Evidence map›Paper›PMID 31839569›Full record

ArticleCell stem cell2020

High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia.

Sriram Vaidyanathan, Ameen A Salahudeen, Zachary M Sellers, Dawn T Bravo, Shannon S Choi, Arpit Batish, Wei Le, Ron Baik, Sean de la O, Milan P Kaushik and 14 more

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cell stem cell, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 97 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
97citing papers in PubMed, 1 pooled it
11.7field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

97 citing papers in PubMed, 1 synthesis or guideline pooled it, 129 citations in OpenAlex.

  1. CRISPR for cystic fibrosis: Advances and insights from a systematic review.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Pooled it
  2. Review
  3. Review
  4. Article
  5. Review
  6. Review
  7. Review
  8. Review
  9. Article
  10. Article
  11. Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Review
  17. Article
  18. THERAPIES FOR NEONATAL DISEASES OF THE SURFACTANT SYSTEM.Transactions of the American Clinical and Climatological Association · 2025
    Review
  19. Airway Disease Modeling with Gene-Edited Human Basal Cell Transplantation.American journal of respiratory and critical care medicine · 2025
    Article
  20. Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025
    Article

37 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

24 authors at 4 institutions in 1 country.

Sriram VaidyanathanDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA.
Ameen A SalahudeenDepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA.
Zachary M SellersDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA.
Dawn T BravoDepartment of Otolaryngology-Head and Neck Surgery, Stanford, CA 94305, USA.
Shannon S ChoiDepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA.
Arpit BatishDepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA.
Wei LeDepartment of Otolaryngology-Head and Neck Surgery, Stanford, CA 94305, USA.
Ron BaikDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA.
Sean de la ODepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA.
Milan P KaushikDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA.
Noah GalperDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA.
Ciaran M LeeDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.
Christopher A TeranDepartment of Otolaryngology-Head and Neck Surgery, Stanford, CA 94305, USA.
Jessica H YooDepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA.
Gang BaoDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.
Eugene H ChangDepartment of Otolaryngology, University of Arizona, Tucson, Tucson, AZ 85724, USA.
Zara M PatelDepartment of Otolaryngology-Head and Neck Surgery, Stanford, CA 94305, USA.
Peter H HwangDepartment of Otolaryngology-Head and Neck Surgery, Stanford, CA 94305, USA.
Jeffrey J WineDepartment of Psychology, Stanford University, Stanford, CA 94305, USA.
Carlos E MillaDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA.
Tushar J DesaiDepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA. Electronic address: tdesai@stanford.edu.
Jayakar V NayakDepartment of Otolaryngology-Head and Neck Surgery, Stanford, CA 94305, USA. Electronic address: jnayak@stanford.edu.
Calvin J KuoDepartment of Internal Medicine, Stanford University, Stanford, CA 94305, USA. Electronic address: cjkuo@stanford.edu.
Matthew H PorteusDepartment of Pediatrics, Stanford University, Stanford, CA 94304, USA. Electronic address: mporteus@stanford.edu.
Stanford University · USStanford Medicine · USRice University · USUniversity of Arizona · US

Funding

Modeling Oral Cancer in Primary Organoid CultureK08DE027730 · NIDCR · UNIVERSITY OF ILLINOIS AT CHICAGO · PI SALAHUDEEN, AMEEN A. · 2018 to 2022
$708k
NIDCR NIH HHS K08 DE027730
6 · The paper itself

Abstract

Cystic fibrosis (CF) is a monogenic disorder caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Mortality in CF patients is mostly due to respiratory sequelae. Challenges with gene delivery have limited attempts to treat CF using in vivo gene therapy, and low correction levels have hindered ex vivo gene therapy efforts. We have used Cas9 and adeno-associated virus 6 to correct the ΔF508 mutation in readily accessible upper-airway basal stem cells (UABCs) obtained from CF patients. On average, we achieved 30%-50% allelic correction in UABCs and bronchial epithelial cells (HBECs) from 10 CF patients and observed 20%-50% CFTR function relative to non-CF controls in differentiated epithelia. Furthermore, we successfully embedded the corrected UABCs on an FDA-approved porcine small intestinal submucosal membrane (pSIS), and they retained differentiation capacity. This study supports further development of genetically corrected autologous airway stem cell transplant as a treatment for CF.

Indexed as

Cystic FibrosisAnimalsCell DifferentiationCystic Fibrosis Transmembrane Conductance RegulatorEpithelial CellsEpitheliumHumansStem CellsSwineCFTR protein, humanCystic Fibrosis Transmembrane Conductance Regulatorairway stem cellsbasal cellsCas9cell therapyCFCFTRCRISPRcystic fibrosisF508delgenome editing

Identifiers

PMID31839569
PMCPMC10908575
OpenAlexW2994805025

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.