What Can RNA-Based Therapy Do for Monogenic Diseases?

by Luka A. Clarke, Margarida D. Amaral
Published 2023-01-01

Cross-talk of inflammatory mediators and airway epithelium reveals the cystic fibrosis transmembrane conductance regulator as a major target

by Filipa B. Simões, Arthur Kmit, Margarida D. Amaral
Published 2021-12-01

What Role Does CFTR Play in Development, Differentiation, Regeneration and Cancer?

by Margarida D. Amaral, Margarida C. Quaresma, Ines Pankonien
Published 2020-04-01

Neuronal reprograming of protein homeostasis by calcium-dependent regulation of the heat shock response.

by M Catarina Silva, Margarida D Amaral, Richard I Morimoto
Published 2013-08-01

CFTR, Cell Junctions and the Cytoskeleton

by Ines Pankonien, Margarida C. Quaresma, Cláudia S. Rodrigues, Margarida D. Amaral
Published 2022-02-01

Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids

by Iris A. L. Silva, Violeta Railean, Aires Duarte, Margarida D. Amaral
Published 2021-05-01

Impact of KLF4 on Cell Proliferation and Epithelial Differentiation in the Context of Cystic Fibrosis

by Luís Sousa, Ines Pankonien, Filipa B. Simões, Marc Chanson, Margarida D. Amaral
Published 2020-09-01

Rare Trafficking CFTR Mutations Involve Distinct Cellular Retention Machineries and Require Different Rescuing Strategies

by Sofia S. Ramalho, Iris A. L. Silva, Margarida D. Amaral, Carlos M. Farinha
Published 2021-12-01

KLF4 Acts as a wt-CFTR Suppressor through an AKT-Mediated Pathway

by Luis Sousa, Ines Pankonien, Luka A Clarke, Iris Silva, Karl Kunzelmann, Margarida D Amaral
Published 2020-07-01

A genetic screening strategy identifies novel regulators of the proteostasis network.

by M Catarina Silva, Susan Fox, Monica Beam, Happy Thakkar, Margarida D Amaral, Richard I Morimoto
Published 2011-12-01

Absence of EPAC1 Signaling to Stabilize CFTR in Intestinal Organoids

by João F. Ferreira, Iris A. L. Silva, Hugo M. Botelho, Margarida D. Amaral, Carlos M. Farinha
Published 2022-07-01

Synergy in Cystic Fibrosis Therapies: Targeting SLC26A9

by Madalena C. Pinto, Margarida C. Quaresma, Iris A. L. Silva, Violeta Railean, Sofia S. Ramalho, Margarida D. Amaral
Published 2021-12-01

Integrity and Stability of PTC Bearing CFTR mRNA and Relevance to Future Modulator Therapies in Cystic Fibrosis

by Luka A. Clarke, Vanessa C. C. Luz, Szymon Targowski, Sofia S. Ramalho, Carlos M. Farinha, Margarida D. Amaral
Published 2021-11-01

Pediatric population with cystic fibrosis in the centre of Portugal: candidates for new therapies

by Juliana Roda, Teresa Teixeira, Iris AI Silva, Teresa Reis Silva, Ricardo Ferreira, Margarida D. Amaral, Guiomar Oliveira
Published 2022-03-01

Additive Potentiation of R334W-CFTR Function by Novel Small Molecules

by Mafalda Bacalhau, Filipa C. Ferreira, Iris A. L. Silva, Camilla D. Buarque, Margarida D. Amaral, Miquéias Lopes-Pacheco
Published 2023-01-01

Mutation-class dependent signatures outweigh disease-associated processes in cystic fibrosis cells

by Lúcia Santos, Rui Nascimento, Aires Duarte, Violeta Railean, Margarida D. Amaral, Patrick T. Harrison, Margarida Gama-Carvalho, Carlos M. Farinha
Published 2023-02-01

Personalized medicine: Function of CFTR variant p.Arg334Trp is rescued by currently available CFTR modulators

by Violeta Railean, Cláudia S. Rodrigues, Sofia S. Ramalho, Iris A. L. Silva, Jan Bartosch, Carlos M. Farinha, Ines Pankonien, Margarida D. Amaral
Published 2023-03-01

The K+ channel opener 1-EBIO potentiates residual function of mutant CFTR in rectal biopsies from cystic fibrosis patients.

by Eva K Roth, Stephanie Hirtz, Julia Duerr, Daniel Wenning, Irmgard Eichler, Hans H Seydewitz, Margarida D Amaral, Marcus A Mall
Published 2011-01-01

Full Rescue of F508del-CFTR Processing and Function by CFTR Modulators Can Be Achieved by Removal of Two Regulatory Regions

by Inna Uliyakina, Hugo M. Botelho, Ana C. da Paula, Sara Afonso, Miguel J. Lobo, Verónica Felício, Carlos M. Farinha, Margarida D. Amaral
Published 2020-06-01

CyFi-MAP: an interactive pathway-based resource for cystic fibrosis

by Catarina Pereira, Alexander Mazein, Carlos M. Farinha, Michael A. Gray, Karl Kunzelmann, Marek Ostaszewski, Irina Balaur, Margarida D. Amaral, Andre O. Falcao
Published 2021-11-01