Rula Zain

About me
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CV
Public outreach and news

Research

Research area/interest: Chemical Biology of nucleic acids, non-B-DNA
structures in gene regulation, structure-specific nucleic acid binding of
small molecules

Articles

Article: BLOOD ADVANCES. 2024;8(8):1981-1990
In BTK, phosphorylated Y223 in the SH3 domain mirrors catalytic activity, but does not in fl uence biological function
Estupinan HY; Bouderlique T; He C; Berglof A; Cappelleri A; Frengen N; Zain R; Karlsson MCI; Mansson R; Smith CIE
Article: NUCLEIC ACIDS RESEARCH (NAR). 2023;51(16):8532-8549
Large-scale expansions of Friedreich's ataxia GAA•TTC repeats in an experimental human system: role of DNA replication and prevention by LNA-DNA oligonucleotides and PNA oligomers
Rastokina A; Cebrian J; Mozafari N; Mandel NH; Smith CIE; Lopes M; Zain R; Mirkin SM
Article: HUMAN MUTATION. 2023;2023:1-12
BTKbase, Bruton Tyrosine Kinase Variant Database in X-Linked Agammaglobulinemia: Looking Back and Ahead
Schaafsma GCP; Vaeliaho J; Wang Q; Bergloef A; Zain R; Smith CIE; Vihinen M
Journal article: OPEN ACCESS GOVERNMENT. 2023;39(1):22-23
Classification of rare diseases: The case of the ultra-and hyper-rare
Smith E; Zain R; Hagey DW
Article: FRONTIERS IN BIOSCIENCE-LANDMARK. 2023;28(6):124
Original Exposure to Therapeutic BTK Inhibitors Induces Phenocopying of Btk29A Mutants in the Fruit Fly Drosophila melanogaster
Hamada-Kawaguchi N; Nore BF; Zain R; Engstroem Y; Smith CIE; Yamamoto D
Article: BLOOD ADVANCES. 2023;7(9):1929-1943
Pirtobrutinib targets BTK C481S in ibrutinib-resistant CLL but second-site BTK mutations lead to resistance
Naeem A; Utro F; Wang Q; Cha J; Vihinen M; Martindale S; Zhou Y; Ren Y; Tyekucheva S; Kim AS; Fernandes SM; Saksena G; Rhrissorrakrai K; Levovitz C; Danysh BP; Slowik K; Jacobs RA; Davids MS; Lederer JA; Zain R; Smith CIE; Leshchiner I; Parida L; Getz G; Brown JR
Article: NUCLEIC ACID THERAPEUTICS. 2023;33(2):117-131
Chemical Modifications and Design Influence the Potency of Huntingtin Antigene Oligonucleotides
Saher O; Zaghloul EMM; Umek T; Hagey DWW; Mozafari N; Danielsen MBB; Gouda ASS; Lundin KEE; Jorgensen PTT; Wengel J; Smith CIE; Zain R
Article: NUCLEIC ACID THERAPEUTICS. 2022;32(3):221-233
2′-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity
Honcharenko D; Rocha CSJ; Lundin KE; Maity J; Milton S; Tedebark U; Murtola M; Honcharenko M; Slaitas A; Smith CIE; Zain R; Stromberg R
Article: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. 2022;23(6):3103
Structural Insights into Human Adenovirus Type 4 Virus-Associated RNA I
Bergquist H; Inturi R; Zain R; Punga T
Article: ADVANCED HEALTHCARE MATERIALS. 2022;11(5):e2101658
Growth Media Conditions Influence the Secretion Route and Release Levels of Engineered Extracellular Vesicles
Bost JP; Saher O; Hagey D; Mamand DR; Liang X; Zheng W; Corso G; Gustafsson O; Gorgens A; Smith CIE; Zain R; El Andaloussi S; Gupta D
Article: LEUKEMIA. 2022;36(2):476-481
Covid-19 in patients with chronic lymphocytic leukemia: clinical outcome and B- and T-cell immunity during 13 months in consecutive patients
Blixt L; Bogdanovic G; Buggert M; Gao Y; Hober S; Healy K; Johansson H; Kjellander C; Mravinacova S; Muschiol S; Nilsson P; Palma M; Pin E; Smith CIE; Stromberg O; Chen MS; Zain R; Hansson L; Osterborg A
Article: NUCLEIC ACID THERAPEUTICS. 2021;31(6):443-456
Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System
Umek T; Olsson T; Gissberg O; Saher O; Zaghloul EM; Lundin KE; Wengel J; Hanse E; Zetterberg H; Vizlin-Hodzic D; Smith CIE; Zain R
Article: LEUKEMIA. 2021;35(5):1317-1329
BTK gatekeeper residue variation combined with cysteine 481 substitution causes super-resistance to irreversible inhibitors acalabrutinib, ibrutinib and zanubrutinib
Estupinan HY; Wang Q; Berglof A; Schaafsma GCP; Shi Y; Zhou L; Mohammad DK; Yu L; Vihinen M; Zain R; Smith CIE
Article: HEMASPHERE. 2021;5(5):e564
Ibrutinib Has Time-dependent On- and Off-target Effects on Plasma Biomarkers and Immune Cells in Chronic Lymphocytic Leukemia
Mulder TA; Pena-Perez L; Berglof A; Meinke S; Estupinan HY; Heimersson K; Zain R; Mansson R; Smith CIE; Palma M
Article: PHARMACEUTICS. 2021;13(1):116
Lipophilic Peptide Dendrimers for Delivery of Splice-Switching Oligonucleotides
Daralnakhla H; Saher O; Zamolo S; Bazaz S; P. Bost J; Heitz M; Lundin KE; El Andaloussi S; Darbre T; Reymond J-L; Zain R; Smith CIE
Article: BLOOD ADVANCES. 2020;4(11):2439-2450
Novel mouse model resistant to irreversible BTK inhibitors: a tool identifying new therapeutic targets and side effects
Estupinan HY; Bouderlique T; He C; Berglof A; Gupta D; Saher O; Daza Cruz MA; Pena-Perez L; Yu L; Zain R; Karlsson MCI; Mansson R; Smith CIE
Article: PHARMACEUTICS. 2019;11(12):E666-666
Sugar and Polymer Excipients Enhance Uptake and Splice-Switching Activity of Peptide-Dendrimer/Lipid/Oligonucleotide Formulations
Saher O; Lehto T; Gissberg O; Gupta D; Gustafsson O; EL Andaloussi S; Darbre T; Lundin KE; Smith CIE; Zain R
Article: MOLECULES. 2019;24(6):E1180-1180
Oligonucleotide-Palladacycle Conjugates as Splice-Correcting Agents
Hande M; Saher O; Lundin KE; Smith CIE; Zain R; Lonnberg T
Article: MOLECULES. 2019;24(5):E1000-1000
Oligonucleotide Binding to Non-B-DNA in MYC
Umek T; Sollander K; Bergquist H; Wengel J; Lundin KE; Smith CIE; Zain R
Article: METHODS IN MOLECULAR BIOLOGY. 2019;2036:3-16
Chemical Development of Therapeutic Oligonucleotides.
Lundin KE; Gissberg O; Smith CIE; Zain R
Article: PLOS ONE. 2019;14(2):e0211651
The ability of locked nucleic acid oligonucleotides to pre-structure the double helix: A molecular simulation and binding study
Xu Y; Gissberg O; Pabon-Martinez YV; Wengel J; Lundin KE; Smith CIE; Zain R; Nilsson L; Villa A
Article: EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS. 2018;132:29-40
Novel peptide-dendrimer/lipid/oligonucleotide ternary complexes for efficient cellular uptake and improved splice-switching activity
Saher O; Rocha CSJ; Zaghioul EM; Wiklander OPB; Zamolo S; Heitz M; Ezzat K; Gupta D; Reymond J-L; Zain R; Hollfelder F; Darbre T; Lundin KE; El Andaloussi S; Smith CIE
Article: BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS. 2018;504(4):749-752
Translocation-generated ITK-FER and ITK-SYK fusions induce STAT3 phosphorylation and CD69 expression
Fathi NN; Mohammad DK; Gorgens A; El Andaloussi S; Zain R; Nore BF; Smith CIE
Article: SCIENTIFIC REPORTS. 2017;7(1):11043
LNA effects on DNA binding and conformation: from single strand to duplex and triplex structures
Pabon-Martinez YV; Xu Y; Villa A; Lundin KE; Geny S; Nguyen C-H; Pedersen EB; Jorgensen PT; Wengel J; Nilsson L; Smith CIE; Zain R
Article: ACS OMEGA. 2017;2(5):2165-2177
Role of Pseudoisocytidine Tautomerization in Triplex-Forming Oligonucleotides: In Silico and in Vitro Studies
Hartono YD; Pabon-Martinez YV; Uyar A; Wengel J; Lundin KE; Zain R; Smith CIE; Nilsson L; Villa A
Article: NUCLEIC ACIDS RESEARCH (NAR). 2017;45(9):5153-5169
CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression
Zaghloul EM; Gissberg O; Moreno PMD; Siggens L; Hallbrink M; Jorgensen AS; Ekwall K; Zain R; Wengel J; Lundin KE; Smith CIE
Article: NUCLEIC ACID THERAPEUTICS. 2016;26(6):381-391
Four Novel Splice-Switch Reporter Cell Lines: Distinct Impact of Oligonucleotide Chemistry and Delivery Vector on Biological Activity
Rocha CSJ; Lundin KE; Behlke MA; Zain R; EL Andaloussi S; Smith CIE
Article: ORGANIC AND BIOMOLECULAR CHEMISTRY. 2016;14(23):5210-5213
Clamping of RNA with PNA enables targeting of microRNA
Ghidini A; Bergquist H; Murtola M; Punga T; Zain R; Stromberg R
Article: NUCLEIC ACID THERAPEUTICS. 2016;26(3):183-189
Delivery, Effect on Cell Viability, and Plasticity of Modified Aptamer Constructs
Gissberg O; Zaghloul EM; Lundin KE; Chi-Hung N; Landras-Guetta C; Wengel J; Zain R; Smith CIE
Article: NUCLEIC ACIDS RESEARCH (NAR). 2016;44(5):2007-2019
Next-generation bis-locked nucleic acids with stacking linker and 2′-glycylamino-LNA show enhanced DNA invasion into supercoiled duplexes
Geny S; Moreno PMD; Krzywkowski T; Gissberg O; Andersen NK; Isse AJ; El-Madani AM; Lou C; Pabon YV; Anderson BA; Zaghloul EM; Zain R; Hrdlicka PJ; Jorgensen PT; Nilsson M; Lundin KE; Pedersen EB; Wengel J; Smith CIE
Article: PLOS ONE. 2016;11(11):e0165788
Disruption of Higher Order DNA Structures in Friedreich's Ataxia (GAA)_n Repeats by PNA or LNA Targeting
Bergquist H; Rocha CSJ; Alvarez-Asencio R; Chi-Hung N; Rutland MW; Smith CIE; Good L; Nielsen PE; Zain R
Article: JOURNAL OF BIOLOGICAL CHEMISTRY. 2015;290(8):5174-5189
A Distinct Triplex DNA Unwinding Activity of ChlR1 Helicase
Guo M; Hundseth K; Ding H; Vidhyasagar V; Inoue A; Chi-Hung N; Zain R; Lee JS; Wu Y
Article: NUCLEIC ACIDS RESEARCH (NAR). 2013;41(5):3257-3273
Development of bis-locked nucleic acid (bisLNA) oligonucleotides for efficient invasion of supercoiled duplex DNA
Moreno PMD; Geny S; Pabon YV; Bergquist H; Zaghloul EM; Rocha CSJ; Oprea II; Bestas B; El Andaloussi S; Jorgensen PT; Pedersen EB; Lundin KE; Zain R; Wengel J; Smith CIE
Article: CHEMBIOCHEM. 2009;10(16):2629-2637
Structure-Specific Recognition of Friedreich's Ataxia (GAA)_n Repeats by Benzoquinoquinoxaline Derivatives
Bergquist H; Nikravesh A; Fernandez RD; Larsson V; Nguyen C-H; Good L; Zain R
Article: MOLECULAR THERAPY. 2007;15(8):1537-1542
Antisense PNA accumulates in Escherichia coli and mediates a long post-antibiotic effect
Nikravesh A; Dryselius R; Faridani OR; Goh S; Sadeghizadeh M; Behmanesh M; Ganyu A; Klok EJ; Zain R; Good L
Article: JOURNAL OF MOLECULAR BIOLOGY. 2005;351(4):776-783
Benzoquinoquinoxaline derivatives stabilize and cleave H-DNA and repress transcription downstream of a triplex-forming sequence
Amiri H; Nekhotiaeva N; Sun JS; Nguyen CH; Grierson DS; Good L; Zain R
Article: CHEMBIOCHEM. 2004;5(11):1550-1557
Triple-helix directed cleavage of double-stranded DNA by benzoquinoquinoxaline-1,10-phenanthroline conjugates
Zaid A; Sun JS; Nguyen CH; Bisagni E; Garestier T; Grierson DS; Zain R
Article: CHEMBIOCHEM. 2003;4(9):856-862
Optimization of triple-helix-directed DNA cleavage by benzoquinoquinoxaline-ethylenediaminetetraacetic acid conjugates
Zain R; Polverari D; Nguyen CH; Blouquit Y; Bisagni E; Garestier T; Grierson DS; Sun JS
Article: CHEMISTRY AND BIOLOGY. 1999;6(11):771-777
Design of a triple-helix-specific cleaving reagent
Zain R; Marchand C; Sun JS; Nguyen CH; Bisagni E; Garestier T; Hélène C
Journal article: CHEMINFORM. 1997;28(48):no
ChemInform Abstract: Studies on Nucleoside Phosphonates and Their Derivatives. A Progress Report
BOLLMARK M; KERS A; KERS I; SZABO T; ZAIN R; STAWINSKI J; CIESLAK J; JANKOWSKA J; KRASZEWSKI A
Article: NUCLEOSIDES, NUCLEOTIDES AND NUCLEIC ACIDS. 1997;16(5-6):679-684
Studies on nucleoside phosphonates and their derivatives. A progress report
Bollmark M; Kers A; Kers I; Szabo T; Zain R; Stawinski J
Journal article: CHEMINFORM. 1997;28(5):no
ChemInform Abstract: Nucleoside H‐Phosphonates. Part 17. Synthetic and 31P NMR Studies on the Preparation of Dinucleoside H‐Phosphonothioates.
ZAIN R; STAWINSKI J
Article: JOURNAL OF ORGANIC CHEMISTRY. 1996;61(19):6617-6622
Nucleoside H-phosphonates .17. Synthetic and P-31 NMR studies on the preparation of dinucleoside H-phosphonothioates
Zain R; Stawinski J
Journal article: CHEMINFORM. 1996;27(25):no
ChemInform Abstract: Nucleoside Phosphonates. Development of Synthetic Methods and Reagents
KERS A; KERS I; KRASZEWSKI A; SOBKOWSKI M; SZABO T; THELIN M; ZAIN R; STAWINSKI J
Article: TETRAHEDRON LETTERS. 1996;37(20):3537-3540
A new entry to nucleoside phosphorofluoridate and nucleoside phosphorofluoridothioate diesters
Bollmark M; Zain R; Stawinski J
Article: JOURNAL OF THE CHEMICAL SOCIETY, PERKIN TRANSACTIONS 2. 1996;(5):795-799
Nucleoside H-phosphonates .16. P-31 NMR studies on the transformation of nucleoside H-phosphonate monoesters into a monofunctional tervalent intermediate, nucleoside acyl silyl phosphite
Zain R; Stawinski J
Article: NUCLEOSIDES, NUCLEOTIDES AND NUCLEIC ACIDS. 1996;15(1-3):361-378
Nucleoside phosphonates. Development of synthetic methods and reagents
Kers A; Kers I; Kraszewski A; Sobkowski M; Szabo T; Thelin M; Zain R; Stawinski J
Article: JOURNAL OF ORGANIC CHEMISTRY. 1995;60(25):8241-8244
Nucleoside H-phosphonates .15. Preparation of nucleoside H-phosphonothioate monoesters from the corresponding nucleoside H-phosphonates
Zain R; Stromberg R; Stawinski J
Journal article: COLLECTION OF CZECHOSLOVAK CHEMICAL COMMUNICATIONS. 1993;58(s1):79-81
Hydrolytic stability of nucleoside H-phosphonate and H-phosphonothioate diesters
Stawinski J; Strömberg R; Zain R
Journal article: CHEMINFORM. 1992;23(47):no
ChemInform Abstract: Stereospecific Oxidation and Oxidative Coupling of H‐Phosphonate and H‐ Phosphonothioate Diesters.
STAWINSKI J; STROEMBERG R; ZAIN R
Journal article: TETRAHEDRON LETTERS. 1992;33(22):3185-3188
Stereospecific oxidation and oxidative coupling of H-phosphonate and H-phosphonothioate diesters
Stawiński J; Strömberg R; Zain R
Journal article: NUCLEIC ACIDS SYMPOSIUM SERIES (2004). 1991;(24):95-98
Synthesis and some reactions of nucleoside H-phosphonothioate esters.
Stawinski J; Strömberg R; Szabo T; Thelin M; Westman E; Zain R
Journal article: NUCLEOSIDES, NUCLEOTIDES AND NUCLEIC ACIDS. 1991;10(1-3):515-516
Studies on the Conversion of Nucleoside H-Phosphonate Monoesters into the Corresponding H-Phosphonothioates
Stawinski J; Stromberg R; Zain R
Journal article: NUCLEIC ACIDS SYMPOSIUM SERIES (2004). 1991;(24):230
Some chemical and stereochemical aspects of oxidation of nucleoside H-phosphonothioate diesters.
Stawinski J; Strömberg R; Zain R
Journal article: CHEMINFORM. 1990;21(43):no
ChemInform Abstract: Nucleoside H‐Phosphonates. Part 12. Synthesis of Nucleoside 3′‐(Hydrogen Phosphonothioate) Monoesters via Phosphinate Intermediates.
STAWINSKI J; THELIN M; WESTMAN E; ZAIN R
Journal article: JOURNAL OF ORGANIC CHEMISTRY. 1990;55(11):3503-3506
Nucleoside H-phosphonates. 12. Synthesis of nucleoside 3'-(hydrogen phosphonothioate) monoesters via phosphinate intermediates
Stawinski J; Thelin M; Westman E; Zain R
Journal article: COLLECTION OF CZECHOSLOVAK CHEMICAL COMMUNICATIONS. 1990;55(s1):141-144
Studies on the preparation of nucleoside H-phosphonothioates
Stawinski J; Szabó T; Thelin M; Westman E; Zain R
Journal article: NUCLEIC ACIDS SYMPOSIUM SERIES (2004). 1989;(21):47-48
Recent studies on nucleoside H-phosphonothioate and nucleoside methylphosphonothioate synthesis.
Stawiński J; Szabó T; Thelin M; Westman E; Zain R
Journal article: TETRAHEDRON LETTERS. 1989;30(16):2157-2160
Nucleoside H-phosphonates. X. Studies on nucleoside hydrogenphosphonothioate diester synthesis
Stawiński J; Thelin M; Zain R
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All other publications

Letter: EUROPEAN JOURNAL OF HAEMATOLOGY. 2024;113(1):130-131
Reduced clone size upon BTK inhibitor resistance mutations relates to toxicity caused by inherited PLCG2 gain-of-function variations
Smith CIE; Zain R
Conference publication: BLOOD. 2022;140:6981-6982
Resistance to the Non-Covalent BTK Inhibitor Pirtobrutinib
Naeem AS; Utro F; Wang Q; Cha J; Vihinen M; Martindale SP; Zhou Y; Tyekucheva S; Kim AS; Fernandes SM; Fardoun R; Saksena G; Rhrissorrakrai K; Levovitz C; Danysh BP; Slowik K; Jacobs R; Davids MS; Zain R; Smith ECI; Leshchiner I; Parida L; Getz G; Brown JR
Review: SCANDINAVIAN JOURNAL OF IMMUNOLOGY. 2022;95(4):e13153
Do reduced numbers of plasmacytoid dendritic cells contribute to the aggressive clinical course of COVID-19 in chronic lymphocytic leukaemia?
Smith CIE; Zain R; osterborg A; Palma M; Buggert M; Bergman P; Bryceson Y
Editorial comment: FRONTIERS IN IMMUNOLOGY. 2021;12:804735
New Insights on Bruton's Tyrosine Kinase Inhibitors
Smith CIE; Brown JR; Zain R
Review: FRONTIERS IN IMMUNOLOGY. 2021;12:694853
Structure-Function Relationships of Covalent and Non-Covalent BTK Inhibitors
Zain R; Vihinen M
Review: FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY. 2021;9:630942
Comparative Analysis of BTK Inhibitors and Mechanisms Underlying Adverse Effects
Estupinan HY; Berglof A; Zain R; Smith CIE
Conference publication: BLOOD. 2019;134:5759
Combination of Gatekeeper Mutations and Cysteine 481 Replacement Causes Super Resistance to the Irreversible BTK Inhibitors Ibrutinib, Acalabrutinib and Zanubrutinib
Velasquez HYE; Shi Y; Mohammad DK; Wang Q; Vihinen M; Zain R; Berglof A; Smith ECI
Review: NEUROTHERAPEUTICS. 2019;16(2):248-262
Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases
Zain R; Smith CIE
Review: ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY. 2019;59:605-630
Therapeutic Oligonucleotides: State of the Art
Smith CIE; Zain R
Conference publication: MOLECULAR THERAPY. 2017;25(5):123
Oligonucleotides Targeting CTG Repeats in DNA Down-Regulate Huntingtin
Smith CIE; Zaghloul EM; Gissberg O; Zain R; Wengel J; Lundin KE
Conference publication: HUMAN GENE THERAPY. 2015;26(10):A47
How to optimize TFO binding of DNA in the context of bisLNA
Pabon YV; Lundin KE; Wengel J; Smith CIE; Zain R
Conference publication: HUMAN GENE THERAPY. 2015;26(10):A44
Aptamer delivery of a potent antitumor LNA-oligonucleotide
Gissberg O; Zaghloul E; Lundin K; Zain R; Wengel J; Smith CIE
Review: CELLULAR AND MOLECULAR LIFE SCIENCES. 2003;60(5):862-870
Do natural DNA triple-helical structures occur and function in vivo?
Zain R; Sun JS
Published conference paper: NUCLEOSIDES, NUCLEOTIDES AND NUCLEIC ACIDS. 1997;16(7-9):1661-1662
Studies on nucleoside H-phosphonothioates as synthons in the synthesis of oligonucleotide analogues
Zain R; Bollmark M; Stawinski J
Published conference paper: COLLECTION OF CZECHOSLOVAK CHEMICAL COMMUNICATIONS. 1996;61:S68-S71
Efficient method for the preparation of nucleoside phosphorofluoridates and nucleoside phosphorofluoridothioates
Bollmark M; Zain R; Stawinski J
Published conference paper: NUCLEOSIDES, NUCLEOTIDES AND NUCLEIC ACIDS. 1995;14(3-5):711-714
CONVERSION OF NUCLEOSIDE H-PHOSPHONATE MONOESTERS TO THE CORRESPONDING H-PHOSPHONOTHIOATES - P-31 NMR-STUDIES
STAWINSKI J; ZAIN R

Grants

Diagnostic, prevention and treatment platform for tandem-repeat expansion diseases.
VINNOVA
15 May 2022 - 14 May 2024
Unique Drug Candidate for Treatment and Prevention of Nucleotide Triplet-Repeat Diseases
VINNOVA
15 June 2019 - 15 August 2021
Unique Drug Candidate for Treatment and Prevention of Huntington’s Disease
VINNOVA
15 June 2018 - 15 June 2019

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