Publications

• Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocol.
  PLoS One Volume: 20 Page(s): e0316721
  01/01/2025 Authors: Swenson CE; Hunt WR; Manfredi C; Beltran DJ; Hong JS; Davis BR; Suzuki S; Barill C; Rab A; Chico C
• Evaluation of elexacaftor-tezacaftor-ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trial.
  Lancet Respir Med Volume: 12 Page(s): 947 - 957
  12/01/2024 Authors: Solomon GM; Linnemann RW; Rich R; Streby A; Buehler B; Hunter E; Vijaykumar K; Hunt WR; Brewington JJ; Rab A
• Translation velocity determines the efficacy of engineered suppressor tRNAs on pathogenic nonsense mutations.
  Nat Commun Volume: 15 Page(s): 2957
  04/05/2024 Authors: Bharti N; Santos L; Davyt M; Behrmann S; Eichholtz M; Jimenez-Sanchez A; Hong JS; Rab A; Sorscher EJ; Albers S
• A Novel 7H-[1,2,4]Triazolo[3,4-b]thiadiazine-based Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Directed toward Treatment of Cystic Fibrosis.
  ACS Med Chem Lett Volume: 14 Page(s): 1338 - 1343
  10/12/2023 Authors: Rab A; Yang X; Tracy WF; Hong JS; Joshi D; Manfredi C; Ponnaluri SS; Kolykhalov AA; Qui M; Fu H
• Elexacaftor/VX-445-mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics.
  J Biol Chem Volume: 299 Page(s): 105242
  10/01/2023 Authors: Kim M; McDonald EF; Sabusap CMP; Timalsina B; Joshi D; Hong JS; Rab A; Sorscher EJ; Plate L
• Elexacaftor/VX-445-mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics.
  bioRxiv
  02/04/2023 Authors: Kim M; McDonald EF; Sabusap CMP; Timalsina B; Joshi D; Hong JS; Rab A; Sorscher EJ; Plate L
• Targeted Gene Insertion for Functional CFTR Restoration in Airway Epithelium.
  Front Genome Ed Volume: 4 Page(s): 847645
  01/01/2022 Authors: Barill C; Suzuki S; Rab A; Sorscher EJ; Davis BR
• A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion.
  Nat Commun Volume: 12 Page(s): 4358
  07/16/2021 Authors: Sharma J; Du M; Wong E; Mutyam V; Li Y; Chen J; Wangen J; Thrasher K; Fu L; Peng N
• Stability Prediction for Mutations in the Cytosolic Domains of Cystic Fibrosis Transmembrane Conductance Regulator.
  J Chem Inf Model Volume: 61 Page(s): 1762 - 1777
  04/26/2021 Authors: Bahia MS; Khazanov N; Zhou Q; Yang Z; Wang C; Hong JS; Rab A; Sorscher EJ; Brouillette CG; Hunt JF
• Derivation of Airway Basal Stem Cells from Human Pluripotent Stem Cells.
  Cell Stem Cell Volume: 28 Page(s): 79 - 95.e8
  01/07/2021 Authors: Hawkins FJ; Suzuki S; Beermann ML; Barill C; Wang R; Villacorta-Martin C; Berical A; Jean JC; Le Suer J; Matte T
• The CFTR P67L variant reveals a key role for N-terminal lasso helices in channel folding, maturation, and pharmacologic rescue.
  J Biol Chem Volume: 296 Page(s): 100598
  01/01/2021 Authors: Sabusap CM; Joshi D; Simhaev L; Oliver KE; Senderowitz H; van Willigen M; Braakman I; Rab A; Sorscher EJ; Hong JS
• G551D mutation impairs PKA-dependent activation of CFTR channel that can be restored by novel GOF mutations.
  Am J Physiol Lung Cell Mol Physiol Volume: 319 Page(s): L770 - L785
  11/01/2020 Authors: Wang W; Fu L; Liu Z; Wen H; Rab A; Hong JS; Kirk KL; Rowe SM
• IPS-DERIVED AIRWAY CELL RESPONSE TO CFTR MODULATION: STUDY OF TRIPLE COMBINATION THERAPY IN CF PATIENTS WITHOUT AN APPROVED MODULATOR
  Volume: 55 Page(s): S207 - S207
  10/01/2020 Authors: Linnemann RW; Streby A; Rab A; Solomon GM; Hunter E; Hailemichael T; Driggers WC; Slaten K; Hathorne H; Hong JS
• IMPROVED FUNCTIONAL RESCUE OF G551D CFTR BY TRIPLE COMBINATION THERAPY
  Volume: 55 Page(s): S188 - S189
  10/01/2020 Authors: Rab A; Suzuki S; Disha J; Barilla C; Goller K; Manfredi C; Flores JK; Streby A; Stecenko A; Feldman RJ
• AIRWAY BASAL CELLS: MAINTAINING STEM CELL PROPERTIES DURING DERIVATION AND EXPANSION
  Volume: 55 Page(s): S236 - S236
  10/01/2020 Authors: Suzuki S; Barilla C; Hawkins F; Rab A; Beermann M; Matthias N; Randell S; Sorscher EJ; Kotton D; Davis BR
• APPLICATION OF IPSC-DERIVED AIRWAY EPITHELIUM FOR EVALUATING THE RESPONSE OF RARE OR UNKNOWN CFTR MUTATIONS TO MODULATORS
  Volume: 55 Page(s): S193 - S193
  10/01/2020 Authors: Barilla C; Suzuki S; Rab A; Goller K; Hong JS; Driggers WC; Flores J; Feldman RJ; Solomon GM; Stecenko A
• UTILIZING YEAST PHENOMICS TO DISCOVER GENE INTERACTION NETWORKS THAT INFLUENCE BIOGENESIS OF CFTR NONSENSE ALLELES
  Volume: 55 Page(s): S118 - S119
  10/01/2020 Authors: Oliver KE; Mao Y; Laflin S; Linscott K; Gaines E; Ali H; Hong JS; Rab A; Sorscher EJ; Hartman JL
• Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction.
  Mol Ther Volume: 28 Page(s): 1684 - 1695
  07/08/2020 Authors: Suzuki S; Crane AM; Anirudhan V; Barill C; Matthias N; Randell SH; Rab A; Sorscher EJ; Kerschner JL; Yin S
• Modeling Cystic Fibrosis (CF) with Induced Pluripotent Stem Cells
  Volume: 201
  01/01/2020 Authors: Wang R; Agrawal P; Schlaeger T; Simone-Roach C; Martino CV; Rab A; Tang Y; Zhang Y; Suzuki S; Davis BR
• Slowing ribosome velocity restores folding and function of mutant CFTR.
  J Clin Invest Volume: 129 Page(s): 5236 - 5253
  12/02/2019 Authors: Oliver KE; Rauscher R; Mijnders M; Wang W; Wolpert MJ; Maya J; Sabusap CM; Kesterson RA; Kirk KL; Rab A
• DIRECTED DIFFERENTIATION OF IPSC INTO AIRWAY BASAL CELLS
  Volume: 54 Page(s): S211 - S212
  10/01/2019 Authors: Suzuki S; Hawkins F; Barilla C; Crane A; Huang X; Beermann M; Rab A; Sorscher EJ; Kotton D; Davis BR
• GENE EDITING OF CYSTIC FIBROSIS AIRWAY BASAL CELLS
  Volume: 54 Page(s): S211 - S211
  10/01/2019 Authors: Suzuki S; Crane A; Anirudhan V; Matthias N; Rab A; Sorscher EJ; Kerschner JL; Yin S; Harris A; Conway A
• CFTR MRNA EXPRESSION ANALYSIS UTILIZING A REPOSITORY OF BANKED PRIMARY AIRWAY EPITHELIAL SAMPLES
  Volume: 54 Page(s): S248 - S248
  10/01/2019 Authors: Manfredi C; Rab A; Sorscher EJ; Hong JS
• GENE EDITING OF W1282X CFTR ONTO A NATIVE GENETIC BACKGROUND, AND IMPLICATIONS FOR IONOCYTE-DEPENDENT TRANSEPITHELIAL TRANSPORT
  Volume: 54 Page(s): S216 - S216
  10/01/2019 Authors: Rab A; Waller L; Gustin J; Manfredi C; Disha J; Lipman C; Hong JS; Sorscher EJ
• PARTIAL RESCUE OF G542X-AND W1282X-CFTR IS ACHIEVED FOLLOWING SUPPRESSION OF SPECIFIC RIBOSOMAL COMPONENTS
  Volume: 54 Page(s): S199 - S200
  10/01/2019 Authors: Oliver KE; Ali H; Rauscher R; Bampi GB; Santos S; Rab A; Hong JS; Ignatova Z; Sorscher EJ; Hartman JL
• VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level.
  Sci Rep Volume: 9 Page(s): 13460
  09/17/2019 Authors: Cui G; Stauffer BB; Imhoff BR; Rab A; Hong JS; Sorscher EJ; McCarty NA
• SUPPRESSION OF PEROXISOME AND RIBOSOMAL CONSTITUENTS PARTIALLY RESTORES PLASMA MEMBRANE LOCALIZATION AND FUNCTION OF W1282X-CFTR
  Volume: 53 Page(s): 209 - 210
  09/01/2018 Authors: Ali H; Icyuz M; Kelly S; Rab A; Hong JS; Bedwell D; Hartman JL; Sorscher EJ; Oliver KE
• CFTR PHOSPHORYLATION LEVEL MODULATES THE EFFICACY OF VX-770-MEDIATED POTENTIATION OF NUMEROUS HUMAN CFTR DISEASE-ASSOCIATED VARIANTS
  Volume: 53 Page(s): 110 - 111
  09/01/2018 Authors: Cui G; Stauffer BB; Imhoff B; Rab A; Hong JS; Sorscher EJ; McCarty NA
• EVALUATION OF W1282X CFTR IN THE FRT MODEL AS A MEANS TO TEST SMALL MOLECULE INTERVENTIONS
  Volume: 53 Page(s): 213 - 213
  09/01/2018 Authors: Manfredi C; Rab A; Hong JS; Joshi D; Mahiou J; Mense M; Sorscher EJ
• EXAMPLES OF CFTR "SUPER-RESPONDERS," AS WELL AS VARIANTS WITH NEGLIGIBLE ACTIVITY AND MINIMAL PHARMACOLOGIC CORRECTION
  Volume: 53 Page(s): 168 - 168
  09/01/2018 Authors: Rab A; Joshi D; Oliver KE; Sorseher EJ; Hong JS
• STRUCTURE OF CFTR N-TERMINAL LASSO HELICES AS A GUIDE FOR EVALUATING MUTANT CFTR CHANNEL FOLDING, MATURATION, AND PHARMACOLOGIC RESCUE
  Volume: 53 Page(s): 152 - 152
  09/01/2018 Authors: Sabusap CP; Joshi D; Rab A; Solomon GM; Simhaev L; Sorscher EJ; Senderowitz H; Hong JS
• THERATYPING RARE AND COMMON CFTR MISSENSE VARIANTS BASED ON RESIDUAL FUNCTION AND PHENOTYPE
  Volume: 53 Page(s): 262 - 263
  09/01/2018 Authors: Raraigh KS; Han ST; Rab A; Matthew P; Evans TA; Davis E; McCague A; Joynt A; Lu Z; Sharma N
• RIBOSOMAL PROTEIN L12 IS A MOLECULAR TARGET SUITABLE FOR RESCUE OF CFTR DEFECTS WITH MULTIPLE DISEASE SUBCATEGORIES
  Volume: 53 Page(s): 148 - 148
  09/01/2018 Authors: Oliver KE; Rauscher R; Ali H; Icyuz M; Rab A; Hong JS; Hartman JL; Ignatova Z; Sorscher EJ
• Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators.
  JCI Insight Volume: 3
  07/26/2018 Authors: Han ST; Rab A; Pellicore MJ; Davis EF; McCague AF; Evans TA; Joynt AT; Lu Z; Cai Z; Raraigh KS
• High-Throughput Yeast Phenomics Identifies Genetic Modifiers that Partially Rescue Human W1282X-CFTR
  Volume: 197
  01/01/2018 Authors: Ali H; Oliver KE; Icyuz M; Kelly S; Rab A; Hong JS; Bedwell DM; Hartman JL; Sorscher EJ
• THERAPY-BASED CLASSIFICATION OF RARE CFTR MUTATIONS
  Volume: 52 Page(s): S330 - S330
  09/01/2017 Authors: Rab A; Manfredi C; Joshi D; Sorscher EJ; Hong JS
• CFTR2 MUTATIONS IN NBD1: CORRELATION BETWEEN IN VITRO THERMAL INSTABILITY AND SEVERITY OF FOLDING DEFECT
  Volume: 52 Page(s): S235 - S235
  09/01/2017 Authors: Yang Z; Zhou Q; Rab A; Hong JS; Khazanov N; Senderowitz H; Sorscher EJ
• MODULATOR RESPONSIVENESS OF CFTR VARIANTS ASSOCIATED WITH REPLACEMENT AT THE SAME POSITION BY DISTINCT AMINO ACIDS: S549N AND S549R
  Volume: 52 Page(s): S234 - S234
  09/01/2017 Authors: Rab A; Al-Salmani M; Hong JS; Cai Z; Fass UW; Sorscher EJ; Sheppard DN
• IDENTIFICATION AND PROFILING OF SMALL MOLECULES DISPLAYING CFTR FUNCTIONAL RESCUE ACTIVITY IN FRT CELLS STABLY TRANSFECTED WITH Y122X, G542X, OR W1282X cDNA
  Volume: 52 Page(s): S328 - S328
  09/01/2017 Authors: Bihler H; Harrington J; Musisi I; Cheng Y; Mercadante D; Hong JS; Rab A; Sorscher EJ; Saltz J; Mahiou J
• THE P67L CFTR BIOGENESIS DEFECT AND ROLE OF N-TERMINAL LASSO HELICES THAT IMPACT CFTR FOLDING AND MATURATION
  Volume: 52 Page(s): S232 - S232
  09/01/2017 Authors: Sabusap CP; Rab A; Oliver KE; Hong J; Sorscher EJ
• MOLECULAR STUDIES AND DRUG DEVELOPMENT FOR THE CFTR W1282X VARIANT
  Volume: 52 Page(s): S327 - S327
  09/01/2017 Authors: Manfredi C; Rab A; Molina SA; Joshi D; Hong J; Ehrhardt A; Sorscher EJ
• COMBINATIONS OF TRAFFICKING/FOLDING CORRECTORS AND READTHROUGH MODULATORS AS THERAPEUTIC APPROACH FOR CFTR PTC MUTATIONS
  PEDIATRIC PULMONOLOGY Volume: 51 Page(s): 208 - 209
  10/01/2016 Authors: Liang F; Wong E; Jordan NJ; Haibo S; Fitzpatrick LM; Hong JS; Rab A; Sorscher EJ; Mense M
• HIGH-THROUGHPUT SCREENING FOR READTHROUGH MODULATORS OF CFTR PTC MUTATIONS Y122X AND W1282X
  PEDIATRIC PULMONOLOGY Volume: 51 Page(s): 293 - 293
  10/01/2016 Authors: Haibo S; Wong E; Jordan NJ; Fitzpatrick LM; Hong JS; Rab A; Sorscher EJ; Liang F; Mense M
• MOLECULAR ANALYSIS OF THE CFTR W1282X MUTATION
  PEDIATRIC PULMONOLOGY Volume: 51 Page(s): 293 - 294
  10/01/2016 Authors: Manfredi C; Rab A; Joshi D; Wang W; Kirk K; Saltz J; Mense M; Mahiou J; Sorscher EJ; Hong JS
• MODEL SYSTEMS FOR INVESTIGATING MOLECULAR PATHOGENESIS OF RARE CFTR ALLELES
  PEDIATRIC PULMONOLOGY Volume: 51 Page(s): 256 - 257
  10/01/2016 Authors: Rab A; Wen H; Manfredi C; Ehrhardt A; Joshi D; Sorscher EJ; Hong JS
• FUNCTIONAL CHARACTERIZATION OF ESTABLISHED AND NOVEL NONSENSE MUTATION READTHROUGH MODULATORS IN ENGINEERED FRT CELL LINES AND PRIMARY HUMAN BRONCHIAL EPITHELIAL (HBE) CELLS
  PEDIATRIC PULMONOLOGY Volume: 51 Page(s): 264 - 264
  10/01/2016 Authors: Bihler H; Harrington J; Musisi I; Mercadante D; Hong JS; Rab A; Sorscher EJ; Saltz J; Mahiou J; Wong E
• Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators.
  PLoS One Volume: 11 Page(s): e0152232
  01/01/2016 Authors: Wang W; Hong JS; Rab A; Sorscher EJ; Kirk KL
• In Vitro Longitudinal Relaxivity Profile of Gd(ABE-DTTA), an Investigational Magnetic Resonance Imaging Contrast Agent.
  PLoS One Volume: 11 Page(s): e0149260
  01/01/2016 Authors: Varga-Szemes A; Kiss P; Rab A; Suranyi P; Lenkey Z; Simor T; Bryant RG; Elgavish GA
• EPITHELIAL CELL MODELS EXPRESSING DIVERSE CFTR MUTATIONS FOR STUDIES OF DISEASE MECHANISM AND DRUG DISCOVERY
  PEDIATRIC PULMONOLOGY Volume: 50 Page(s): 259 - 259
  10/01/2015 Authors: Rab A; Hong JS; Wen H; Sorscher EJ
• DISTINCT PREMATURE TERMINATION CODON MUTATIONS OF CFTR MAY BENEFIT FROM SPECIFIC THERAPEUTIC APPROACHES TO CORRECTION AND READ-THROUGH MODULATION
  PEDIATRIC PULMONOLOGY Volume: 50 Page(s): 202 - 202
  10/01/2015 Authors: Liang F; Jordan NJ; Shang H; Wong E; Schneider C; Hong JS; Rab A; Sorscher EJ; Bihler H; Mense M
• F508 CFTR surface stability is regulated by DAB2 and CHIP-mediated ubiquitination in post-endocytic compartments.
  PLoS One Volume: 10 Page(s): e0123131
  01/01/2015 Authors: Fu L; Rab A; Tang LP; Bebok Z; Rowe SM; Bartoszewski R; Collawn JF
• Chymase mediates injury and mitochondrial damage in cardiomyocytes during acute ischemia/reperfusion in the dog.
  PLoS One Volume: 9 Page(s): e94732
  01/01/2014 Authors: Zheng J; Wei C-C; Hase N; Shi K; Killingsworth CR; Litovsky SH; Powell PC; Kobayashi T; Ferrario CM; Rab A
• The silent codon change I507-ATC->ATT contributes to the severity of the F508 CFTR channel dysfunction.
  FASEB J Volume: 27 Page(s): 4630 - 4645
  11/01/2013 Authors: Lazrak A; Fu L; Bali V; Bartoszewski R; Rab A; Havasi V; Keiles S; Kappes J; Kumar R; Lefkowitz E
• Cigarette smoke and CFTR: implications in the pathogenesis of COPD.
  Am J Physiol Lung Cell Mol Physiol Volume: 305 Page(s): L530 - L541
  10/15/2013 Authors: Rab A; Rowe SM; Raju SV; Bebok Z; Matalon S; Collawn JF
• Dab2 is a key regulator of endocytosis and post-endocytic trafficking of the cystic fibrosis transmembrane conductance regulator.
  Biochem J Volume: 441 Page(s): 633 - 643
  01/15/2012 Authors: Fu L; Rab A; Tang LP; Rowe SM; Bebok Z; Collawn JF
• The unfolded protein response (UPR)-activated transcription factor X-box-binding protein 1 (XBP1) induces microRNA-346 expression that targets the human antigen peptide transporter 1 (TAP1) mRNA and governs immune regulatory genes.
  J Biol Chem Volume: 286 Page(s): 41862 - 41870
  12/02/2011 Authors: Bartoszewski R; Brewer JW; Rab A; Crossman DK; Bartoszewska S; Kapoor N; Fuller C; Collawn JF; Bebok Z
• CFTR expression regulation by the unfolded protein response.
  Volume: 491 Page(s): 3 - 24
  01/01/2011 Authors: Bartoszewski R; Rab A; Fu L; Bartoszewska S; Collawn J; Bebok Z
• ROLE OF ADAPTOR PROTEINS IN CFTR DOWN-REGULATION
  Page(s): 151 - 152
  01/01/2011 Authors: Fu L; Rab A; Bebok Z; Collawn JF
• Impaired Galactose Metabolism by Lithium Treatment Leads to Increased Unfolded Protein Response and Elevated O-Glcnac Modification of Proteins
  Volume: 20 Page(s): 1481 - 1482
  11/01/2010 Authors: Nagy T; Frank D; Poor VS; Rab A; Kovacs GL; Miseta A
• O-GlcNAc modification of proteins affects volume regulation in Jurkat cells.
  Eur Biophys J Volume: 39 Page(s): 1207 - 1217
  07/01/2010 Authors: Nagy T; Balasa A; Frank D; Rab A; Rideg O; Kotek G; Magyarlaki T; Bogner P; Kovcs GL; Miseta A
• Activation of the unfolded protein response by deltaF508 CFTR.
  Am J Respir Cell Mol Biol Volume: 39 Page(s): 448 - 457
  10/01/2008 Authors: Bartoszewski R; Rab A; Jurkuvenaite A; Mazur M; Wakefield J; Collawn JF; Bebok Z
• The mechanism of cystic fibrosis transmembrane conductance regulator transcriptional repression during the unfolded protein response.
  J Biol Chem Volume: 283 Page(s): 12154 - 12165
  05/02/2008 Authors: Bartoszewski R; Rab A; Twitty G; Stevenson L; Fortenberry J; Piotrowski A; Dumanski JP; Bebok Z
• Restoration of W1282X CFTR activity by enhanced expression.
  Am J Respir Cell Mol Biol Volume: 37 Page(s): 347 - 356
  09/01/2007 Authors: Rowe SM; Varga K; Rab A; Bebok Z; Byram K; Li Y; Sorscher EJ; Clancy JP
• VCP/p97 AAA-ATPase does not interact with the endogenous wild-type cystic fibrosis transmembrane conductance regulator.
  Am J Respir Cell Mol Biol Volume: 36 Page(s): 706 - 714
  06/01/2007 Authors: Goldstein RF; Niraj A; Sanderson TP; Wilson LS; Rab A; Kim H; Bebok Z; Collawn JF
• Endoplasmic reticulum stress and the unfolded protein response regulate genomic cystic fibrosis transmembrane conductance regulator expression.
  Am J Physiol Cell Physiol Volume: 292 Page(s): C756 - C766
  02/01/2007 Authors: Rab A; Bartoszewski R; Jurkuvenaite A; Wakefield J; Collawn JF; Bebok Z
• Activation of the unfolded protein response (UPR) by Delta F508 CFTR and transcriptional suppression of CFTR expression by the UPR
  Page(s): 214 - 214
  01/01/2007 Authors: Bartoszewski R; Rab A; Wakefield J; Bebok Z
• Hexose phosphorylation and the putative calcium channel component Mid1p are required for the hexose-induced transient elevation of cytosolic calcium response in Saccharomyces cerevisiae.
  Mol Microbiol Volume: 44 Page(s): 1299 - 1308
  06/01/2002 Authors: Tks-Fzesi M; Bedwell DM; Repa I; Sipos K; Smegi B; Rab A; Miseta A