An expanding protocol to validate nasospheroids as a functional assay to study airway CFTR-fluid transport

Maite Calucho; Silvia Gartner; Raquel García-Pérez; Llorenç Badiella; Sandra Cobo; Sandra Rovira; Laura Garriga; Paula Fernández; Eduardo Tizzano

doi:10.1183/13993003.congress-2020.329

An expanding protocol to validate nasospheroids as a functional assay to study airway CFTR-fluid transport

Maite Calucho, Silvia Gartner, Raquel García-Pérez, Llorenç Badiella, Sandra Cobo, Sandra Rovira, Laura Garriga, Paula Fernández, Eduardo Tizzano

Departament de Matemàtiques

Producció científica: Altres contribucions

Resum

Apuntàvem a estudiar cèl·lules derivades de pacients amb CF per determinar la funcionalitat del CFTR i la resposta als fàrmacs. Els organoides intestinals són un mètode validat i acceptat per mesurar l'eficàcia preclínica individual dels moduladors del CFTR. Tot i això, aquesta tècnica requereix temps i experiència pràctica. Per tant, investiguem mètodes alternatius i cèl·lules per estudiar la funcionalitat del CFTR. L'objectiu del nostre estudi era validar la metodologia prèviament notificada de nasosferoides (Guimbellot et al 2017) i aplicar aquesta tècnica per a estudiar la funcionalitat del CFTR i predir respostes dels pacients amb fibrosi quística a diferents fàrmacs.

Idioma original	Anglès
Edició	Suppl 64
Volum	56
DOIs	https://doi.org/10.1183/13993003.congress-2020.329
Estat de la publicació	Publicada - d’oct. 2020

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10.1183/13993003.congress-2020.329

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@misc{37546779656547e085bd22034ee403dc,

title = "An expanding protocol to validate nasospheroids as a functional assay to study airway CFTR-fluid transport",

abstract = "We aimed to study CF patient derived cells to determine CFTR functionality and drug response. Intestinal organoids is a validated and accepted method to measure the preclinical individual efficacy of CFTR modulators. However, this technique is time consuming and requires hands-on experience. Therefore, we investigated alternative methods and cells to study CFTR function. The aim of our study was to validate the previously reported methodology of nasospheroids (Guimbellot Jet al 2017) and to apply this technique to study CFTR functionality and predict responses of cystic fibrosis patients to different drugs. Nasospheroids from 7 controls and 7 CF patients presenting different genotypes were established in culture for ≤10 days. Cells were studied by live-cell microscopy. To validate our results, a statistical equation was developed to obtain personalized mixed linear models (R v3.1.2 software)",

author = "Maite Calucho and Silvia Gartner and Raquel Garc{\'i}a-P{\'e}rez and Lloren{\c c} Badiella and Sandra Cobo and Sandra Rovira and Laura Garriga and Paula Fern{\'a}ndez and Eduardo Tizzano",

year = "2020",

month = oct,

doi = "10.1183/13993003.congress-2020.329",

language = "English",

volume = "56",

edition = "Suppl 64",

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T1 - An expanding protocol to validate nasospheroids as a functional assay to study airway CFTR-fluid transport

AU - Calucho, Maite

AU - Gartner, Silvia

AU - García-Pérez, Raquel

AU - Badiella, Llorenç

AU - Cobo, Sandra

AU - Rovira, Sandra

AU - Garriga, Laura

AU - Fernández, Paula

AU - Tizzano, Eduardo

PY - 2020/10

Y1 - 2020/10

N2 - We aimed to study CF patient derived cells to determine CFTR functionality and drug response. Intestinal organoids is a validated and accepted method to measure the preclinical individual efficacy of CFTR modulators. However, this technique is time consuming and requires hands-on experience. Therefore, we investigated alternative methods and cells to study CFTR function. The aim of our study was to validate the previously reported methodology of nasospheroids (Guimbellot Jet al 2017) and to apply this technique to study CFTR functionality and predict responses of cystic fibrosis patients to different drugs. Nasospheroids from 7 controls and 7 CF patients presenting different genotypes were established in culture for ≤10 days. Cells were studied by live-cell microscopy. To validate our results, a statistical equation was developed to obtain personalized mixed linear models (R v3.1.2 software)

AB - We aimed to study CF patient derived cells to determine CFTR functionality and drug response. Intestinal organoids is a validated and accepted method to measure the preclinical individual efficacy of CFTR modulators. However, this technique is time consuming and requires hands-on experience. Therefore, we investigated alternative methods and cells to study CFTR function. The aim of our study was to validate the previously reported methodology of nasospheroids (Guimbellot Jet al 2017) and to apply this technique to study CFTR functionality and predict responses of cystic fibrosis patients to different drugs. Nasospheroids from 7 controls and 7 CF patients presenting different genotypes were established in culture for ≤10 days. Cells were studied by live-cell microscopy. To validate our results, a statistical equation was developed to obtain personalized mixed linear models (R v3.1.2 software)

U2 - 10.1183/13993003.congress-2020.329

DO - 10.1183/13993003.congress-2020.329

M3 - Other contribution

VL - 56

ER -

An expanding protocol to validate nasospheroids as a functional assay to study airway CFTR-fluid transport

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