Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells

被引：0

作者：

Isabelle Rose ^{[1
]}

Miriam Greenwood ^{[2
]}

Matthew Biggart ^{[3
]}

Natalie Baumlin ^{[4
]}

Robert Tarran ^{[3
]}

Stephen L. Hart ^{[2
]}

Deborah L. Baines ^{[1
]}

机构：

[1] City St George’s,Institute of Infection and Immunity, School of Health and Medical Sciences

[2] University of London,Department of Genetics and Genomic Medicine

[3] UCL Great Ormond Street Institute of Child Health,Division of Genetic, Environmental and Inhalational Disease

[4] University of Kansas Medical Center,Department of Internal Medicine

[5] University of Kansas Medical Center,undefined

来源：

Cellular and Molecular Life Sciences | / 82卷 / 1期

关键词：

Class 1 mutation; Transfection; Air–liquid-interface; Nasal; Ciliated cell; Secretory cell;

D O I：

10.1007/s00018-025-05587-y

中图分类号：

学科分类号：

摘要：

引用

共 22 条

[1] RNA editing applied to cystic fibrosis: RESTORE can target G542X CFTR mRNA and revert the nonsense mutation
Titoli, Simona
Barra, Viviana
Gargano, Serena
Di Leonardo, Aldo
Melfi, Raffaella
GENE, 2025, 951
[2] A Novel G542X CFTR Rat Model of Cystic Fibrosis Is Sensitive to Nonsense Mediated Decay
Sharma, Jyoti
Abbott, Joseph
Klaskala, Lauren
Zhao, Guojun
Birket, Susan E.
Rowe, Steven M.
FRONTIERS IN PHYSIOLOGY, 2020, 11
[3] Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids
Nicosia, Lucia
Pranke, Iwona
Latorre, Roberta V.
Murray, Joss B.
Lonetti, Lisa
Cavusoglu-Doran, Kader
Dreano, Elise
Costello, James P.
Carroll, Michael
Melotti, Paola
Sorio, Claudio
Sermet-Gaudelus, Isabelle
Scallan, Martina F.
Harrison, Patrick T.
ISCIENCE, 2025, 28 (03)
[4] Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids
Crawford, Daniel K.
Mullenders, Jasper
Pott, Johanna
Boj, Sylvia F.
Landskroner-Eiger, Shira
Goddeeris, Matthew M.
JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (03) : 436 - 442
[5] Cultivation and characterisation of human airway epithelial cells with potential for CFTR editing for the development of cystic fibrosis therapies
Avgerinou, A.
Ofrim, M.
Woodall, M.
Walker, A.
O'Callaghan, C.
Baines, D.
Hart, S.
Bonfanti, P.
HUMAN GENE THERAPY, 2018, 29 (12) : A106 - A107
[6] Hyperechogenic bowel loops and meconiurn ileus in a fetus carrying the D1152H and G542X cystic fibrosis CFTR mutations
Orgad, S
Berkenstadt, M
Achiron, R
Yahav, Y
Gazit, E
Barkai, G
Loewenthal, R
PRENATAL DIAGNOSIS, 2002, 22 (07) : 636 - 637
[7] Expression of gain-of-function CFTR in cystic fibrosis airway cells restores epithelial function better than wild-type or codon-optimized CFTR
Woodall, Maximillian
Tarran, Robert
Lee, Rhianna
Prins, Stella
Counsell, John
Vergani, Paola
Hart, Stephen
Baines, Deborah
MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2023, 30 : 593 - 605
[8] CFTR NONSENSE MUTATIONS G542X AND W1282X ASSOCIATED WITH SEVERE REDUCTION OF CFTR MESSENGER-RNA IN NASAL EPITHELIAL-CELLS
HAMOSH, A
ROSENSTEIN, BJ
CUTTING, GR
HUMAN MOLECULAR GENETICS, 1992, 1 (07) : 542 - 544
[9] Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors
Krishnamurthy, Sateesh
Traore, Soumba
Cooney, Ashley L.
Brommel, Christian M.
Kulhankova, Katarina
Sinn, Patrick L.
Newby, Gregory A.
Liu, David R.
McCray, Paul B., Jr.
NUCLEIC ACIDS RESEARCH, 2021, 49 (18) : 10558 - 10572
[10] Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells
Bulcaen, Mattijs
Kortleven, Pheline
Liu, Ronald B.
Maule, Giulia
Dreano, Elise
Kelly, Mairead
Ensinck, Marjolein M.
Thierie, Sam
Smits, Maxime
Ciciani, Matteo
Hatton, Aurelie
Chevalier, Benoit
Ramalho, Anabela S.
Solvas, Xavier Casadevall i
Debyser, Zeger
Vermeulen, Francois
Gijsbers, Rik
Sermet-Gaudelus, Isabelle
Cereseto, Anna
Carlon, Marianne S.
CELL REPORTS MEDICINE, 2024, 5 (05)

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