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Research Output 1994 2019

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2019

A central core disease mutation in the Ca2+-binding site of skeletal muscle ryanodine receptor impairs single-channel regulation

Chirasani, V. R., Xu, L., Addis, H. G., Pasek, D. A., Dokholyan, N., Meissner, G. & Yamaguchi, N., Aug 1 2019, In : American Journal of Physiology - Cell Physiology. 317, 2, p. C358-C365

Research output: Contribution to journalArticle

Central Core Myopathy
Ryanodine Receptor Calcium Release Channel
Skeletal Muscle
Binding Sites
Mutation
1 Citation (Scopus)

Cardiomyopathy Mutations in Metavinculin Disrupt Regulation of Vinculin-Induced F-Actin Assemblies

Sarker, M., Lee, H. T., Mei, L., Krokhotin, A., de los Reyes, S. E., Yen, L., Costantini, L. M., Griffith, J., Dokholyan, N., Alushin, G. M. & Campbell, S. L., Apr 5 2019, In : Journal of Molecular Biology. 431, 8, p. 1604-1618 15 p.

Research output: Contribution to journalArticle

Vinculin
Cardiomyopathies
Actins
Mutation
Dimerization
1 Citation (Scopus)

Conformational ensemble of native α-synuclein in solution as determined by short-distance crosslinking constraint-guided discrete molecular dynamics simulations

Brodie, N. I., Popov, K. I., Petrotchenko, E. V., Dokholyan, N. & Borchers, C. H., Mar 1 2019, In : PLoS computational biology. 15, 3, e1006859.

Research output: Contribution to journalArticle

Open Access
Synucleins
Discrete Dynamics
molecular dynamics
protein structure
Molecular Dynamics Simulation

Engineering proteins for allosteric control by light or ligands

Dagliyan, O., Dokholyan, N. & Hahn, K. M., Jun 1 2019, In : Nature Protocols. 14, 6, p. 1863-1883 21 p.

Research output: Contribution to journalArticle

Protein Engineering
Ligands
Light
Guanine
Sirolimus

Inter-Active Site Communication Mediated by the Dimer Interface β-Sheet in the Half-the-Sites Enzyme, Thymidylate Synthase

Sapienza, P. J., Popov, K. I., Mowrey, D. D., Falk, B. T., Dokholyan, N. & Lee, A. L., Jul 30 2019, In : Biochemistry. 58, 30, p. 3302-3313 12 p.

Research output: Contribution to journalArticle

Thymidylate Synthase
Dimers
Catalytic Domain
Nuclear magnetic resonance
Magnetic Resonance Spectroscopy

Limits in accuracy and a strategy of RNA structure prediction using experimental information

Wang, J., Williams, B., Chirasani, V. R., Krokhotin, A., Das, R. & Dokholyan, N., Jan 1 2019, In : Nucleic acids research. 47, 11, p. 5563-5572 10 p.

Research output: Contribution to journalArticle

Open Access
RNA
Structural Models
Computational Biology

MedusaDock 2.0: Efficient and Accurate Protein-Ligand Docking with Constraints

Wang, J. & Dokholyan, N., Jun 24 2019, In : Journal of Chemical Information and Modeling. 59, 6, p. 2509-2515 7 p.

Research output: Contribution to journalArticle

Ligands
drug
Proteins
efficiency
energy
1 Citation (Scopus)

MERTK mediated novel site Akt phosphorylation alleviates SAV1 suppression

Jiang, Y., Zhang, Y., Leung, J. Y., Fan, C., Popov, K. I., Su, S., Qian, J., Wang, X., Holtzhausen, A., Ubil, E., Xiang, Y., Davis, I., Dokholyan, N., Wu, G., Perou, C. M., Kim, W. Y., Earp, H. S. & Liu, P., Dec 1 2019, In : Nature communications. 10, 1, 1515.

Research output: Contribution to journalArticle

Open Access
phosphorylation
Phosphorylation
Chemical activation
retarding
activation
1 Citation (Scopus)

Molecular mechanisms of heterogeneous oligomerization of huntingtin proteins

Bonfanti, S., Lionetti, M. C., Fumagalli, M. R., Chirasani, V. R., Tiana, G., Dokholyan, N., Zapperi, S. & La Porta, C. A. M., Dec 1 2019, In : Scientific reports. 9, 1, 7615.

Research output: Contribution to journalArticle

Open Access
Huntington Disease
Molecular Conformation
Fluorescence Resonance Energy Transfer
Prions
Optical Imaging

Rationally designed carbohydrate-occluded epitopes elicit HIV-1 Env-specific antibodies

Zhu, C., Dukhovlinova, E., Council, O., Ping, L., Faison, E. M., Prabhu, S. S., Potter, E. L., Upton, S. L., Yin, G., Fay, J. M., Kincer, L. P., Spielvogel, E., Campbell, S. L., Benhabbour, S. R., Ke, H., Swanstrom, R. & Dokholyan, N., Dec 1 2019, In : Nature communications. 10, 1, 948.

Research output: Contribution to journalArticle

human immunodeficiency virus
carbohydrates
antibodies
HIV-1
Epitopes
5 Citations (Scopus)

RNA-DNA fibers and polygons with controlled immunorecognition activate RNAi, FRET and transcriptional regulation of NF-κB in human cells

Ke, W., Hong, E., Saito, R. F., Rangel, M. C., Wang, J., Viard, M., Richardson, M., Khisamutdinov, E. F., Panigaj, M., Dokholyan, N., Chammas, R., Dobrovolskaia, M. A. & Afonin, K. A., Jan 1 2019, In : Nucleic acids research. 47, 3, p. 1350-1361 12 p.

Research output: Contribution to journalArticle

Open Access
RNA Interference
Nucleic Acids
RNA
DNA
Biosensing Techniques

Stabilization of μ-opioid receptor facilitates its cellular translocation and signaling

Zhu, C., Han, Q., Samoshkin, A., Convertino, M., Linton, A., Faison, E. M., Ji, R. R., Diatchenko, L. & Dokholyan, N., Oct 1 2019, In : Proteins: Structure, Function and Bioinformatics. 87, 10, p. 878-884 7 p.

Research output: Contribution to journalArticle

Opioid Receptors
Morphine
Stabilization
Signal transduction
Mutation

Uncovered Dynamic Coupling Resolves the Ambiguous Mechanism of Phenylalanine Hydroxylase Oxygen Binding

Reilley, D. J., Popov, K. I., Dokholyan, N. & Alexandrova, A. N., May 30 2019, In : Journal of Physical Chemistry B. 123, 21, p. 4534-4539 6 p.

Research output: Contribution to journalArticle

Phenylalanine Hydroxylase
phenylalanine
Molecular dynamics
Enzymes
Iron
2 Citations (Scopus)

Viral DNA Binding to NLRC3, an Inhibitory Nucleic Acid Sensor, Unleashes STING, a Cyclic Dinucleotide Receptor that Activates Type I Interferon

Li, X., Deng, M., Petrucelli, A. S., Zhu, C., Mo, J., Zhang, L., Tam, J. W., Ariel, P., Zhao, B., Zhang, S., Ke, H., Li, P., Dokholyan, N., Duncan, J. A. & Ting, J. P. Y., Mar 19 2019, In : Immunity. 50, 3, p. 591-599.e6

Research output: Contribution to journalArticle

Interferon alpha-beta Receptor
Viral DNA
Nucleic Acids
Interferons
Interferon Type I

β-Methylamino-L-alanine substitution of serine in SOD1 suggests a direct role in ALS etiology

Proctor, E. A., Mowrey, D. D. & Dokholyan, N., Jul 1 2019, In : PLoS computational biology. 15, 7, e1007225.

Research output: Contribution to journalArticle

Open Access
etiology
Amyotrophic Lateral Sclerosis
Alanine
alanine
serine
2018
2 Citations (Scopus)

Ca 2 -mediated activation of the skeletal-muscle ryanodine receptor ion channel

Xu, L., Chirasani, V. R., Carter, J. S., Pasek, D. A., Dokholyan, N., Yamaguchi, N. & Meissner, G., Jan 1 2018, In : Journal of Biological Chemistry. 293, 50, p. 19501-19509 9 p.

Research output: Contribution to journalArticle

Ryanodine Receptor Calcium Release Channel
Ion Channels
Muscle
Skeletal Muscle
Chemical activation
4 Citations (Scopus)

Computational design of chemogenetic and optogenetic split proteins

Dagliyan, O., Krokhotin, A., Ozkan-Dagliyan, I., Deiters, A., Der, C. J., Hahn, K. M. & Dokholyan, N., Dec 1 2018, In : Nature communications. 9, 1, 4042.

Research output: Contribution to journalArticle

Optogenetics
proteins
Proteins
assembly
Biological Phenomena
1 Citation (Scopus)

G4941K substitution in the pore-lining S6 helix of the skeletal muscle ryanodine receptor increases RyR1 sensitivity to ytosolic and luminal Ca 2+

Xu, L., Mowrey, D. D., Chirasani, V. R., Wang, Y., Pasek, D. A., Dokholyan, N. & Meissner, G., Jan 1 2018, In : Journal of Biological Chemistry. 293, 6, p. 2015-2028 14 p.

Research output: Contribution to journalArticle

S 6
Ryanodine Receptor Calcium Release Channel
Linings
Muscle
Skeletal Muscle
9 Citations (Scopus)

High-speed atomic force microscopy reveals structural dynamics of α -synuclein monomers and dimers

Zhang, Y., Hashemi, M., Lv, Z., Williams, B., Popov, K. I., Dokholyan, N. & Lyubchenko, Y. L., Mar 28 2018, In : Journal of Chemical Physics. 148, 12, 123322.

Research output: Contribution to journalArticle

Synucleins
dynamic structural analysis
Structural dynamics
Dimers
Atomic force microscopy
8 Citations (Scopus)

Large SOD1 aggregates, unlike trimeric SOD1, do not impact cell viability in a model of amyotrophic lateral sclerosis

Zhu, C., Beck, M. V., Griffith, J. D., Deshmukh, M. & Dokholyan, N., May 1 2018, In : Proceedings of the National Academy of Sciences of the United States of America. 115, 18, p. 4661-4665 5 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Cell Survival
Motor Neurons
Poisons
Neurodegenerative Diseases

Ligand binding to a remote site thermodynamically corrects the F508del mutation in the human cystic fibrosis transmembrane conductance regulator

Wang, C., Aleksandrov, A. A., Yang, Z., Forouhar, F., Proctor, E. A., Kota, P., An, J., Kaplan, A., Khazanov, N., Boël, G., Stockwell, B. R., Senderowitz, H., Dokholyan, N., Riordan, J. R., Brouillette, C. G. & Hunt, J. F., Jan 1 2018, In : Journal of Biological Chemistry. 293, 46, p. 17685-17704 20 p.

Research output: Contribution to journalArticle

Cystic Fibrosis Transmembrane Conductance Regulator
Ligands
Mutation
Nucleotides
Molecules

Mapping allosteric linkage to channel gating by extracellular domains in the human epithelial sodium channel

Shobair, M., Popov, K. I., Dang, Y. L., He, H., Jackson Stutts, M. & Dokholyan, N., Jan 1 2018, In : Journal of Biological Chemistry. 293, 10, p. 3675-3684 10 p.

Research output: Contribution to journalArticle

Epithelial Sodium Channels
Chromosome Mapping
Degenerin Sodium Channels
Cysteine
Chemical activation
6 Citations (Scopus)

miRNA-711 Binds and Activates TRPA1 Extracellularly to Evoke Acute and Chronic Pruritus

Han, Q., Liu, D., Convertino, M., Wang, Z., Jiang, C., Kim, Y. H., Luo, X., Zhang, X., Nackley, A., Dokholyan, N. & Ji, R. R., Aug 8 2018, In : Neuron. 99, 3, p. 449-463.e6

Research output: Contribution to journalArticle

Pruritus
MicroRNAs
Lymphoma
Ion Channels
S 6
5 Citations (Scopus)

P62-dependent phase separation of patient-derived KEap1 mutations and Nrf2

Cloer, E. W., Siesser, P. F., Cousins, E. M., Goldfarb, D., Mowrey, D. D., Harrison, J. S., Weir, S. J., Dokholyan, N. & Major, M. B., Nov 1 2018, In : Molecular and cellular biology. 38, 22, e0064417.

Research output: Contribution to journalArticle

Mutation
Polyubiquitin
Phenotype
Autophagy
Molecular Dynamics Simulation
5 Citations (Scopus)

Reversible and Tunable Photoswitching of Protein Function through Genetic Encoding of Azobenzene Amino Acids in Mammalian Cells

Luo, J., Samanta, S., Convertino, M., Dokholyan, N. & Deiters, A., Oct 18 2018, In : ChemBioChem. 19, 20, p. 2178-2185 8 p.

Research output: Contribution to journalArticle

Cells
Amino Acids
Firefly Luciferases
Biological Phenomena
Light
9 Citations (Scopus)

RNA Fibers as Optimized Nanoscaffolds for siRNA Coordination and Reduced Immunological Recognition

Rackley, L., Stewart, J. M., Salotti, J., Krokhotin, A., Shah, A., Halman, J. R., Juneja, R., Smollett, J., Lee, L., Roark, K., Viard, M., Tarannum, M., Vivero-Escoto, J., Johnson, P. F., Dobrovolskaia, M. A., Dokholyan, N., Franco, E. & Afonin, K. A., Nov 28 2018, In : Advanced Functional Materials. 28, 48, 1805959.

Research output: Contribution to journalArticle

RNA
Small Interfering RNA
fibers
Fibers
engineers
5 Citations (Scopus)

Structural complexity and functional diversity of plant NADPH oxidases

Kaur, G., Guruprasad, K., Temple, B. R. S., Shirvanyants, D. G., Dokholyan, N. & Pati, P. K., Jan 1 2018, In : Amino Acids. 50, 1, p. 79-94 16 p.

Research output: Contribution to journalArticle

NADPH Oxidase
Plant Structures
Gene Duplication
Protein Folding
Molecular Dynamics Simulation
4 Citations (Scopus)

Tyrosine phosphorylation switching of a G protein

Li, B., Tunc-Ozdemir, M., Urano, D., Jia, H., Werth, E. G., Mowrey, D. D., Hicks, L. M., Dokholyan, N., Torres, M. P. & Jones, A. M., Jan 1 2018, In : Journal of Biological Chemistry. 293, 13, p. 4752-4766 15 p.

Research output: Contribution to journalArticle

Phosphorylation
GTP Phosphohydrolases
GTP-Binding Proteins
Tyrosine
GTP-Binding Protein Regulators
2017
6 Citations (Scopus)

A Structural Model for Vinculin Insertion into PIP2-Containing Membranes and the Effect of Insertion on Vinculin Activation and Localization

Thompson, P. M., Ramachandran, S., Case, L. B., Tolbert, C. E., Tandon, A., Pershad, M., Dokholyan, N., Waterman, C. M. & Campbell, S. L., Feb 7 2017, In : Structure. 25, 2, p. 264-275 12 p.

Research output: Contribution to journalArticle

Vinculin
Structural Models
Focal Adhesions
Membranes
Lipid Bilayers
10 Citations (Scopus)

Comparative Visualization of the RNA Suboptimal Conformational Ensemble In Vivo

Woods, C. T., Lackey, L., Williams, B., Dokholyan, N., Gotz, D. & Laederach, A., Jul 25 2017, In : Biophysical journal. 113, 2, p. 290-301 12 p.

Research output: Contribution to journalArticle

RNA
Acylation
Hydroxyl Radical
Adenine
Riboswitch
9 Citations (Scopus)

Direct identification of base-paired RNA nucleotides by correlated chemical probing

Krokhotin, A., Mustoe, A. M., Weeks, K. M. & Dokholyan, N., Jan 1 2017, In : RNA. 23, 1, p. 6-13 8 p.

Research output: Contribution to journalArticle

Nucleotides
RNA
Base Pairing
8 Citations (Scopus)

Engineering Pak1 Allosteric Switches

Dagliyan, O., Karginov, A. V., Yagishita, S., Gale, M. E., Wang, H., Dermardirossian, C., Wells, C. M., Dokholyan, N., Kasai, H. & Hahn, K. M., Jul 21 2017, In : ACS Synthetic Biology. 6, 7, p. 1257-1262 6 p.

Research output: Contribution to journalArticle

p21-Activated Kinases
Sirolimus
Chemical activation
Switches
Graph theory
4 Citations (Scopus)

Ion-pulling simulations provide insights into the mechanisms of channel opening of the skeletal muscle ryanodine receptor

Mowrey, D. D., Xu, L., Mei, Y., Pasek, D. A., Meissner, G. & Dokholyan, N., Jan 1 2017, In : Journal of Biological Chemistry. 292, 31, p. 12947-12958 12 p.

Research output: Contribution to journalArticle

Ryanodine Receptor Calcium Release Channel
Muscle
Skeletal Muscle
Ions
Constriction
1 Citation (Scopus)

Molecular Mechanisms of the R61T Mutation in Apolipoprotein E4: A Dynamic Rescue

Williams, B., Convertino, M., Das, J. & Dokholyan, N., Nov 21 2017, In : Biophysical journal. 113, 10, p. 2192-2198 7 p.

Research output: Contribution to journalArticle

Apolipoprotein E4
Mutation
Alzheimer Disease
Apolipoprotein E2
Apolipoprotein E3
25 Citations (Scopus)

Physicochemical code for quinary protein interactions in Escherichia coli

Mu, X., Choi, S., Lang, L., Mowray, D., Dokholyan, N., Danielsson, J. & Oliveberg, M., Jun 6 2017, In : Proceedings of the National Academy of Sciences of the United States of America. 114, 23, p. E4556-E4563

Research output: Contribution to journalArticle

Escherichia coli
Proteins
Bacterial Proteins
Escherichia coli Proteins
Hydrophobic and Hydrophilic Interactions
15 Citations (Scopus)

Predicting the functional consequences of non-synonymous single nucleotide polymorphisms in IL8 gene

Dakal, T. C., Kala, D., Dhiman, G., Yadav, V., Krokhotin, A. & Dokholyan, N., Dec 1 2017, In : Scientific reports. 7, 1, 6525.

Research output: Contribution to journalArticle

Interleukin-8
Single Nucleotide Polymorphism
Genes
Sequence Homology
Amino Acid Substitution
2 Citations (Scopus)

Redundant Functions for Nap1 and Chz1 in H2A.Z Deposition

Dronamraju, R., Ramachandran, S., Jha, D. K., Adams, A. T., Difiore, J. V., Parra, M. A., Dokholyan, N. & Strahl, B. D., Dec 1 2017, In : Scientific reports. 7, 1, 10791.

Research output: Contribution to journalArticle

Heterochromatin
Histone Chaperones
Molecular Dynamics Simulation
Histones
DNA Damage
48 Citations (Scopus)

RNA-Puzzles Round III: 3D RNA structure prediction of five riboswitches and one ribozyme

Miao, Z., Adamiak, R. W., Antczak, M., Batey, R. T., Becka, A. J., Biesiada, M., Boniecki, M. J., Bujnicki, J. M., Chen, S. J., Cheng, C. Y., Chou, F. C., Ferré-D'Amaré, A. R., Das, R., Dawson, W. K., Ding, F., Dokholyan, N., Dunin-Horkawicz, S., Geniesse, C., Kappel, K., Kladwang, W. & 26 others, Krokhotin, A., Łach, G. E., Major, F., Mann, T. H., Magnus, M., Pachulska-Wieczorek, K., Patel, D. J., Piccirilli, J. A., Popenda, M., Purzycka, K. J., Ren, A., Rice, G. M., Santalucia, J., Sarzynska, J., Szachniuk, M., Tandon, A., Trausch, J. J., Tian, S., Wang, J., Weeks, K. M., Williams, B., Xiao, Y., Xu, X., Zhang, D., Zok, T. & Westhof, E., May 1 2017, In : RNA. 23, 5, p. 655-672 18 p.

Research output: Contribution to journalArticle

Riboswitch
Catalytic RNA
RNA
Ligands
Glutamine
17 Citations (Scopus)

Solving protein structures using short-distance cross-linking constraints as a guide for discrete molecular dynamics simulations

Brodie, N. I., Popov, K. I., Petrotchenko, E. V., Dokholyan, N. & Borchers, C. H., Jul 5 2017, In : Science Advances. 3, 7, e1700479.

Research output: Contribution to journalArticle

Molecular Dynamics Simulation
Tacrolimus Binding Proteins
Proteins
Myoglobin
Deuterium
4 Citations (Scopus)

Structure modeling of RNA using sparse NMR constraints

Williams, B., Zhao, B., Tandon, A., Ding, F., Weeks, K. M., Zhang, Q. & Dokholyan, N., Dec 15 2017, In : Nucleic acids research. 45, 22, p. 12638-12647 10 p.

Research output: Contribution to journalArticle

RNA
Molecular Dynamics Simulation
Molecular Conformation
Benchmarking
Nucleotide Motifs
7 Citations (Scopus)

Transmembrane helical interactions in the CFTR channel pore

Das, J., Aleksandrov, A. A., Cui, L., He, L., Riordan, J. R. & Dokholyan, N., Jun 1 2017, In : PLoS computational biology. 13, 6, e1005594.

Research output: Contribution to journalArticle

Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator
cystic fibrosis
Conductance
Regulator
2016
1 Citation (Scopus)

A hidden aggregation-prone structure in the heart of hypoxia inducible factor prolyl hydroxylase

Hadi-Alijanvand, H., Proctor, E. A., Ding, F., Dokholyan, N. & Moosavi-Movahedi, A. A., May 1 2016, In : Proteins: Structure, Function and Bioinformatics. 84, 5, p. 611-623 13 p.

Research output: Contribution to journalArticle

Prolyl Hydroxylases
Agglomeration
Protein Unfolding
Proteins
Molecular Dynamics Simulation
11 Citations (Scopus)

A modified PATH algorithm rapidly generates transition states comparable to those found by other well established algorithms

Chandrasekaran, S. N., Das, J., Dokholyan, N. & Carter, C. W., Jan 1 2016, In : Structural Dynamics. 3, 1, 012101.

Research output: Contribution to journalArticle

Proteins
Structural properties
Crystal structure
Trajectories
proteins
11 Citations (Scopus)

A Phosphomimetic Mutation Stabilizes SOD1 and Rescues Cell Viability in the Context of an ALS-Associated Mutation

Fay, J. M., Zhu, C., Proctor, E. A., Tao, Y., Cui, W., Ke, H. & Dokholyan, N., Nov 1 2016, In : Structure. 24, 11, p. 1898-1906 9 p.

Research output: Contribution to journalArticle

Amyotrophic Lateral Sclerosis
Cell Survival
Mutation
Poisons
Motor Neurons
9 Citations (Scopus)
Factor Xa
activity structure
Classifiers
Ligands
ranking
55 Citations (Scopus)

Engineering extrinsic disorder to control protein activity in living cells

Dagliyan, O., Tarnawski, M., Chu, P. H., Shirvanyants, D., Schlichting, I., Dokholyan, N. & Hahn, K. M., Dec 16 2016, In : Science. 354, 6318, p. 1441-1444 4 p.

Research output: Contribution to journalArticle

Molecular Motor Proteins
Optogenetics
Proteins
Guanosine
Guanine
5 Citations (Scopus)

Gain-of-function mutation W493R in the epithelial sodium channel allosterically reconfigures intersubunit coupling

Shobair, M., Dagliyan, O., Kota, P., Dang, Y. L., He, H., Stutts, M. J. & Dokholyan, N., Feb 19 2016, In : Journal of Biological Chemistry. 291, 8, p. 3682-3692 11 p.

Research output: Contribution to journalArticle

Epithelial Sodium Channels
Molecular Dynamics Simulation
Water-Electrolyte Imbalance
Liddle Syndrome
Sodium
1 Citation (Scopus)

Harnessing Nature's Diversity: Discovering organophosphate bioscavenger characteristics among low molecular weight proteins

Jacob, R. B., Michaels, K. C., Anderson, C. J., Fay, J. M. & Dokholyan, N., Nov 15 2016, In : Scientific reports. 6, 37175.

Research output: Contribution to journalArticle

Organophosphates
Organophosphate Poisoning
Molecular Weight
Acetylcholinesterase
Paraoxon
39 Citations (Scopus)

Nonnative SOD1 trimer is toxic to motor neurons in a model of amyotrophic lateral sclerosis

Proctor, E. A., Fee, L., Tao, Y., Redler, R. L., Fay, J. M., Zhang, Y., Lv, Z., Mercer, I. P., Deshmukh, M., Lyubchenko, Y. L. & Dokholyan, N., Jan 19 2016, In : Proceedings of the National Academy of Sciences of the United States of America. 113, 3, p. 614-619 6 p.

Research output: Contribution to journalArticle

Poisons
Amyotrophic Lateral Sclerosis
Motor Neurons
Mutation
Disease Progression
12 Citations (Scopus)

Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770

Gentzsch, M., Ren, H. Y., Houck, S. A., Quinney, N. L., Cholon, D. M., Sopha, P., Chaudhry, I. G., Das, J., Dokholyan, N., Randell, S. H. & Cyr, D. M., Jan 1 2016, In : American Journal of Physiology - Lung Cellular and Molecular Physiology. 311, 3, p. L550-L559

Research output: Contribution to journalArticle

Cystic Fibrosis
Mutation
Therapeutics
ivacaftor
lumacaftor