Genetic medicines for CF: Hype versus reality

A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116 · see citing works

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

Zetsche, B., Gootenberg, J. S., Abudayyeh, O. O., Slaymaker, I. M., Makarova, K. S., Essletzbichler, P., Volz, S. E., Joung, J., John van der, O. O. S. T., Regev, A., et al. (2015) · Cell · doi:10.1016/j.cell.2015.09.038 · Cited by 4,750 · see citing works

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects

Kleinstiver, B. P., Pattanayak, V., Prew, M. S., Tsai, S. Q., Nguyen, N. T., Zheng, Z., & Joung, J. K. (2016) · Nature · doi:10.1038/nature16526 · Cited by 2,673 · see citing works

Rationally engineered Cas9 nucleases with improved specificity

Slaymaker, I. M., Gao, L., Zetsche, B., Scott, D. A., Yan, W. X., & Zhang, F. (2016) · Science · doi:10.1126/science.aad5227 · Cited by 2,414 · see citing works

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

Karikó, K., Muramatsu, H., Welsh, F. A., Ludwig, J., Kato, H., Akira, S., & Weissman, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.200 · Cited by 1,930 · see citing works

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Urnov, F. D., Miller, J. C., Lee, Y.-L., Beausejour, C. M., Rock, J. M., Augustus, S., Jamieson, A. C., Porteus, M. H., Gregory, P. D., & Holmes, M. C. (2005) · Nature · doi:10.1038/nature03556 · Cited by 1,642 · see citing works

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

Schwank, G., Koo, B.-K., Sasselli, V., Dekkers, J. F., Heo, I., Demircan, T., Sasaki, N., Boymans, S., Cuppen, E., van der Ent, C. K., et al. (2013) · Cell Stem Cell · doi:10.1016/j.stem.2013.11.002 · Cited by 1,424 · see citing works

Gene Editing of <i>CCR5</i> in Autologous CD4 T Cells of Persons Infected with HIV

Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1300662 · Cited by 1,357 · see citing works

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., Baldoli, C., Martino, S., Calabria, A., Canale, S., et al. (2013) · Science · doi:10.1126/science.1233158 · Cited by 1,180 · see citing works

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

Richardson, C. D., Ray, G. J., DeWitt, M. A., Curie, G. L., & Corn, J. E. (2016) · Nature Biotechnology · doi:10.1038/nbt.3481 · Cited by 1,098 · see citing works

Cystic fibrosis: A worldwide analysis ofCFTR mutations?correlation with incidence data and application to screening

Bobadilla, J. L., Macek, M., Fine, J. P., & Farrell, P. M. (2002) · Human Mutation · doi:10.1002/humu.10041 · Cited by 1,032 · see citing works

Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination

Smithies, O., Gregg, R. G., Boggs, S. S., Koralewski, M. A., & Kucherlapati, R. (1985) · Nature · doi:10.1038/317230a0 · Cited by 977 · see citing works

In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium

Rosenfeld, M. A., Yoshimura, K., Trapnell, B. C., Yoneyama, K., Rosenthal, E. R., Dalemans, W., Fukayama, M., Bargon, J., Stier, L. E., Stratford-Perricaudet, L. D., et al. (1992) · Cell · doi:10.1016/0092-8674(92)90213-v · Cited by 930 · see citing works

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

Karikó, K., Muramatsu, H., Ludwig, J., & Weissman, D. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr695 · Cited by 898 · see citing works

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Lombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y.-L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1353 · Cited by 845 · see citing works

Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis

Crystal, R. G., McElvaney, N. G., Rosenfeld, M. A., Chu, C.-S., Mastrangeli, A., Hay, J. G., Brody, S. L., Jaffe, H. A., Eissa, N. T., & Danel, C. J. (1994) · Nature Genetics · doi:10.1038/ng0994-42 · Cited by 824 · see citing works

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

Kormann, M. S. D., Hasenpusch, G., Aneja, M. K., Nica, G., Flemmer, A. W., Herber-Jonat, S., Huppmann, M., Mays, L. E., Illenyi, M., Schams, A., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1733 · Cited by 733 · see citing works

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

Mussolino, C., Morbitzer, R., Lütge, F., Dannemann, N., Lahaye, T., & Cathomen, T. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr597 · Cited by 714 · see citing works

Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

Caplen, N. J., Alton, E. W. F. W., Mddleton, P. G., Dorin, J. R., Stevenson, B. J., Gao, X., Durham, S. R., Jeffery, P. K., Hodson, M. E., Coutelle, C. C., et al. (1995) · Nature Medicine · doi:10.1038/nm0195-39 · Cited by 710 · see citing works

Origins of Cystic Fibrosis Lung Disease

Stoltz, D. A., Meyerholz, D. K., & Welsh, M. J. (2015) · New England Journal of Medicine · doi:10.1056/nejmra1300109 · Cited by 669 · see citing works

Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis.

Zabner, J., Couture, L., Gregory, R., Graham, S., Smith, A. E., & Welsh, M. (1993) · Cell · doi:10.1016/0092-8674(93)80063-k · Cited by 657 · see citing works

High frequency targeting of genes to specific sites in the mammalian genome

THOMAS, K. (1986) · Cell · doi:10.1016/0092-8674(86)90463-0 · Cited by 635 · see citing works

Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

Sosnay, P. R., Siklosi, K. R., Van Goor, F., Kaniecki, K., Yu, H., Sharma, N., Ramalho, A. S., Amaral, M. D., Dorfman, R., Zielenski, J., et al. (2013) · Nature Genetics · doi:10.1038/ng.2745 · Cited by 630 · see citing works

Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies

Poirot, L., Philip, B., Schiffer-Mannioui, C., Le Clerre, D., Chion-Sotinel, I., Derniame, S., Potrel, P., Bas, C., Lemaire, L., Galetto, R., et al. (2015) · Cancer Research · doi:10.1158/0008-5472.can-14-3321 · Cited by 605 · see citing works

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Li, H., Haurigot, V., Doyon, Y., Li, T., Wong, S. Y., Bhagwat, A. S., Malani, N., Anguela, X. M., Sharma, R., Ivanciu, L., et al. (2011) · Nature · doi:10.1038/nature10177 · Cited by 548 · see citing works

Genetic basis of variable exon 9 skipping in cystic fibrosis transmembrane conductance regulator mRNA

Chu, C.-S., Trapnell, B. C., Curristin, S. M., Cutting, G. R., & Crystal, R. G. (1993) · Nature Genetics · doi:10.1038/ng0293-151 · Cited by 516 · see citing works

Airway Surface Dehydration in Cystic Fibrosis: Pathogenesis and Therapy

Boucher, R. C. (2007) · Annual Review of Medicine · doi:10.1146/annurev.med.58.071905.105316 · Cited by 509 · see citing works

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

Palfi, S., Gurruchaga, J. M., Ralph, G. S., Lepetit, H., Lavisse, S., Buttery, P. C., Watts, C., Miskin, J., Kelleher, M., Deeley, S., et al. (2014) · The Lancet · doi:10.1016/s0140-6736(13)61939-x · Cited by 452 · see citing works

Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy

Hyde, S. C., Gill, D. R., Higgins, C. F., Trezise, A. E. O., MacVinish, L. J., Cuthbert, A. W., Ratcliff, R. A., Evans, M. J., & Colledge, W. H. (1993) · Nature · doi:10.1038/362250a0 · Cited by 435 · see citing works

Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial

Alton, E. W. F. W., Stern, M., Farley, R., Jaffé, A., Chadwick, S., Phillips, J., Davies, J. C., Smith, S. N., Browning, J. A., Davies, M., et al. (1999) · The Lancet · doi:10.1016/s0140-6736(98)06532-5 · Cited by 434 · see citing works

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Alton, E. W. F. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A. C., Brand, J., Buchan, R., Calcedo, R., et al. (2015) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(15)00245-3 · Cited by 428 · see citing works

Germ line transmission and expression of a corrected HPRT gene produced by gene targeting in embryonic stem cells

Thompson, S., Clarke, A. R., Pow, A. M., Hooper, M., & Melton, D. W. (1989) · Cell · doi:10.1016/0092-8674(89)90905-7 · Cited by 425 · see citing works

Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes

Li, T., Huang, S., Zhao, X., Wright, D. A., Carpenter, S. L., Spalding, M. H., Weeks, D. P., & Yang, B. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr188 · Cited by 414 · see citing works

Repeated Adeno-Associated Virus Serotype 2 Aerosol-Mediated Cystic Fibrosis Transmembrane Regulator Gene Transfer to the Lungs of Patients With Cystic Fibrosis

Moss, R. B., Rodman, D. M., Spencer, L. T., Aitken, M. L., Zeitlin, P. L., Waltz, D. A., Milla, C. E., Brody, A. S., Clancy, J. P., Ramsey, B. W., et al. (2004) · CHEST Journal · doi:10.1378/chest.125.2.509 · Cited by 389 · see citing works

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

Firth, A. L., Menon, T., Parker, G. S., Qualls, S. J., Lewis, B. M., Ke, E., Dargitz, C., Wright, R., Khanna, A., Gage, F. H., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.062 · Cited by 327 · see citing works

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

Holkers, M., Maggio, I., Liu, J., Janssen, J. M., Miselli, F., Mussolino, C., Recchia, A., Cathomen, T., & Gonçalves, M. A. F. V. (2013) · Nucleic Acids Research · doi:10.1093/nar/gks1446 · Cited by 312 · see citing works

Obligate Ligation-Gated Recombination (ObLiGaRe): Custom-designed nuclease-mediated targeted integration through nonhomologous end joiningPreprint

Maresca, M., Lin, V. G., Guo, N., & Yang, Y. (2012) · Genome Research · doi:10.1101/gr.145441.112 · Cited by 310 · see citing works

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

Hyde, S. C., Pringle, I. A., Abdullah, S., Lawton, A. E., Davies, L. A., Varathalingam, A., Nunez-Alonso, G. A., Green, A.-M., Bazzani, R. P., Jones, S., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1399 · Cited by 308 · see citing works

Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo

Kobinger, G. P., Weiner, D. J., Yu, Q., & Wilson, J. M. (2001) · Nature Biotechnology · doi:10.1038/85664 · Cited by 307 · see citing works

Gene Conversion Tracts from Double-Strand Break Repair in Mammalian Cells

Elliott, B., Richardson, C. A., Winderbaum, J., Nickoloff, J. A., & Jasin, M. (1998) · Molecular and Cellular Biology · doi:10.1128/mcb.18.1.93 · Cited by 305 · see citing works

Airway acidification initiates host defense abnormalities in cystic fibrosis mice

Shah, V. S., Meyerholz, D. K., Tang, X. X., Reznikov, L. R., Alaiwa, M. H. A., Ernst, S. E., Karp, P. H., Wohlford-Lenane, C., Heilmann, K. P., Leidinger, M., et al. (2016) · Science · doi:10.1126/science.aad5589 · Cited by 285 · see citing works

Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing

Miyaoka, Y., Berman, J. R., Cooper, S., Mayerl, S. J., Chan, A. H., Zhang, B., Karlin-Neumann, G. A., & Conklin, B. R. (2016) · Scientific Reports · doi:10.1038/srep23549 · Cited by 258 · see citing works

Efficient gene transfer to airway epithelium using recombinant Sendai virus

Yonemitsu, Y., Kitson, C., Ferrari, S., Farley, R., Griesenbach, U., Judd, D., Steel, R., Scheid, P., Zhu, J., Jeffery, P. K., et al. (2000) · Nature Biotechnology · doi:10.1038/79463 · Cited by 244 · see citing works

Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis

Hyde, S. C., Southern, K., Gileadi, U., Fitzjohn, E., Mofford, K., Waddell, B., Gooi, H. C., Goddard, C., Hannavy, K., Smyth, S., et al. (2000) · Gene Therapy · doi:10.1038/sj.gt.3301212 · Cited by 230 · see citing works

Rapid Expansion of Human Epithelial Stem Cells Suitable for Airway Tissue Engineering

Butler, C. R., Hynds, R. E., Gowers, K. H. C., Lee, D. D. H., Brown, J., Crowley, C., TEIXEIRA, V. H., Smith, C. M., Urbani, L., Hamilton, N., et al. (2016) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201507-1414oc · Cited by 217 · see citing works

Safety and Biological Efficacy of an Adeno‐Associated Virus Vector–Cystic Fibrosis Transmembrane Regulator (AAV‐CFTR) in the Cystic Fibrosis Maxillary Sinus

Wagner, J. A., Messner, A. H., Moran, M. L., Daifuku, R., Kouyama, K., Desch, J. K., Manley, S. A., Norbash, A., Conrad, C., Friborg, S., et al. (1999) · The Laryngoscope · doi:10.1097/00005537-199902000-00017 · Cited by 209 · see citing works

Mouse models of cystic fibrosis: Phenotypic analysis and research applications

Wilke, M., Buijs‐Offerman, R. M., Aarbiou, J., Colledge, W. H., Sheppard, D. N., Touqui, L., Bot, A. G. M., Jorna, H., de Jonge, H. R., & Scholte, B. J. (2011) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(11)60020-9 · Cited by 200 · see citing works

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

Crane, A. M., Kramer, P., Bui, J. H., Chung, W. J., Li, X. S., Gonzalez‐Garay, M. L., Hawkins, F. J., Liao, W., Mora, D. T., Choi, S., et al. (2015) · Stem Cell Reports · doi:10.1016/j.stemcr.2015.02.005 · Cited by 194 · see citing works

CFTR Delivery to 25% of Surface Epithelial Cells Restores Normal Rates of Mucus Transport to Human Cystic Fibrosis Airway Epithelium

Zhang, L., Button, B. M., Gabriel, S. E., Burkett, S. E., Yan, Y. K., Skiadopoulos, M. H., Dang, Y. L., Vogel, L., McKay, T. R., Mengos, A. E., et al. (2009) · PLoS Biology · doi:10.1371/journal.pbio.1000155 · Cited by 194 · see citing works

Lentivirus pre-packed with Cas9 protein for safer gene editing

Choi, J., Dang, Y., Abraham, S., Ma, H., Zhang, J., Guo, H., Cai, Y., Mikkelsen, J. G., Wu, H., Shankar, P., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.27 · Cited by 192 · see citing works

A Clinical Inflammatory Syndrome Attributable to Aerosolized Lipid–DNA Administration in Cystic Fibrosis

Ruiz, F. E., Clancy, J. P., Perricone, M. A., Bebok, Z. M., Hong, J. S., Cheng, S. H., Meeker, D. P., Young, K. R., Schoumacher, R., Weatherly, M. R., et al. (2001) · Human Gene Therapy · doi:10.1089/104303401750148667 · Cited by 184 · see citing works

Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy.

Griesenbach, U., & Alton, E. (2009) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2008.09.010 · Cited by 163 · see citing works

Characterization of Defects in Ion Transport and Tissue Development in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Knockout Rats

Tuggle, K. L., Birket, S. E., Cui, X., Hong, J. S., Warren, J., Reid, L., Chambers, A., Ji, D., Gamber, K. M., Chu, K. K., et al. (2014) · PLoS ONE · doi:10.1371/journal.pone.0091253 · Cited by 159 · see citing works

Origins of Cystic Fibrosis Lung Disease

Stoltz, D. A., Meyerholz, D. K., & Welsh, M. J. (2015) · New England Journal of Medicine · doi:10.1056/nejmc1502191 · Cited by 153 · see citing works

Cystic Fibrosis Transmembrane Conductance Regulator Intracellular Processing, Trafficking, and Opportunities for Mutation-Specific Treatment

Rogan, M. P., Stoltz, D. A., & Hornick, D. B. (2011) · CHEST Journal · doi:10.1378/chest.10-2077 · Cited by 152 · see citing works

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

Mahiny, A. J., Dewerth, A., Mays, L. E., Alkhaled, M., Mothes, B., Malaeksefat, E., Loretz, B., Rottenberger, J., Brosch, D. M., Reautschnig, P., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3241 · Cited by 139 · cited 1× · see citing works

Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium

McNeer, N. A., Anandalingam, K. K., Fields, R. J., Caputo, C. R., Kopic, S., Gupta, A., Quijano, E., Polikoff, L. A., Kong, Y., Bahal, R., et al. (2015) · Nature Communications · doi:10.1038/ncomms7952 · Cited by 139 · see citing works

Gene transfer of CFTR to airway epithelia: low levels of expression are sufficient to correct Cl − transport and overexpression can generate basolateral CFTR

Farmen, S. L., Karp, P. H., Ng, P., Palmer, D. J., Koehler, D. R., Hu, J., Beaudet, A. L., Zabner, J., & Welsh, M. J. (2005) · American Journal of Physiology-Lung Cellular and Molecular Physiology · doi:10.1152/ajplung.00049.2005 · Cited by 138 · see citing works

Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes

Mitomo, K., Uta Griesenbach, D., Inoue, M., Somerton, L., Meng, C., Akiba, E., Tabata, T., Ueda, Y., Frankel, G. M., Farley, R., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.13 · Cited by 135 · see citing works

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

Stoltz, D. A., Rokhlina, T., Ernst, S. E., Pezzulo, A. A., Ostedgaard, L. S., Karp, P. H., Samuel, M., Reznikov, L. R., Rector, M. V., Gansemer, N. D., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci68867 · Cited by 119 · see citing works

New animal models of cystic fibrosis

Keiser, N. W., & Engelhardt, J. F. (2011) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0b013e32834b14c9 · Cited by 114 · see citing works

Understanding how cystic fibrosis mutations disrupt CFTR function: From single molecules to animal models

Wang, Y., Wrennall, J. A., Cai, Z., Li, H., & Sheppard, D. N. (2014) · The International Journal of Biochemistry & Cell Biology · doi:10.1016/j.biocel.2014.04.001 · Cited by 114 · see citing works

Modified Foxp3 mRNA protects against asthma through an IL-10-dependent mechanism

Mays, L. E., Ammon-Treiber, S., Mothes, B., Alkhaled, M., Rottenberger, J., Müller-Hermelink, E. S., Grimm, M., Mezger, M., Beer-Hammer, S., von Stebut, E., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci65351 · Cited by 113 · see citing works

Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy

Uta Griesenbach, D., Inoue, M., Meng, C., Farley, R., Chan, M., Newman, N. K., Brum, A. M., You, J., Kerton, A., Shoemark, A., et al. (2012) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201206-1056oc · Cited by 108 · see citing works

Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments

Goncz, K. K., Kunzelmann, K., Xu, Z., & Gruenert, D. C. (1998) · Human Molecular Genetics · doi:10.1093/hmg/7.12.1913 · Cited by 107 · see citing works

Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases

Cai, Y., Bak, R. O., & Mikkelsen, J. G. (2014) · eLife · doi:10.7554/elife.01911 · Cited by 105 · see citing works

Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung

McLachlan, G., Davidson, H. M., Holder, E. L., Davies, L. A., Pringle, I. A., Jones, S., Baker, A., Tennant, P. A., Gordon, C., Vrettou, C., et al. (2011) · Gene Therapy · doi:10.1038/gt.2011.55 · Cited by 104 · see citing works

Cystic Fibrosis Gene Therapy in the UK and Elsewhere

Uta Griesenbach, D., Pytel, K. M., & Alton, E. W. F. W. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.027 · Cited by 94 · see citing works

Lentivirus Vector Can Be Readministered to Nasal Epithelia without Blocking Immune Responses

Sinn, P. L., Arias, A. C., Brogden, K. A., & McCray, P. B. (2008) · Journal of Virology · doi:10.1128/jvi.00227-08 · Cited by 94 · see citing works

Gene targeting of CFTR DNA in CF epithelial cells.

Kunzelmann, K., Legendre, J., Knoell, D. L., Escobar, L. C., Xu, Z., & Gruenert, D. C. (1996) · PubMed · Cited by 92 · see citing works

Overcoming the Cystic Fibrosis Sputum Barrier to Leading Adeno-associated Virus Gene Therapy Vectors

Schuster, B. S., Kim, A. J., Kays, J. C., Kanzawa, M. M., Guggino, W. B., Boyle, M., Rowe, S. M., Muzyczka, N., Suk, J. S., & Hanes, J. S. (2014) · Molecular Therapy · doi:10.1038/mt.2014.89 · Cited by 90 · see citing works

Correction of the ΔF508 Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator Gene by Zinc-Finger Nuclease Homology-Directed Repair

Lee, C. M., Flynn, R., Hollywood, J. A., Scallan, M. F., & Harrison, P. T. (2012) · BioResearch open access · doi:10.1089/biores.2012.0218 · Cited by 84 · see citing works

Pseudotyped Lentivirus Vectors Derived from Simian Immunodeficiency Virus SIVagm with Envelope Glycoproteins from Paramyxovirus

Kobayashi, M., Iida, A., Ueda, Y., & Hasegawa, M. (2003) · Journal of Virology · doi:10.1128/jvi.77.4.2607-2614.2003 · Cited by 76 · see citing works

Expression of ΔF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR

Goncz, K., Colosimo, A., Dallapiccola, B., Gagné, L., Hong, K., Novelli, G., Papahadjopoulos, D., Sawa, T., Schreier, H., Wiener-Kronish, J. P., et al. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301476 · Cited by 75 · see citing works

Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer

Munye, M. M., Tagalakis, A. D., Barnes, J. L., Brown, R. E., McAnulty, R. J., Howe, S. J., & Hart, S. L. (2016) · Scientific Reports · doi:10.1038/srep23125 · Cited by 72 · see citing works

Cellular and viral splicing factors can modify the splicing pattern of CFTR transcripts carrying splicing mutations

Nissim‐Rafinia, M. (2000) · Human Molecular Genetics · doi:10.1093/hmg/9.12.1771 · Cited by 70 · see citing works

TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells

Ramalingam, S., Annaluru, N., Kandavelou, K., & Chandrasegaran, S. (2014) · Current Gene Therapy · doi:10.2174/1566523214666140918101725 · Cited by 64 · see citing works

Poly (D, L-lactide-co-glycolide)/DNA microspheres to facilitate prolonged transgene expression in airway epithelium in vitro, ex vivo and in vivo

Stern, M., Ulrich, K., Geddes, D. M., & Alton, E. W. F. W. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301994 · Cited by 62 · see citing works

Gene Therapy for Primary Immunodeficiencies

Rivat, C., Santilli, G., Gaspar, H. B., & Thrasher, A. J. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.116 · Cited by 60 · see citing works

A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial

Alton, E. W. F. W., Boyd, A., Porteous, D. J., Davies, G., Davies, J. C., Uta Griesenbach, D., Higgins, T., Gill, D. R., Hyde, S. C., & Innes, J. A. (2015) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201506-1193le · Cited by 56 · see citing works

Lysophosphatidylcholine as an adjuvant for lentiviral vector mediated gene transfer to airway epithelium: effect of acyl chain length

Cmielewski, P., Anson, D. S., & Parsons, D. (2010) · Respiratory Research · doi:10.1186/1465-9921-11-84 · Cited by 52 · see citing works

Lentiviral Vector Gene Transfer to Porcine Airways

Sinn, P. L., Cooney, A. L., Oakland, M., Dylla, D. E., Wallen, T. J., Pezzulo, A. A., Chang, E. H., & McCray, P. B. (2012) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2012.47 · Cited by 47 · see citing works

High efficiency gene transfer to airways of mice using influenza hemagglutinin pseudotyped lentiviral vectors

Patel, M., Giddings, A. M., Sechelski, J. B., & Olsen, J. C. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2695 · Cited by 44 · see citing works

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

Suzuki, S., Sargent, R. G., Illek, B., Fischer, H., Esmaeili-Shandiz, A., Yezzi, M. J., Lee, A., Yang, Y., Kim, S., Renz, P., et al. (2016) · Molecular Therapy: Nucleic Acids · doi:10.1038/mtna.2015.43 · Cited by 43 · see citing works

Native Small Airways Secrete Bicarbonate

Shamsuddin, A. K. M., & Quinton, P. M. (2013) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2013-0418oc · Cited by 41 · see citing works

Reversal of cystic fibrosis phenotype in a cultured Δ508 cystic fibrosis transmembrane conductance regulator cell line by oligonucleotide insertion

Zamecnik, P. C., Raychowdhury, M. K., TABATADZE, D. R., & Cantiello, H. F. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0401933101 · Cited by 40 · see citing works

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung

Alton, E. W. F. W., Boyd, A., Cheng, S. H., Davies, J. C., Davies, L. A., Dayan, A. D., Gill, D. R., Uta Griesenbach, D., Higgins, T., Hyde, S. C., et al. (2013) · Gene Therapy · doi:10.1038/gt.2013.61 · Cited by 39 · see citing works

Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice

Ostrowski, L. E., Yin, W., Diggs, P. S., Rogers, T. D., O’Neal, W. K., & Grubb, B. R. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302994 · Cited by 37 · see citing works

The Committee for Advanced Therapies' of the European Medicines Agency Reflection Paper on Management of Clinical Risks Deriving from Insertional Mutagenesis

Aiuti, A., Cossu, G., de Felipe, P., Galli, M. C., Narayanan, G., Renner, M., Stahlbom, A., Schneider, C. K., & Voltz-Girolt, C. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.119 · Cited by 35 · see citing works

The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep

Alton, E. W. F. W., Baker, A., Baker, E., Boyd, A., Cheng, S. H., Coles, R. L., Collie, D., Davidson, H. E., Davies, J. C., Gill, D. R., et al. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.09.023 · Cited by 34 · see citing works

Validation of Nasal Potential Difference Measurements in Gut-Corrected CF Knockout Mice

Uta Griesenbach, D., Smith, S. N., Farley, R., Singh, C., & Alton, E. W. F. W. (2008) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2007-0385oc · Cited by 25 · see citing works

Ion transport across CF and normal murine olfactory and ciliated epithelium

Grubb, B. R., Rogers, T. D., Boucher, R. C., & Ostrowski, L. E. (2009) · American Journal of Physiology-Cell Physiology · doi:10.1152/ajpcell.00578.2008 · Cited by 21 · see citing works

Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer

Uta Griesenbach, D., Jones, S., Holder, E. L., Munkonge, F. M., Wodehouse, T., Smith, S. N., Wasowicz, M. Y., Pringle, I. A., Casamayor, I., Chan, M., et al. (2009) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2009-0075oc · Cited by 19 · see citing works

Lung gene therapy—How to capture illumination from the light already present in the tunnel

Xia, E., Munegowda, M. A., Cao, H., & Hu, J. (2014) · Genes & Diseases · doi:10.1016/j.gendis.2014.06.001 · Cited by 17 · see citing works

WS1.5 Restoration of the CFTR function by antisense oligonucleotide splicing modulation

Sinai, M. I., Wilton, S. D., & Kerem, B. (2014) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(14)60008-4 · Cited by 4 · see citing works

Cystic fibrosis

Shteinberg, M., Haq, I. J., Polineni, D., & Davies, J. C. (2021) · The Lancet · doi:10.1016/s0140-6736(20)32542-3 · Cited by 682 · see citing works

Anion carriers as potential treatments for cystic fibrosis: transport in cystic fibrosis cells, and additivity to channel-targeting drugs

Li, H., Valkenier, H., Thorne, A. G., Dias, C. M., Cooper, J. A., Kieffer, M., Busschaert, N., Gale, P. A., Sheppard, D. N., & Davis, A. P. (2019) · Chemical Science · doi:10.1039/c9sc04242c · Cited by 144 · see citing works

Future therapies for cystic fibrosis

Allen, L., Allen, L. J., Carr, S. B., Davies, G., Downey, D. G., Egan, M. E., Forton, J. T., Gray, R. D., HAWORTH, C. S., Horsley, A. R., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-36244-2 · Cited by 143 · see citing works

Cas9/gRNA targeted excision of cystic fibrosis-causing deep-intronic splicing mutations restores normal splicing of CFTR mRNA

Sanz, D. J., Hollywood, J. A., Scallan, M. F., & Harrison, P. T. (2017) · PLOS ONE · doi:10.1371/journal.pone.0184009 · Cited by 92 · see citing works

Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Maule, G., Casini, A., Montagna, C., Ramalho, A. S., De Boeck, K., Debyser, Z., Carlon, M. S., Petris, G., & Cereseto, A. (2019) · Nature Communications · doi:10.1038/s41467-019-11454-9 · Cited by 86 · see citing works

Personalized or Precision Medicine? The Example of Cystic Fibrosis

Marson, F. A. L. (2017) · Frontiers in Pharmacology · doi:10.3389/fphar.2017.00390 · Cited by 70 · see citing works

New horizons for cystic fibrosis treatment

Fajac, I., & Boeck, K. D. (2016) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2016.11.009 · Cited by 58 · see citing works

Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique

Marangi, M., & Pistritto, G. (2018) · Frontiers in Pharmacology · doi:10.3389/fphar.2018.00396 · Cited by 58 · see citing works

Toward inclusive therapy with CFTR modulators: Progress and challenges

Guimbellot, J. S., Sharma, J., & Rowe, S. M. (2017) · Pediatric Pulmonology · doi:10.1002/ppul.23773 · Cited by 45 · see citing works

Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing

Erwood, S., Laselva, O., Bily, T. M. I., Brewer, R. A., Rutherford, A. H., Bear, C. E., & Ivakine, E. A. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.05.002 · Cited by 39 · see citing works

Genetic therapies for cystic fibrosis lung disease

Hart, S. L., & Harrison, P. T. (2017) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2017.10.006 · Cited by 37 · see citing works

Effective viral-mediated lung gene therapy: is airway surface preparation necessary?

McCarron, A., Cmielewski, P., Drysdale, V., Parsons, D., & Donnelley, M. (2022) · Gene Therapy · doi:10.1038/s41434-022-00332-7 · Cited by 36 · see citing works

One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies

Ensinck, M. M., & Carlon, M. S. (2022) · Cells · doi:10.3390/cells11121868 · Cited by 31 · see citing works

The Interface of Gene Editing with Regenerative Medicine

Farag, V., Devey, E. A., & Leong, K. W. (2024) · Engineering · doi:10.1016/j.eng.2024.10.019 · Cited by 30 · see citing works

Gene and Base Editing as a Therapeutic Option for Cystic Fibrosis—Learning from Other Diseases

Mention, K., Santos, L., & Harrison, P. T. (2019) · Genes · doi:10.3390/genes10050387 · Cited by 30 · see citing works

AJRCCM : 100-Y ear A nniversary . Progress along the Pathway of Discovery Leading to Treatment and Cure of Cystic Fibrosis

Ramsey, B. W., & Welsh, M. J. (2017) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201702-0266ed · Cited by 27 · see citing works

Current landscape of cystic fibrosis gene therapy

Plasschaert, L. W., MacDonald, K. D., & Moffit, J. S. (2024) · Frontiers in Pharmacology · doi:10.3389/fphar.2024.1476331 · Cited by 27 · see citing works

Nucleic acid therapies for cystic fibrosis

Sasaki, S., & Guo, S. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0696 · Cited by 24 · see citing works

Applications of CRISPR systems in respiratory health: Entering a new ‘red pen’ era in genome editing

Moses, C., & Kaur, P. (2019) · Respirology · doi:10.1111/resp.13527 · Cited by 22 · see citing works

Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis

Cuyx, S., & Boeck, K. D. (2019) · Seminars in Respiratory and Critical Care Medicine · doi:10.1055/s-0039-1696664 · Cited by 21 · see citing works

The revolution of personalized pharmacotherapies for cystic fibrosis: what does the future hold?

Oliver, K. E., Carlon, M. S., Pedemonte, N., & Lopes‐Pacheco, M. (2023) · Expert Opinion on Pharmacotherapy · doi:10.1080/14656566.2023.2230129 · Cited by 18 · see citing works

Re-imagining cystic fibrosis care: next generation thinking

Rang, C., Keating, D., Wilson, J. W., & Kotsimbos, T. C. (2020) · European Respiratory Journal · doi:10.1183/13993003.02443-2019 · Cited by 18 · see citing works

A beginner's guide to gene editing

Harrison, P. T., & Hart, S. L. (2017) · Experimental Physiology · doi:10.1113/ep086047 · Cited by 17 · see citing works

Roadmap for an early gene therapy for cystic fibrosis airway disease

Carlon, M. S., Vidović, D., & Birket, S. E. (2017) · Prenatal Diagnosis · doi:10.1002/pd.5164 · Cited by 16 · see citing works

Rewriting CFTR to cure cystic fibrosis

Maule, G., Ensinck, M. M., Bulcaen, M., & Carlon, M. S. (2021) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2020.12.018 · Cited by 16 · see citing works

Assembly and Functional Analysis of an S/MAR Based Episome with the Cystic Fibrosis Transmembrane Conductance Regulator Gene

Rocco, D., Pompili, B., Castellani, S., Morini, E., Cavinato, L., Cimino, G. G., Mariggiò, M. A., Guarnieri, S., Conese, M., Porto, P. D., et al. (2018) · International Journal of Molecular Sciences · doi:10.3390/ijms19041220 · Cited by 13 · see citing works

Anticipating New Treatments for Cystic Fibrosis: A Global Survey of Researchers

Cabral, B. P., Terlizzi, V., Laselva, O., Filho, C. G. C., & Mota, F. B. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11051283 · Cited by 12 · see citing works

Worldwide trends in heart and lung transplantation: Guarding the most precious gift ever

von Dossow, V., Costa, J. A., D’Ovidio, F., & Marczin, N. (2017) · Best practice & research. Clinical anaesthesiology · doi:10.1016/j.bpa.2017.08.001 · Cited by 12 · see citing works

Current Practices and Potential Nanotechnology Perspectives for Pain Related to Cystic Fibrosis

Trandafir, L. M., Leon, M. M., Frăsinariu, O. E., Baciu, G., Dodi, G., & Cojocaru, E. (2019) · Journal of Clinical Medicine · doi:10.3390/jcm8071023 · Cited by 12 · see citing works

Will Airway Gene Therapy for Cystic Fibrosis Improve Lung Function? New Imaging Technologies Can Help Us Find Out

Parsons, D., & Donnelley, M. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.153 · Cited by 11 · see citing works

Powerful tools for genetic modification: Advances in gene editing

Roesch, E. A., & Drumm, M. L. (2017) · Pediatric Pulmonology · doi:10.1002/ppul.23791 · Cited by 10 · see citing works

CFTR and Cystic Fibrosis: A Need for Personalized Medicine

Bradbury, N. A. (2020) · Physiology in health and disease · doi:10.1007/978-3-030-55454-5_15 · Cited by 5 · see citing works

CFTR and Cystic Fibrosis: A Need for Personalized Medicine

Bradbury, N. A. (2015) · Springer eBooks · doi:10.1007/978-1-4939-3366-2_24 · Cited by 5 · see citing works

The patient voice in research — Supporting actor or starring role?

Rowbotham, N. J., & Smyth, A. R. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.03.001 · Cited by 5 · see citing works

Precision medicine advances in cystic fibrosis: Exploring genetic pathways for targeted therapies

Abinesh, R. S., Madhav, R., Sastri, K. T., Meghana, G. S., Akhila, A. R., & Balamuralidhara, V. (2024) · Life Sciences · doi:10.1016/j.lfs.2024.123186 · Cited by 4 · see citing works

Molecular Physiology and Pharmacology of the Cystic Fibrosis Transmembrane Conductance Regulator

Al-Salmani, M., Sondo, E., Balut, C., Sheppard, D. N., Singh, A. K., & Pedemonte, N. (2020) · Physiology in health and disease · doi:10.1007/978-3-030-55454-5_16 · Cited by 4 · see citing works

New era of cystic fibrosis: Full mutational analysis and personalized therapy

Lucarelli, M. (2017) · World Journal of Medical Genetics · doi:10.5496/wjmg.v7.i1.1 · Cited by 3 · see citing works

Sistema CRISPR/Cas: Edición genómica de precisión

Maidana, R. L. B. R., Méndez-Ferreira, A., Candia, N. B., Nara, E., & Ríos, D. F. (2020) · Memorias del Instituto de Investigaciones en Ciencias de la Salud · doi:10.18004/mem.iics/1812-9528/2020.018.01.97-107 · Cited by 3 · see citing works

Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

Elboraie, S., Kafetzis, K. N., Shrivastava, R., & Tagalakis, A. D. (2021) · IntechOpen eBooks · doi:10.5772/intechopen.92434 · Cited by 2 · see citing works

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Targeted Therapeutics for Cystic Fibrosis

Veselu, D., Yeh, H., Rodrat, M., Bradbury, J. D., Fornies, Y. M., Li, H., Shoemark, D. K., Oliveira, A. S. F., Hwang, T., & Sheppard, D. N. (2024) · Springer eBooks · doi:10.1007/978-3-031-52197-3_8 · Cited by 2 · see citing works

Nucleic acid-based therapeutic strategies for modulator-refractory cystic fibrosis-causing variants

Leahy, M. D., Zollo, I., Clarke, L. A., & Farinha, C. M. (2026) · Human Molecular Genetics · doi:10.1093/hmg/ddag058 · Cited by 1 · see citing works

Overview of CFTR Modulators and Gene Therapy

Rang, C., Kotsimbos, T. C., & Wilson, J. W. (2020) · IntechOpen eBooks · doi:10.5772/intechopen.91022 · Cited by 1 · see citing works

Perspectives on Genetic Medicine for Cystic Fibrosis

Smirnikhina, S. (2022) · Current Gene Therapy · doi:10.2174/1566523222666220720102556 · Cited by 1 · see citing works

Cystic fibrosis

Casey, M., & Jones, A. (2023) · Medicine · doi:10.1016/j.mpmed.2023.08.001 · Cited by 1 · see citing works

Breathing Easier: A Well-tolerated Corrector for F508del

Nichols, D. P. (2017) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201709-1957ed

Cystic Fibrosis: New Insights into Therapeutic Approaches

Tosco, A., Villella, V. R., Raia, V., Kroemer, G., & Maiuri, L. (2019) · Current Respiratory Medicine Reviews · doi:10.2174/1573398x15666190702151613

CFTR Corrector Therapies/New Therapies

Lewis, D. P. (2020) · Springer eBooks · doi:10.1007/978-3-030-25909-9_14

Gene Editing for CF

Drumm, M. L. (2020) · Respiratory medicine · doi:10.1007/978-3-030-42382-7_25

Cystic fibrosis

Ewence, A. E., & Jones, A. (2020) · Medicine · doi:10.1016/j.mpmed.2020.02.003

Cystic Fibrosis – results of CFTR modulators in Croatia

Tješić-Drinković, D., Bambir, I., Tješić-Drinković, D., Dugac, A. V., Bambiar, I., Baretić, M., Basara, L., Bogdanić, A., Bzik, D., Džubur, F., et al. (2023) · Liječnički vjesnik · doi:10.26800/lv-145-supl5-27

Inflammation pulmonaire et rôle des microARN dans la mucoviscidose

Bardin, P. (2019) · theses.fr (ABES) · doi:10.70675/df28d166zb847z4bdbz979az3c6afebed201

Effet des thérapies protéiques sur l’axe intestin-poumon dans la mucoviscidose

Lussac‐Sorton, F. (2024) · doi:10.70675/5167078fzc81dz457az88b6zce4defb6df49

Molecular pathogenesis and emerging genetic targets in alpha-1 antitrypsin deficiency associated lung disease

Kaur, P., Vancha, H., Pal, B., Garg, P., Pamu, S., Yanadaiah, J. P., Sadhu, N. K., Deepa, M., & Yanadaiah, P. (2026) · Human Gene · doi:10.1016/j.humgen.2026.201571

Recent Advances in Targeted Genetic Medicines for Cystic Fibrosis

Tagalakis, A. (2018) · BiblioBoard Library Catalog (Open Research Library)

Gene therapy for cystic fibrosis: Challenges and prospects

Sui, H., Xu, X., Su, Y., Gong, Z., Yao, M., Liu, X., Zhang, T., Jiang, Z., Bai, T., Wang, J., et al. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2022.1015926 · Cited by 51 · see citing works

Gene Therapy for Cystic Fibrosis Lung Disease: Overcoming the Barriers to Translation to the Clinic

Donnelley, M., & Parsons, D. (2018) · Frontiers in Pharmacology · doi:10.3389/fphar.2018.01381 · Cited by 42 · see citing works

Gene therapy for cystic fibrosis: steady progress, should do well

Alton, E. W. F. W., & Geddes, D. M. (1997) · European Respiratory Journal · doi:10.1183/09031936.97.10020257 · Cited by 4 · see citing works

Emerging gene therapies for cystic fibrosis

Miah, K. M., Hyde, S. C., & Gill, D. R. (2019) · Expert Review of Respiratory Medicine · doi:10.1080/17476348.2019.1634547 · Cited by 39 · see citing works