Translational Research in Cystic Fibrosis: From Bench to Beside

Cystic fibrosis is the most common life-limiting recessive genetic disorder in Caucasian populations, characterized by the involvement of exocrine glands, causing multisystemic comorbidities. Since the first descriptions of pancreatic and pulmonary involvement in children, technological development...

Full description

Saved in:
Bibliographic Details
Published inFrontiers in pediatrics Vol. 10; p. 881470
Main Authors Garcia, Laura de Castro E, Petry, Lucas Montiel, Germani, Pedro Augusto Van Der Sand, Xavier, Luiza Fernandes, de Barros, Paula Barros, Meneses, Amanda da Silva, Prestes, Laura Menestrino, Bittencourt, Luana Braga, Pieta, Marina Puerari, Friedrich, Frederico, Pinto, Leonardo Araújo
Format Journal Article
LanguageEnglish
Published Switzerland Frontiers Media S.A 16.05.2022
Subjects
CFTR modulators
cystic fibrosis
Pediatrics
quality of life
translational research
treatment
treatment
translational research
quality of life
CFTR modulators
cystic fibrosis
Online AccessGet full text

Cover

Abstract Cystic fibrosis is the most common life-limiting recessive genetic disorder in Caucasian populations, characterized by the involvement of exocrine glands, causing multisystemic comorbidities. Since the first descriptions of pancreatic and pulmonary involvement in children, technological development and basic science research have allowed great advances in the diagnosis and treatment of cystic fibrosis. The great search for treatments that acted at the genetic level, despite not having found a cure for this disease, culminated in the creation of CFTR modulators, highly effective medications for certain groups of patients. However, there are still many obstacles behind the treatment of the disease to be discussed, given the wide variety of mutations and phenotypes involved and the difficulty of access that permeate these new therapies around the world.
AbstractList Cystic fibrosis is the most common life-limiting recessive genetic disorder in Caucasian populations, characterized by the involvement of exocrine glands, causing multisystemic comorbidities. Since the first descriptions of pancreatic and pulmonary involvement in children, technological development and basic science research have allowed great advances in the diagnosis and treatment of cystic fibrosis. The great search for treatments that acted at the genetic level, despite not having found a cure for this disease, culminated in the creation of CFTR modulators, highly effective medications for certain groups of patients. However, there are still many obstacles behind the treatment of the disease to be discussed, given the wide variety of mutations and phenotypes involved and the difficulty of access that permeate these new therapies around the world.
Author Prestes, Laura Menestrino
Pieta, Marina Puerari
Friedrich, Frederico
Petry, Lucas Montiel
Xavier, Luiza Fernandes
Meneses, Amanda da Silva
Bittencourt, Luana Braga
de Barros, Paula Barros
Pinto, Leonardo Araújo
Garcia, Laura de Castro E
Germani, Pedro Augusto Van Der Sand
AuthorAffiliation Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul , Porto Alegre , Brazil
AuthorAffiliation_xml – name: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul , Porto Alegre , Brazil
Author_xml – sequence: 1
  givenname: Laura de Castro E
  surname: Garcia
  fullname: Garcia, Laura de Castro E
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 2
  givenname: Lucas Montiel
  surname: Petry
  fullname: Petry, Lucas Montiel
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 3
  givenname: Pedro Augusto Van Der Sand
  surname: Germani
  fullname: Germani, Pedro Augusto Van Der Sand
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 4
  givenname: Luiza Fernandes
  surname: Xavier
  fullname: Xavier, Luiza Fernandes
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 5
  givenname: Paula Barros
  surname: de Barros
  fullname: de Barros, Paula Barros
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 6
  givenname: Amanda da Silva
  surname: Meneses
  fullname: Meneses, Amanda da Silva
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 7
  givenname: Laura Menestrino
  surname: Prestes
  fullname: Prestes, Laura Menestrino
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 8
  givenname: Luana Braga
  surname: Bittencourt
  fullname: Bittencourt, Luana Braga
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 9
  givenname: Marina Puerari
  surname: Pieta
  fullname: Pieta, Marina Puerari
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 10
  givenname: Frederico
  surname: Friedrich
  fullname: Friedrich, Frederico
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
– sequence: 11
  givenname: Leonardo Araújo
  surname: Pinto
  fullname: Pinto, Leonardo Araújo
  organization: Centro Infant, Department of Pediatrics, School of Medicine, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil
BackLink https://www.ncbi.nlm.nih.gov/pubmed/35652053$$D View this record in MEDLINE/PubMed
BookMark eNpVkctPGzEQxq2KqjzKvadqj70kHdv78PTQqkSkRUJCQvRszXonYLSxU3tTif8eh1AEvng0j983mu9YHIQYWIhPEuZaG_y62vAwV6DU3BhZd_BOHCmF7UzpFg5exYfiNOd7KA87aGTzQRzqpm0UNPpI_LhJFPJIk4-BxuqaM1Nyd5UP1eIhT95VS9-nmH3-Vi1TXFdnHEp5iiXIfuCP4v2Kxsynz_-J-LM8v1n8nl1e_bpY_LycOQ0dzDTWbW9AI_EKAZGpqNc1IknAoeOaWuy4lwiSjB7MUPJSNgO1bUfsjD4RF3vuEOnebpJfU3qwkbx9SsR0aymVdUe2Q9-DMa4B4-oaeqKiigbBEYFBxML6vmdttv2aB8dhSjS-gb6tBH9nb-M_i7LG5gnw5RmQ4t8t58mufXY8jhQ4brNVbac6UKapSyvsW105Yk68epGRYHc22p2Ndmej3dtYRj6_Xu9l4L9p-hG8CZlv
CitedBy_id crossref_primary_10_3390_jcm12113704
crossref_primary_10_1186_s13104_023_06388_x
crossref_primary_10_3389_fped_2022_1114549
Cites_doi 10.3389/fphar.2018.01490
10.1542/peds.12.5.549
10.1016/S2213-2600(19)30337-6
10.1016/S0140-6736(20)32542-3
10.1126/science.2772657
10.1016/0092-8674(95)90241-4
10.3978/j.issn.2072-1439.2011.09.09
10.1542/peds.23.3.545
10.1056/NEJMoa1709847
10.1001/archpedi.1938.01980140114013
10.1186/s12967-017-1193-9
10.1016/j.stemcr.2019.04.014
10.1126/science.2475911
10.1056/NEJMoa1409547
10.1093/hmg/ddz139
10.1016/j.phrs.2020.105267
10.3389/fphar.2019.00514
10.3389/fphar.2016.00275
10.1056/NEJMoa1807120
10.1126/science.2570460
10.1091/mbc.e14-04-0935
10.1016/S0140-6736(19)32597-8
10.1056/NEJMoa1908639
10.1016/j.gastre.2015.12.006
10.1146/annurev-genom-083117-021329
10.3389/fphar.2019.01662
10.1590/S1806-37562017000000065
10.1056/NEJMoa1709846
10.1177/2472555219849375
10.1016/j.jcf.2021.01.007
10.1016/S2213-2600(17)30215-1
ContentType Journal Article
Copyright Copyright © 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto.
Copyright © 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto. 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto
Copyright_xml – notice: Copyright © 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto.
– notice: Copyright © 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto. 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto
DBID NPM
AAYXX
CITATION
7X8
5PM
DOA
DOI 10.3389/fped.2022.881470
DatabaseName PubMed
CrossRef
MEDLINE - Academic
PubMed Central (Full Participant titles)
DOAJ Directory of Open Access Journals
DatabaseTitle PubMed
CrossRef
MEDLINE - Academic
DatabaseTitleList PubMed


CrossRef
Database_xml – sequence: 1
  dbid: DOA
  name: Directory of Open Access Journals
  url: https://www.doaj.org/
  sourceTypes: Open Website
– sequence: 2
  dbid: NPM
  name: PubMed
  url: https://proxy.k.utb.cz/login?url=http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed
  sourceTypes: Index Database
DeliveryMethod fulltext_linktorsrc
Discipline Medicine
EISSN 2296-2360
EndPage 881470
ExternalDocumentID oai_doaj_org_article_dbb088c508c440baa39a9890caa08999
10_3389_fped_2022_881470
35652053
Genre Journal Article
Review
GrantInformation_xml – fundername: ;
GroupedDBID 53G
5VS
9T4
AAFWJ
ACGFO
ACGFS
ACXDI
ADBBV
ADRAZ
AFPKN
ALMA_UNASSIGNED_HOLDINGS
AOIJS
BAWUL
BCNDV
DIK
EMOBN
GROUPED_DOAJ
GX1
HYE
IAO
IEA
IHR
IHW
IPNFZ
KQ8
M48
M~E
NPM
OK1
PGMZT
RIG
RNS
RPM
AAYXX
CITATION
7X8
5PM
ID FETCH-LOGICAL-c3070-3946b8039aef9099ea0534499a109d7e4a697eb1901a83d8da10115da667aec83
IEDL.DBID RPM
ISSN 2296-2360
IngestDate Tue Oct 22 15:16:29 EDT 2024
Tue Sep 17 21:24:48 EDT 2024
Sat Oct 26 01:18:18 EDT 2024
Thu Nov 21 21:39:54 EST 2024
Sat Sep 28 08:21:30 EDT 2024
IsDoiOpenAccess true
IsOpenAccess true
IsPeerReviewed true
IsScholarly true
Keywords treatment
translational research
quality of life
CFTR modulators
cystic fibrosis
Language English
License Copyright © 2022 Garcia, Petry, Germani, Xavier, Barros, Meneses, Prestes, Bittencourt, Pieta, Friedrich and Pinto.
This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
LinkModel DirectLink
MergedId FETCHMERGED-LOGICAL-c3070-3946b8039aef9099ea0534499a109d7e4a697eb1901a83d8da10115da667aec83
Notes ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
This article was submitted to Pediatric Pulmonology, a section of the journal Frontiers in Pediatrics
Edited by: Mauricio Tomas Caballero, Consejo Nacional de Investigaciones Científicas y Técnicas (CONICET), Argentina
Reviewed by: Petra Pallagi, University of Szeged, Hungary
OpenAccessLink https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9149599/
PMID 35652053
PQID 2672702854
PQPubID 23479
PageCount 1
ParticipantIDs doaj_primary_oai_doaj_org_article_dbb088c508c440baa39a9890caa08999
pubmedcentral_primary_oai_pubmedcentral_nih_gov_9149599
proquest_miscellaneous_2672702854
crossref_primary_10_3389_fped_2022_881470
pubmed_primary_35652053
PublicationCentury 2000
PublicationDate 20220516
PublicationDateYYYYMMDD 2022-05-16
PublicationDate_xml – month: 5
  year: 2022
  text: 20220516
  day: 16
PublicationDecade 2020
PublicationPlace Switzerland
PublicationPlace_xml – name: Switzerland
PublicationTitle Frontiers in pediatrics
PublicationTitleAlternate Front Pediatr
PublicationYear 2022
Publisher Frontiers Media S.A
Publisher_xml – name: Frontiers Media S.A
References Sant'agnese (B6) 1953; 12
Jensen (B14) 1995; 83
Veit (B15) 2016; 27
B29
Rommens (B10) 1989; 245
Shteinberg (B1) 2021; 397
Ratjen (B28) 2017; 5
Bell (B34) 2020; 8
Heijerman (B24) 2019; 394
Ramos (B36) 2021; 20
Athanazio (B7) 2017
Keating (B16) 2018; 379
Fonseca (B30) 2020; 162
Kerem (B8) 1989; 245
Taylor-Cousar (B26) 2017; 377
Berg (B21) 2019; 24
Rowe (B25) 2017; 377
Middleton (B23) 2019; 381
B32
B11
B12
Matthes (B17) 2018; 379
Rafeeq (B31) 2017; 15
B35
B37
Lopes (B13) 2020; 10
Andersen (B4) 1938; 56
Merkert (B19) 2019; 12
Gibson (B5) 1959; 23
Riordan (B9) 1989; 245
Wainwright (B27) 2015; 373
Lopes-Pacheco (B22) 2016; 7
O'Neal (B18) 2018; 19
De Wilde (B20) 2019; 10
Yan (B33) 2019; 28
Sturm (B2) 2012; 4
Navarro (B3) 2016; 39
References_xml – ident: B12
– ident: B35
– volume: 379
  start-page: 1612
  year: 2018
  ident: B17
  article-title: Variable responses to CFTR correctors in vitro: estimating the design effect in precision medicine
  publication-title: Front Pharmacol.
  doi: 10.3389/fphar.2018.01490
  contributor:
    fullname: Matthes
– volume: 12
  start-page: 549
  year: 1953
  ident: B6
  article-title: Abnormal electrolyte composition of sweat in cystic fibrosis of the pancreas; clinical significance and relationship to the disease
  publication-title: Pediatrics.
  doi: 10.1542/peds.12.5.549
  contributor:
    fullname: Sant'agnese
– volume: 8
  start-page: 65
  year: 2020
  ident: B34
  article-title: The future of cystic fibrosis care: a global perspective
  publication-title: Lancet Respir Med.
  doi: 10.1016/S2213-2600(19)30337-6
  contributor:
    fullname: Bell
– volume: 397
  start-page: 2195
  year: 2021
  ident: B1
  article-title: Cystic fibrosis
  publication-title: Lancet.
  doi: 10.1016/S0140-6736(20)32542-3
  contributor:
    fullname: Shteinberg
– volume: 245
  start-page: 1059
  year: 1989
  ident: B10
  article-title: Identification of the cystic fibrosis gene: chromosome walking and jumping
  publication-title: Science.
  doi: 10.1126/science.2772657
  contributor:
    fullname: Rommens
– volume: 83
  start-page: 129
  year: 1995
  ident: B14
  article-title: Multiple proteolytic systems, including the proteasome, contribute to CFTR processing
  publication-title: Cell.
  doi: 10.1016/0092-8674(95)90241-4
  contributor:
    fullname: Jensen
– ident: B29
– volume: 4
  start-page: 48
  year: 2012
  ident: B2
  article-title: An advanced stochastic model for mucociliary particle clearance in cystic fibrosis lungs
  publication-title: J Thorac Dis.
  doi: 10.3978/j.issn.2072-1439.2011.09.09
  contributor:
    fullname: Sturm
– volume: 23
  start-page: 545
  year: 1959
  ident: B5
  article-title: A test for concentration of electrolytes in sweat in cystic fibrosis of the pancreas utilizing pilocarpine by iontophoresis
  publication-title: Pediatrics.
  doi: 10.1542/peds.23.3.545
  contributor:
    fullname: Gibson
– volume: 377
  start-page: 2024
  year: 2017
  ident: B25
  article-title: Tezacaftor–ivacaftor in residual-function heterozygotes with cystic fibrosis
  publication-title: N Engl J Med.
  doi: 10.1056/NEJMoa1709847
  contributor:
    fullname: Rowe
– volume: 56
  start-page: 344
  year: 1938
  ident: B4
  article-title: Cystic fibrosis of the pancreas and its relation to celiac disease: a clinical and pathologic study
  publication-title: Am J Dis Child.
  doi: 10.1001/archpedi.1938.01980140114013
  contributor:
    fullname: Andersen
– volume: 15
  start-page: 1
  year: 2017
  ident: B31
  article-title: Cystic fibrosis: current therapeutic targets and future approaches
  publication-title: J Transl Med.
  doi: 10.1186/s12967-017-1193-9
  contributor:
    fullname: Rafeeq
– volume: 12
  start-page: 1389
  year: 2019
  ident: B19
  article-title: High-throughput screening for modulators of CFTR activity based on genetically engineered cystic fibrosis disease-specific iPSCs
  publication-title: Stem Cell Rep.
  doi: 10.1016/j.stemcr.2019.04.014
  contributor:
    fullname: Merkert
– ident: B37
– ident: B11
– volume: 245
  start-page: 1066
  year: 1989
  ident: B9
  article-title: Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA
  publication-title: Science.
  doi: 10.1126/science.2475911
  contributor:
    fullname: Riordan
– volume: 373
  start-page: 220
  year: 2015
  ident: B27
  article-title: Lumacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR
  publication-title: N Engl J Med.
  doi: 10.1056/NEJMoa1409547
  contributor:
    fullname: Wainwright
– volume: 28
  start-page: R88
  year: 2019
  ident: B33
  article-title: Advances in gene therapy for cystic fibrosis lung disease
  publication-title: Hum Mol Genet.
  doi: 10.1093/hmg/ddz139
  contributor:
    fullname: Yan
– volume: 162
  start-page: 105267
  year: 2020
  ident: B30
  article-title: Cystic fibrosis: physiopathology and the latest pharmacological treatments
  publication-title: Pharmacol Research.
  doi: 10.1016/j.phrs.2020.105267
  contributor:
    fullname: Fonseca
– volume: 10
  start-page: 514
  year: 2019
  ident: B20
  article-title: Identification of GLPG/ABBV-2737, a novel class of corrector, which exerts functional synergy with other CFTR modulators
  publication-title: Front Pharmacol.
  doi: 10.3389/fphar.2019.00514
  contributor:
    fullname: De Wilde
– volume: 7
  start-page: 275
  year: 2016
  ident: B22
  article-title: CFTR modulators: shedding light on precision medicine for cystic fibrosis
  publication-title: Front Pharmacol.
  doi: 10.3389/fphar.2016.00275
  contributor:
    fullname: Lopes-Pacheco
– volume: 379
  start-page: 1612
  year: 2018
  ident: B16
  article-title: VX-445–Tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles
  publication-title: N Engl J Med.
  doi: 10.1056/NEJMoa1807120
  contributor:
    fullname: Keating
– volume: 245
  start-page: 1073
  year: 1989
  ident: B8
  article-title: Identification of the cystic fibrosis gene: genetic analysis
  publication-title: Science.
  doi: 10.1126/science.2570460
  contributor:
    fullname: Kerem
– volume: 27
  start-page: 424
  year: 2016
  ident: B15
  article-title: From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
  publication-title: Mol Biol Cell.
  doi: 10.1091/mbc.e14-04-0935
  contributor:
    fullname: Veit
– volume: 394
  start-page: 1940
  year: 2019
  ident: B24
  article-title: Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
  publication-title: Lancet.
  doi: 10.1016/S0140-6736(19)32597-8
  contributor:
    fullname: Heijerman
– volume: 381
  start-page: 1809
  year: 2019
  ident: B23
  article-title: Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele
  publication-title: N Engl J Med.
  doi: 10.1056/NEJMoa1908639
  contributor:
    fullname: Middleton
– volume: 39
  start-page: 36
  year: 2016
  ident: B3
  article-title: Historical compilation of cystic fibrosis
  publication-title: Gastroenter Hepatol.
  doi: 10.1016/j.gastre.2015.12.006
  contributor:
    fullname: Navarro
– volume: 19
  start-page: 201
  year: 2018
  ident: B18
  article-title: Cystic fibrosis disease modifiers: complex genetics defines the phenotypic diversity in a monogenic disease
  publication-title: Annu Ver Genomics Hum Genet.
  doi: 10.1146/annurev-genom-083117-021329
  contributor:
    fullname: O'Neal
– volume: 10
  start-page: 1662
  year: 2020
  ident: B13
  article-title: CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine
  publication-title: Front Pharmacol.
  doi: 10.3389/fphar.2019.01662
  contributor:
    fullname: Lopes
– year: 2017
  ident: B7
  article-title: Brazilian guidelines for the diagnosis and treatment of cystic fibrosis Brazilian guidelines for the diagnosis and treatment of cystic fibrosis
  publication-title: J Bras Pneumol.
  doi: 10.1590/S1806-37562017000000065
  contributor:
    fullname: Athanazio
– volume: 377
  start-page: 2013
  year: 2017
  ident: B26
  article-title: Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del
  publication-title: N Engl J Med.
  doi: 10.1056/NEJMoa1709846
  contributor:
    fullname: Taylor-Cousar
– volume: 24
  start-page: 724
  year: 2019
  ident: B21
  article-title: High-throughput surface liquid absorption and secretion assays to identify F508del CFTR correctors using patient primary airway epithelial cultures
  publication-title: SLAS Discov.
  doi: 10.1177/2472555219849375
  contributor:
    fullname: Berg
– ident: B32
– volume: 20
  start-page: 381
  year: 2021
  ident: B36
  article-title: Challenges in the use of highly effective modulator treatment for cystic fibrosis
  publication-title: J Cyst Fibros.
  doi: 10.1016/j.jcf.2021.01.007
  contributor:
    fullname: Ramos
– volume: 5
  start-page: 557
  year: 2017
  ident: B28
  article-title: Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial
  publication-title: Lancet Respir Med.
  doi: 10.1016/S2213-2600(17)30215-1
  contributor:
    fullname: Ratjen
SSID ssj0000970515
Score 2.2345307
SecondaryResourceType review_article
Snippet Cystic fibrosis is the most common life-limiting recessive genetic disorder in Caucasian populations, characterized by the involvement of exocrine glands,...
SourceID doaj
pubmedcentral
proquest
crossref
pubmed
SourceType Open Website
Open Access Repository
Aggregation Database
Index Database
StartPage 881470
SubjectTerms CFTR modulators
cystic fibrosis
Pediatrics
quality of life
translational research
treatment
SummonAdditionalLinks – databaseName: DOAJ Directory of Open Access Journals
  dbid: DOA
  link: http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwrV1LS8QwEA7iQbyIb-uLCF48lO2mSdp4EXexLMJ6cmFvIWlTdkHbZR8H_70zbXfpiuDFW0kDDd9MMjOdyTeE3EfMCOlC5isRWZ8bgeegsL5KBbORUzytqiqHb3Iw4q9jMW61-sKasJoeuAauk1kLGyEFPyLlPLDGhMqoWAWpMZixqq_uBawVTFVnsIqweUmdl4QoTHXymUNiUMZANbocexO37FBF1_-bj_mzVLJle5JDctA4jfS5XuwR2XHFMdkbNmnxE_JUWZyP5r8eXVfT0WlB-19IxEwTiIrLxXTxSJN5-Ul7oJsTuizhAdt1npJR8vLeH_hNZwQ_xT3qh4pLGwcAhcsV-HjOwF7iELyYbqCyyHEjVQSnMBh7E4dZnME4uH6ZkTIyLo3DM7JblIW7INSIwOYwBeKwGAyaMLmzggUuFk7yVDGPPKxx0rOaAEND4ICYasRUI6a6xtQjPQRyMw-pq6sBEKhuBKr_EqhH7tZi0KDqmL8whStXC80waxzglU-PnNdi2XwqBMeUAQgeibYEtrWW7TfFdFLRaSsMEpW6_I_FX5F9xAPLC7rymuwu5yt3A17L0t5WCvoNz_bosA
  priority: 102
  providerName: Directory of Open Access Journals
– databaseName: Scholars Portal Open Access Journals
  dbid: M48
  link: http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwdV3dS9xAEB9EQXwptZ_RWrbQlz6kzW32I1MQUfEQ4frUA9-W3WSvHmiidyfU_96ZJGd7cr6FzZJkZ3ZmfpOZnQH4aqXXJuYyRW1DqrxmPahDiqWWwUZUZZtVOfplzsfq4lJf_jse3RNwvta1435S49n19793D0ck8IfscZK9_TG5jVzzU0ri-kBZcuC3JNlFTvAa9WC_1ctouaEJd5uTaFKZm6yLW659yA5s5wR2ZKbzFZPVVvZfB0efZ1X-Z6aGr-FVjy_FcbchdmEj1m9ge9RH0N_CUWucrvtfgGKZeCemtTh94JrNYkgOdDOfzn-K4ay5ESe0ja_EoqEL7uz5DsbDs9-n52nfRCEtWZzTHJUJRZajjxMkOBg9rUiRn-MHGVY2Km_QksImXOCLvCoqGieUWHljrI9lkb-Hzbqp40cQXmdhQlPIZSvI9mk_iYEIFAsdjSpRJvBtSSd329XKcORjMHkdk9cxeV1H3gROmJBP87jKdTvQzP64XmhcFQIpwZIwZKlUFrynVWCBWek9RysxgS9LNjiSCg51-Do293MnOcCc8enQBD50bHl61ZKtCdgVhq18y-qdenrVVt5G9icR91585j7s8CI5vWBgPsHmYnYfDwi1LMLndjM-Ap4v5SY
  priority: 102
  providerName: Scholars Portal
Title Translational Research in Cystic Fibrosis: From Bench to Beside
URI https://www.ncbi.nlm.nih.gov/pubmed/35652053
https://search.proquest.com/docview/2672702854
https://pubmed.ncbi.nlm.nih.gov/PMC9149599
https://doaj.org/article/dbb088c508c440baa39a9890caa08999
Volume 10
hasFullText 1
inHoldings 1
isFullTextHit
isPrint
link http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV3BbtswDCXaHopdhm1tN29roQK77ODGsSXL3GVYgwXFgBQ7rEBvgiQra4DGDpL0sL8fKdtFM_S0i2HYMiyRNPloUiTAJ51bVYYiT1Fpl0qrWA8ql6JXudMBpY9ZlbPr8upG_rhVt3ughr0wMWnfu8VFc7-8aBZ3MbdytfSjIU9s9HM2QYb1iKN92Cfz-8RFj-oXNfct6UKS5IDhaL4KXBM0z0kqxlJz87eCcEyeqWLHGsWi_c8hzX8TJp9YoOkreNlDR_Gtm-Jr2AvNGzic9cHxI_ga7c59_3dPDDl1YtGIyR8uxyym5Bu3m8Xmi5iu26W4JAm9E9uWTrhp5zHcTL__mlylfX-E1POXmhYoS1dlBdowR0J6wdKKJLkwdpxhrYO0JWrSxWTybVXUVU3XCQDWtiy1Db4qTuCgaZvwDoRVmZvTEPLGKjJrys6DIwKFSoVSeswT-DzQyay6MhiG3Acmr2HyGiav6cibwCUT8nEcF7COF9r1b9Oz0dTOkX7zBA-9lJmzllaBFWbeWg5EYgLnAxsMCTxHMWwT2oeNyTl2nPHGzwTedmx5fNXA1gT0DsN25rJ7h2QsFtXuZer9fz_5AV4wETizYFx-hIPt-iGcEmDZurPo6NNxJquzKKx_AZiy6vY
link.rule.ids 230,314,727,780,784,864,885,2102,24318,27924,27925,53791,53793
linkProvider National Library of Medicine
linkToHtml http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1Lb9swDCa6Dth2Kbq3u3bTgF12cOPYkmXuMqzBgmxrih1aoDdBkpU2QGMHSXrYvx_pR9EMO_VmyDIskTT50aRIgE86tSoPWRqj0i6WVrEeVC5Gr1KnA0rfZFVOz_LJhfx5qS53QPVnYZqkfe_mx9XN4riaXze5lcuFH_R5YoPf0xEyrEccPILHKtM4vOekNwoYNXcuaYOS5ILhYLYMXBU0TUkuhlJz-7eMkEyaqGzLHjVl-_-HNf9Nmbxng8b7sNeBR_GtXeRz2AnVC3gy7cLjL-FrY3luuv97os-qE_NKjP5wQWYxJu-4Xs_XX8R4VS_ECcnotdjUdMFtO1_Bxfj7-WgSdx0SYs_fapyhzF2RZGjDDAnrBUs7kuTE2GGCpQ7S5qhJG5PRt0VWFiWNEwQsbZ5rG3yRvYbdqq7CWxBWJW5GU8gfK8iwKTsLjggUChVy6TGN4HNPJ7NsC2EYciCYvIbJa5i8piVvBCdMyLt5XMK6GahXV6ZjpCmdIw3nCSB6KRNnLe0CC0y8tRyKxAg-9mwwJPIcx7BVqG_XJuXoccJHPyN407Ll7lU9WyPQWwzbWsv2HZKypqx2J1UHD37yAzydnE9PzemPs1_v4BkThPMMhvkh7G5Wt-GI4MvGvW-E9S8PVOx8
linkToPdf http://utb.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwnV1Lb5wwEB61qRT1EqVv0pcr9dIDgQUb416qZluUPjbKoZFys2xjmpWysNrdHPrvM2Mg2q166g0ZI_A3g-cbZpgBeC8zIwqfZ7ES0sbcCNoHhY2VE5mVXnEXsipnZ8XpBf9-KS63Wn2FpH1n58ft9eK4nV-F3MrlwiVjnlhyPpsqovVKJcu6Se7DA5Gjkm056mETVpK6l_SBSXTDVNIsPVUGzTLUjQmX1AIuRzaTpSLfsUmhdP-_-ObfaZNbdqg6hIOBQLLP_YM-gnu-fQz7syFE_gQ-BetzPXzjY2NmHZu3bPqHijKzCj3kbj1ff2TVqluwE9TTK7bp8IBadz6Fi-rrr-lpPHRJiB29r3GueGHLNFfGNwr5nje4Io6OjJmkqpaem0JJ3JHR8Jsyr8sax5EG1qYopPGuzJ_BXtu1_gUwI1Lb4BT0yUo0bsI03iJAvhS-4E5lEXwYcdLLvhiGRieC4NUEryZ4dQ9vBCcE5N08KmMdBrrVbz0IU9fW4i7nkCQ6zlNrDK5ClSp1xlA4UkXwbhSDRrWnWIZpfXez1hlFkFP6_TOC571Y7m41ijUCuSOwnWfZPYOaFkprD5p19N9XvoX98y-V_vnt7MdLeEh4UKrBpHgFe5vVjX-NDGZj3wRdvQWD6u2P
openUrl ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=Translational+Research+in+Cystic+Fibrosis%3A+From+Bench+to+Beside&rft.jtitle=Frontiers+in+pediatrics&rft.au=Garcia%2C+Laura+de+Castro+E&rft.au=Petry%2C+Lucas+Montiel&rft.au=Germani%2C+Pedro+Augusto+Van+Der+Sand&rft.au=Xavier%2C+Luiza+Fernandes&rft.date=2022-05-16&rft.issn=2296-2360&rft.eissn=2296-2360&rft.volume=10&rft.spage=881470&rft_id=info:doi/10.3389%2Ffped.2022.881470&rft_id=info%3Apmid%2F35652053&rft.externalDocID=35652053
thumbnail_l http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=2296-2360&client=summon
thumbnail_m http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=2296-2360&client=summon
thumbnail_s http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=2296-2360&client=summon