D. Amaral; Botelho, Hugo
M. "Rescue of p.Phe508del-CFTR through inhibition of the kinase GRK5: identification of proteins and small molecule mediators".
Paper presented in 20th ECFS Basic Science Conference - European Cystic Fibrosis Society, 2025.
- Caleiro, Mariana F.; Botelho, Hugo M; Margarida D. Amaral; Railean, Violeta. "Inhibition of GRK5 with CCG-273441 as a potential
novel strategy to restore p.Phe508del-CFTR traffic". Paper presented in 4th Chem & Biochem Students Meeting, 2024.
- Rodrigues, Cláudia S.; Railean, Violeta; Pankonien, Ines; Margarida D. Amaral. "Theranostics for People with Cystic Fibrosis
and Rare CFTR Variants". Paper presented in 47th ECFS Conference, 2024.
- Rodrigues, Cláudia S.; Pankonien, Ines; Railean, Violeta; Margarida D. Amaral. "Role of CFTR in Airway Epithelial Differentiation
and Regeneration: Studies from Novel Human Basal Cell Lines". Paper presented in 47th ECFS Conference, 2024.
- Daniela Neugebauer; Mariana Caleiro; Vasco Cachatra; Moiteiro, Cristina; Amaral, Margarida; Botelho, Hugo M. "High content
screening for the characterization of the GRK5-dependent p.Phe508del-CFTR rescue in cystic fibrosis". Paper presented in Spanish
& Portuguese Advanced Optical Microscopy Meeting – SPAOM2023, 2023.
- Daniela Neugebauer; Vasco Cachatra; Moiteiro, Cristina; Amaral, Margarida; Botelho, Hugo M. "Identification of genes that
restore p.Phe508del-CFTR traffic in cystic fibrosis models through GRK5 inhibition". Paper presented in Chem&BioChem –
Postgraduate Students Meeting, DQB/FCUL, 2023.
- Torres, Raquel; Amaral, Margarida; Vukosava M Torres; Pankonien, Ines. "Exploring Epithelial Mesenchymal-Transition (EMT)-associated
factors in Cystic Fibrosis". Paper presented in Oral and Poster presentation at the 3rd Chem & BioChem Students Meeting,
Faculty of Sciences, University Lisboa, Portugal, 2023.
- Reis Joao; Pankonien, Ines; Amaral, Margarida. "The Role of CFTR in Intestinal Epithelial Dilerentiation". Paper presented
in Oral and Poster presentation at the 3rd Chem & BioChem Students Meeting, Faculty of Sciences, University Lisboa, Portugal,
2023.
- Pankonien, Ines; Cláudia Rodrigues; Filipa B. Simões; Santos, Lúcia; Carlos M. Farinha; Margarida D. Amaral. "Role of CFTR
in airway epithelial regeneration and repair: studies from novel human basal cell lines". Paper presented in 18th ECFS
Basic Science Conference, 2023.
- Silva, Iris AL; S. Ramalho, Sofia; Rodrigues, Cláudia S.; Railean, Violeta; Carlos M. Farinha; Margarida D. Amaral. "Functional
Characterization of Rare CFTR Mutations and Response to Modulators is Best Assessed in Both Cellular and Patient-derived Models".
Paper presented in Ciências Research Day, FCUL, 2022.
- Silva, Iris AL; Railean, Violeta; Rodrigues, Cláudia S.; Margarida D. Amaral. "Personalized Medicine for Cystic Fibrosis:
Finding Therapies for all Using Patient-Derived 3D Intestinal Organoids". Paper presented in Innovative 3D-models for understanding
mechanisms underlying lung diseases: powerful tools for translational research Conference, 2022.
- Rodrigues, Cláudia S.; Silva, Iris A. L.; S. Ramalho, Sofia; Carlos M. Farinha; Margarida D. Amaral. "A rare mutation found
in Portuguese individuals with Cystic Fibrosis (I148N) responds to approved CFTR modulators". Paper presented in 17th ECFS
Basic Science Conference, 2022.
- Ferreira, JF; Silva, Iris AL; Botelho, Hugo M; Amaral, Margarida; Farinha, CM. "Exploring the role of EPAC1 to stabilize CFTR
in intestinal cells". Paper presented in 17th ECFS Basic Science Conference, 2022.
- Botelho, Hugo M; Lopes-Pacheco, Miquéias; Pinto, Madalena C.; Silva, Iris AL; Clarke, Luka; Cachatra, V; Neumann, B; et al.
"New kinase therapeutic targets for Cystic Fibrosis from a global functional genomics screen". Paper presented in 17th
ECFS Basic Science Conference, 2022.
- Pankonien, Ines; Quaresma, Margarida C; Rodrigues, Cláudia S.; Amaral, Margarida. "Identification of genes linking CFTR to
airway epithelial regeneration". Paper presented in 17th ECFS Basic Science Conference, 2022.
- Rodrigues, Cláudia S.; Pankonien, Ines; Amaral, Margarida. "Role of CFTR in Airway Epithelial Polarization and Differentiation".
Paper presented in 2nd Chem & BioChem Students Meeting, 2022.
- S. Ramalho, Sofia; Santos, Lúcia; Margarida D. Amaral; André O. Falcão; Carlos M. Farinha. "Exploring the endoplasmic reticulum
retention mechanisms to rescue the rare CFTR mutation N1303K". Paper presented in 17th ECFS Basic Science Conference,
2022.
- Rodrigues, Cláudia S.; Silva, Iris A.L; Railean, Violeta; Pankonien, Ines; Amaral, Margarida. "Personalised Treatment For
Cystic Fibrosis Patients With Rare CFTR Mutations". Paper presented in 2nd International Meeting of the Portuguese Society
of Physiology, 2022.
- Filipa Castela Ferreira; Mafalda Bacalhau; Margarida D. Amaral; Miquéias Lopes-Pacheco. "Identification of Corrector Combinations
among FDA-Approved Drugs to Increase the Rescue of F508del-CFTR Traffic and Function". Paper presented in 5ª Conferência
da redeSAÚDE, 2021.
- Silva, Iris AL; Duarte, Aires; Marson, Fernando; Centeio, Raquel; Dousova, Tereza; Kunzelmann, Karl; Amaral, Margarida. "Electrophysiological
Measurements In Rectal Biopsies: A Better Prognosis Biomarker For CF Disease?". Paper presented in North American Cystic
Fibrosis Conference 2020, 2020.
- Silva, Iris AL; Ramalho, Anabela S; Vonk, Annelotte M; Suen, Sylvia WF; Bierlaagh, M; Amaral, Margarida; Vermeulen, François;
et al. "First Results Of The HIT-CF Organoid Study: Intestinal Organoids Derived From Cf Patients With Ultra-Rare Mutations
Respond To The Combination Of Dirocaftor And Posenacaftor". Paper presented in North American Cystic Fibrosis Conference
2020, 2020.
- Ramalho, AS; Vonk, AM; Silva, Iris AL; Botelho, Hugo M; Bor, R; van Mourik, P; Michel, S; et al. "High reproducibility of
forskolin-induced swelling of intestinal organoids across three academic laboratories". Paper presented in 33rd Annual
North American Cystic Fibrosis Conference, 2019.
- Matos, Ana Margarida; Pinto, Francisco; Barros, Patrícia; Amaral, Margarida; Pepperkok, Rainer; Matos, Paulo. "Plasma membrane-specific
interactome analysis reveals calpain 1 as a druggable modulator of rescued Phe508del-CFTR cell surface stability". Paper presented
in SINAL 2019 ¿ 10th Meeting on Signal Transduction, 2019.
10.1109/elektro.2016.7512136
- Astapenko, D; Botelho, Hugo M; Kolodkin, A; Westerhoff, HV; Amaral, Margarida. "Dynamic modelling of CFTR channel maturation
in Cystic Fibrosis: what controls the channel concentration in the plasma membrane?". Paper presented in The 11th International
Conference on Bioinformatics of Genome Regulation and Structure\Systems Biology, 2018.
- Quaresma, MC; Botelho, Hugo M; Pankonien, I; Pepperkok, R; Amaral, Margarida; Quaresma, Margarida C. "Role of CFTR in epithelial-mesenchymal
transition (EMT) by functional genomics". Paper presented in Encontro Ciência 2018, 2018.
- Lopes-Pacheco, M; Botelho, Hugo M; Amaral, Margarida. "Novel F508del-CFTR traffic correctors among FDA-approved drugs and
mechanism of action of RDR01752 corrector". Paper presented in EMBO Workshop: Proteostasis, 2017.
- Botelho, Hugo M; Lim, SH; Tischer, C; Pepperkok, R; Stagljar, I; Amaral, Margarida. "Validation and classification of genes
releasing misfolded F508del-CFTR from the endoplasmic reticulum". Paper presented in EMBO Workshop: Proteostasis, 2017.
- Pinto, MC; Lérias, JR; Botelho, Hugo M; Awatade, NT; Pepperkok, R; Kunzelmann, K; Amaral, Margarida. "Anoctamin 1 – Identification
of novel regulators and their significance to Cystic Fibrosis". Paper presented in 31st Annual North American Cystic Fibrosis
Conference, 2017.
- Awatade, NT; Felício, V; Ramalho, S; Botelho, Hugo M; Silva, Iris AL; de Poel, E; Vonk, AM; et al. "Functional and biochemical
assessment of correctors and potentiators on the rare Cystic Fibrosis-causing R560S mutation". Paper presented in 31st
Annual North American Cystic Fibrosis Conference, 2017.
- Canato, S; Carvalho, AS; Botelho, Hugo M; Aloria, K; Matthiesen, Rune; Amaral, Margarida; Falcão, AO; Farinha, CM. "Modulation
of protein traffic networks to rescue F508del-CFTR from the endoplasmic reticulum". Paper presented in 40th European Cystic
Fibrosis Conference, 2017.
- Baptista, C; Botelho, Hugo M; Tenreiro, RP; Awatade, NT; Amaral, Margarida; Vieira, H. "From Portuguese natural heritage to
Cystic Fibrosis therapies – a story of nature and technology for pharmaceutical applications". Paper presented in 3rd Annual
Conference Rede Mar, 2017.
- Pinto, MC; Lérias, JR; Botelho, Hugo M; Awatade, NT; Pepperkok, P; Kunzelmann, K; Amaral, Margarida. "Anoctamins 1 and 6 –
Identification of novel traffic regulators and their significance to Cystic Fibrosis". Paper presented in 14th ECFS Basic
Science Conference, 2017.
- Loureiro, Claudia A.; Matos, Ana Margarida; Dias-Alves, Ângela; Pereira, Joana F; Uliyakina, Inna; Barros, Patrícia; Amaral,
Margarida; Matos, Paulo. "A NHERF1 PDZ domain switch averts CHIP Ub ligase-mediated peripheral membrane quality control of
misfolded CFTR". Paper presented in European Cystic Fibrosis Society, 14th ECFS Basic Science Conference, 2017.
- Quaresma, MC; Botelho, Hugo M; Amaral, Margarida. "Role of CFTR in the maintenance of airway epithelial cell differentiation".
Paper presented in 14th ECFS Basic Science Conference, 2017.
- Lérias, JR; Pinto, MC; Botelho, Hugo M; Schreiber, R; Amaral, Margarida; Kunzelmann, K. "Anoctamin 1 traffic and function
is enhanced by tethering proteins family of extended synaptotagmins". Paper presented in 14th ECFS Basic Science Conference,
2017.
- Igreja, S; Botelho, Hugo M; Felício, V; Pagani, G; Pagani, M; Amaral, Margarida. "Validation of a CFTR alternative splicing
reporter system for high-throughput microscopy and identification of novel CFTR regulators of alternative splicing". Paper
presented in 14th ECFS Basic Science Conference, 2017.
- Awatade, NT; Botelho, Hugo M; Felício, V; Clarke, LA; Silva, Iris AL; Marques, Luis; Ramos, M; Amaral, Margarida. "CFTR modulators
enhance function R334W-CFTR both in intestinal organoids and conditionally reprogrammed human nasal epithelial cells". Paper
presented in 14th ECFS Basic Science Conference, 2017.
- Felício, V; Botelho, Hugo M; Igreja, S; Amaral, Margarida. "A high-throughput screening assay to identify factors correcting
CFTR mutations bearing premature termination codons (PTCs)". Paper presented in 14th ECFS Basic Science Conference,
2017.
- Baptista, C; Vieira, H; Tenreiro, R; Awatade, NT; Amaral, Margarida; Botelho, Hugo M. "High throughput microscopy screens
identify F508del-CFTR correctors and ANO1 traffic modulators in a unique Portuguese natural compound library". Paper presented
in 14th ECFS Basic Science Conference, 2017.
- Botelho, Hugo M; Lim, SH; Tischer, C; Pepperkok, R; Stagljar, I; Amaral, Margarida. "A high content siRNA screen at near genomic
scale reveals novel F508del-CFTR therapeutic targets". Paper presented in 14th ECFS Basic Science Conference, 2017.
- Botelho, Hugo M; Quaresma, MC; Amaral, Margarida. "CFTR in the maintenance of epithelial differentiation in the airways".
Paper presented in Chem&BioChem Postgraduate Students Meeting, 2017.
- Santos-Sousa, Luis; Pankonien, Ines; Chanson, Marc; Amaral, Margarida. "KLF4 in differentiation and CFTR trafficking". Paper
presented in PhD BioSys Retreat, Beja, Portugal, 2017.
- Pankonien, Ines; Pepperkok, Rainer; Amaral, Margarida. "Validation of an automated live-cell wound healing assay to identify
genes connecting CFTR traffic with epithelia differentiation". Paper presented in 15th ECFS Basic Science Conference Albufeira,
Portugal, 2017.
- Luís Santos-Sousa; Pankonien, Ines; Chanson, Marc; Amaral, Margarida. "Establishment of a model for assessing CFTR and KLF4
roles in epithelial differentiation". Paper presented in Chem&Biochem Postgraduate Students Meeting, Lisbon, Portugal,
2017.
- Igreja, S; Botelho, Hugo M; Amaral, Margarida. "Identification of novel regulators of alternative splicing by HT microscopy:
implications for Cystic Fibrosis and other human diseases". Paper presented in EMBO Practical Course: High-Throughput Microscopy
for Systems Biology, 2016.
- Pankonien, I; Santos-Sousa, L; Botelho, Hugo M; Amaral, Margarida. "Establishment of cell-based assays of airway epithelial
differentiation and wound healing for automated microscopy". Paper presented in 13th ECFS Basic Science Conference,
2016.
- Awatade, NT; Botelho, Hugo M; Felício, V; Clarke, LA; Ramos, M; Amaral, Margarida. "To assess the efficacy of CFTR modulators
on rare Cystic Fibrosis mutations using forskolin-induced swelling assay in human intestinal organoids". Paper presented in
13th ECFS Basic Science Conference, 2016.
- Awatade, NT; Botelho, Hugo M; Felício, V; Clarke, LA; Ramos, M; Amaral, Margarida. "Assessment of CFTR activity ex vivo as
a basis for personalised medicine". Paper presented in 5th Workshop Structure and Function of Ion Channels and Transporters,
2015.
- Matos, Ana Margarida; Pereira, Joana F; Uliyakina, Inna; Amaral, Margarida; Matos, Paulo. "Anchoring to the actin-cytoskeleton
is not essential to retain functional F508del-CFTR at the plasma membrane". Paper presented in European Cystic Fibrosis
Society, 12th ECFS Basic Science Conference, 2015.
- Igreja, S; Botelho, Hugo M; Marques, Luis; Amaral, Margarida. "In vitro correction of the CFTR 2657+5G>A splicing mutation
by an antisense oligonucleotide". Paper presented in 12th ECFS Basic Science Conference, 2015.
- Botelho, Hugo M; Dahimène, S; Uliyakina, I; Awatade, NT; Proença, MC; Neumann, B; Tischer, C; et al. "Rescuing F508del-CFTR
in airway epithelial cells using a novel traffic-based high content microscopy screening platform". Paper presented in 12th
ECFS Basic Science Conference, 2015.
- Botelho, Hugo M; Dahimène, S; Uliyakina, I; Neumann, B; Tischer, C; Pepperkok, R; Amaral, Margarida. "A high content screening
approach for the identification of CFTR traffic regulators in airway epithelial cells". Paper presented in ESF/EMBO Cell
Polarity and Membrane Traffickin, 2014.
- Botelho, Hugo M; Dahimène, S; Uliyakina, I; Neumann, B; Tischer, C; Pepperkok, R; Amaral, Margarida. "High throughput screen
reveals genes regulating wt-CFTR and rescuing F508del-CFTR traffic". Paper presented in 27th Annual North American Cystic
Fibrosis Conference, 2013.
- Botelho, Hugo M; Dahimène, S; Uliyakina, I; Neumann, B; Tischer, C; Pepperkok, R; Amaral, Margarida. "Identification of CFTR
traffic regulators as a source of novel Cystic Fibrosis therapeutic targets". Paper presented in The 5th EMBO Meeting,
2013.
- Inna Uliyakina; Ana C Da Paula; Marisa Sousa; Verónica Felício; Sara Afonso; Anabela S. Ramalho; Margarida D. Amaral. "Effect
of genetic revertants and small molecules on maturation and function of CFTR without regulatory insertion and regulatory extension.".
Paper presented in FEBS congress 2013, 2013.
- Roxo Rosa, Mónica; Amaral, Margarida; Penque, Deborah. "Comparative Proteome Analysis of CHO Cells Expressing Normal or F508del
CFTR Protein.". Paper presented in 24th European Cystic Fibrosis Conference, June, Viena (Austria), P12., 2001.
|
| Journal article |
- Margarida D. Amaral; Ines Pankonien. "Cystic fibrosis as a paradigmatic disease in bringing science to the bedside". FEBS
Letters (2025): https://doi.org/10.1002/1873-3468.70101.
10.1002/1873-3468.70101
- Catarina Pereira; Margarida D. Amaral; Andre O. Falcao. "CyFidb: A Molecular Atlas for Cystic Fibrosis". Journal of Cystic
Fibrosis (2025): https://doi.org/10.1016/j.jcf.2025.03.011.
10.1016/j.jcf.2025.03.011
- Cláudia S Rodrigues; Violeta Railean; Sofia S Ramalho; Carlos M Farinha; Ines Pankonien; Margarida D Amaral. "Personalized
therapy with CFTR modulators: Response of p.Ile148Asn variant". Journal of Cystic Fibrosis (2025): https://doi.org/10.1016/j.jcf.2025.01.015.
10.1016/j.jcf.2025.01.015
- Hugo M. Botelho; Miquéias Lopes-Pacheco; Madalena C. Pinto; Violeta Railean; Ines Pankonien; Mariana F. Caleiro; Luka A. Clarke;
et al. "Global Functional Genomics Reveals GRK5 as a Cystic Fibrosis Therapeutic Target Synergistic with Current Modulators".
iScience (2025): http://dx.doi.org/10.1016/j.isci.2025.111942.
10.1016/j.isci.2025.111942
- Marcelo Azevedo; David Chafi Zeitune; Renan Lira de Farias; Eduardo Nunes; Mafalda Bacalhau; Margarida D. Amaral; Miquéias
Lopes-Pacheco; Camilla Buarque. "Direct access of 4-acyl-1,2,3-triazoles from acetophenones: A synthetic shortcut for novel
p.Phe508del-CFTR traffic correctors". Journal of Molecular Structure (2025): http://dx.doi.org/10.1016/j.molstruc.2024.139897.
10.1016/j.molstruc.2024.139897
- Margarida D. Amaral; Ines Pankonien. "Theranostics vs theratyping or theranostics plus theratyping?". Journal of Cystic
Fibrosis (2025): https://doi.org/10.1016/j.jcf.2024.09.013.
10.1016/j.jcf.2024.09.013
- Joana Pacheco; Leonor Almeida; Rita Boaventura; Ana Luisa Pereira; Claudia S. Rodrigues; Violeta Railean; Iris AL. Silva;
et al. "N1303K (p.Asn1303Lys) Variant: Expanding Frontiers in the Treatment of Cystic Fibrosis". Respiratory Medicine
(2025): https://www.sciencedirect.com/science/article/pii/S0954611125003014.
In press • 10.1016/j.rmed.2025.108238
- Elena K. Schneider-Futschik; Yimin Zhu; Danni Li; Mark D. Habgood; Bao N. Nguyen; Ines Pankonien; Margarida D. Amaral; Laura
E. Downie; Holly R. Chinnery. "The role of CFTR in the eye, and the effect of early highly effective modulator treatment for
cystic fibrosis on eye health". Progress in Retinal and Eye Research (2024): http://dx.doi.org/10.1016/j.preteyeres.2024.101299.
10.1016/j.preteyeres.2024.101299
- Marlou Bierlaagh; Anabela S. Ramalho; Iris A.L. Silva; Annelotte M. Vonk; Rutger M. van den Bor; Peter van Mourik; Johanna
Pott; et al. "Repeatability and reproducibility of the Forskolin-induced swelling (FIS) assay on intestinal organoids from
people with Cystic Fibrosis". Journal of Cystic Fibrosis (2024): http://dx.doi.org/10.1016/j.jcf.2024.04.014.
10.1016/j.jcf.2024.04.014
- Filipa Castela Ferreira; Margarida D. Amaral; Mafalda Bacalhau; Miquéias Lopes-Pacheco. "PTI-801 (posenacaftor) shares a common
mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR". European Journal of Pharmacology (2024): http://dx.doi.org/10.1016/j.ejphar.2024.176390.
10.1016/j.ejphar.2024.176390
- Marianna Pócsi; Zsolt Fejes; Zsolt Bene; Attila Nagy; István Balogh; Margarida D. Amaral; Milan Macek, Jr.; Béla Nagy, Jr..
"Human epididymis protein 4 (HE4) plasma concentration inversely correlates with the improvement of cystic fibrosis lung disease
in p.Phe508del-CFTR homozygous cases treated with the CFTR modulator lumacaftor/ivacaftor combination". Journal of Cystic
Fibrosis (2023): https://doi.org/10.1016/j.jcf.2023.04.001.
10.1016/j.jcf.2023.04.001
- Margarida D. Amaral. "Using the genome to correct the ion transport defect in cystic fibrosis". The Journal of Physiology
(2023): https://doi.org/10.1113/JP282308.
10.1113/JP282308
- Railean V; Rodrigues CS; Ramalho SS; Silva IAL; Bartosch J; Farinha CM; Ines Pankonien; Amaral MD. "Personalized medicine:
Function of CFTR variant p.Arg334Trp is rescued by currently available CFTR modulators.". Frontiers in molecular biosciences
(2023): https://europepmc.org/articles/PMC10063961.
10.3389/fmolb.2023.1155705
- Santos L; Nascimento R; Duarte A; Railean V; Amaral MD; Patrick Harrison; Gama-Carvalho M; Carlos Farinha. "Mutation-class
dependent signatures outweigh disease-associated processes in cystic fibrosis cells.". Cell & bioscience (2023): https://europepmc.org/articles/PMC9912517.
10.1186/s13578-023-00975-y
- Luka A. Clarke; Margarida D. Amaral. "What Can RNA-Based Therapy Do for Monogenic Diseases?". Pharmaceutics (2023):
https://doi.org/10.3390/pharmaceutics15010260.
10.3390/pharmaceutics15010260
- Mafalda Bacalhau; Filipa C. Ferreira; Iris A. L. Silva; Camilla D. Buarque; Margarida D. Amaral; Miquéias Lopes-Pacheco. "Additive
Potentiation of R334W-CFTR Function by Novel Small Molecules". Journal of Personalized Medicine (2023): https://doi.org/10.3390/jpm13010102.
10.3390/jpm13010102
- MD Amaral; Jo S; Centeio R; Park J; Ousingsawat J; Jeon DK; Talbi K; et al. "The SLC26A9 inhibitor S9-A13 provides no evidence
for a role of SLC26A9 in airway chloride secretion but suggests a contribution to regulation of ASL pH and gastric proton
secretion.". FASEB journal : official publication of the Federation of American Societies for Experimental Biology
(2022): https://doi.org/10.1096/fj.202200313RR.
10.1096/fj.202200313rr
- Amaral MD; Harrison PT. "Development of novel therapeutics for all individuals with CF (the future goes on).". Journal
of cystic fibrosis : official journal of the European Cystic Fibrosis Society (2022): https://doi.org/10.1016/j.jcf.2022.10.007.
10.1016/j.jcf.2022.10.007
- Lisa Rodenburg; Livia Delpiano; Violeta Railean; Raquel Centeio; Madalena Pinto; Shannon M. A. Smits; Isabelle S. van der
Windt; et al. "Drug Repurposing for Cystic Fibrosis: Identification of Drugs That Induce CFTR-Independent Fluid Secretion
in Nasal Organoids". International Journal of Molecular Sciences (2022): https://www.mdpi.com/1422-0067/23/20/12657.
10.3390/ijms232012657
- Margarida C Quaresma; Hugo M Botelho; Ines Pankonien; Cláudia S Rodrigues; Madalena C Pinto; Pau R Costa; Aires Duarte; Margarida
D Amaral. "Exploring YAP1-centered networks linking dysfunctional CFTR to epithelial–mesenchymal transition". Life Science
Alliance (2022): https://doi.org/10.26508/lsa.202101326.
10.26508/lsa.202101326
- Gimano Amatngalim; Lisa Rodenburg; Aalbers BL; Henriette Dreyer; Aarts EM; Sarhane D; Spelier S; et al. "Measuring cystic
fibrosis drug responses in organoids derived from 2D differentiated nasal epithelia.". Life science alliance (2022):
https://europepmc.org/articles/PMC9351388.
10.26508/lsa.202101320
- João F. Ferreira; Iris A. L. Silva; Hugo M. Botelho; Margarida D. Amaral; Carlos M. Farinha. "Absence of EPAC1 Signaling to
Stabilize CFTR in Intestinal Organoids". Cells (2022): https://doi.org/10.3390/cells11152295.
10.3390/cells11152295
- Margarida D. Amaral. "Precision medicine for rare diseases: The times they are A-Changin'". Current Opinion in Pharmacology
63 (2022): 102201-102201. https://doi.org/10.1016/j.coph.2022.102201.
10.1016/j.coph.2022.102201
- "Systems Approaches to Unravel Molecular Function: High-content siRNA Screen Identifies TMEM16A Traffic Regulators as Potential
Drug Targets for Cystic Fibrosis". Journal of Molecular Biology 434 5 (2022): 167436-167436. http://dx.doi.org/10.1016/j.jmb.2021.167436.
10.1016/j.jmb.2021.167436
- Ines Pankonien; Margarida C. Quaresma; Cláudia S. Rodrigues; Margarida D. Amaral. "CFTR, Cell Junctions and the Cytoskeleton".
International Journal of Molecular Sciences (2022): https://doi.org/10.3390/ijms23052688.
10.3390/ijms23052688
- "CFTR interactome mapping using the mammalian membrane two-hybrid high-throughput screening system". Molecular Systems
Biology 18 2 (2022): http://dx.doi.org/10.15252/msb.202110629.
10.15252/msb.202110629
- "Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A". Cells 11 1 (2022): 136-136. http://dx.doi.org/10.3390/cells11010136.
10.3390/cells11010136
- Mafalda Bacalhau; Filipa C. Ferreira; Filipe R. Souza; Arthur Kmit; Verônica D. da Silva; André S. Pimentel; Margarida D.
Amaral; et al. Corresponding author: Miquéias Lopes-Pacheco. "Identification of Novel F508del-CFTR Traffic Correctors Among
Triazole Derivative Compounds". European journal of pharmacology (2022): https://doi.org/10.1016/j.ejphar.2022.175396.
Submitted • 10.1016/j.ejphar.2022.175396
- "Rare Trafficking CFTR Mutations Involve Distinct Cellular Retention Machineries and Require Different Rescuing Strategies".
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Open access • Published • 10.3390/ijms21093133
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sites with multiple organelles". Cellular and Molecular Life Sciences (2020): https://doi.org/10.1007/s00018-020-03523-w.
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Open access • In press • 10.1016/j.xpro.2020.100019
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