Rula Zain

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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 influence biological function.

Estupiñán HY; Bouderlique T; He C; Berglöf A; Cappelleri A; Frengen N; Zain R; Karlsson MCI; Månsson R; Smith CIE
Article: NUCLEIC ACIDS RESEARCH. 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; Cebrián 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

Exposure to Therapeutic BTK Inhibitors Induces Phenocopying of Btk29A Mutants in the Fruit Fly Drosophila melanogaster.

Hamada-Kawaguchi N; Nore BF; Zain R; Engström 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 Anti-Gene Oligonucleotides.

Saher O; Zaghloul EM; Umek T; Hagey DW; Mozafari N; Danielsen MB; Gouda AS; Lundin KE; Jørgensen PT; 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; Strömberg 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; Görgens A; Smith CE; 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; Sällberg Chen M; Zain R; Hansson L; Österborg 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.

Estupiñán HY; Wang Q; Berglöf 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; Peña-Pérez L; Berglöf A; Meinke S; Estupiñán HY; Heimersson K; Zain R; Månsson 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.

Estupiñán HY; Bouderlique T; He C; Berglöf A; Gupta D; Saher O; Daza Cruz MÁ; Peña-Perez L; Yu L; Zain R; Karlsson MCI; Månsson 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; Andaloussi SE; 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; Lönnberg 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; Zaghloul 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; Görgens A; Andaloussi SE; 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; Jørgensen 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. 2017;45(9):5153-5169

CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression.

Zaghloul EM; Gissberg O; Moreno PMD; Siggens L; Hällbrink M; Jørgensen 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 & 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; Strömberg 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; Nguyen C-H; Landras-Guetta C; Wengel J; Zain R; Smith CIE
Article: NUCLEIC ACIDS RESEARCH. 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; Jørgensen 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; Álvarez-Asencio R; Nguyen C-H; 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; Nguyen C-H; Zain R; Lee JS; Wu Y
Article: NUCLEIC ACIDS RESEARCH. 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; Andaloussi SE; Jørgensen 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; Fernández 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 J-S; Nguyen C-H; 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 J-S; Nguyen C-H; 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 C-H; Blouquit Y; Bisagni E; Garestier T; Grierson DS; Sun J-S
Article: CHEMISTRY AND BIOLOGY. 1999;6(11):771-777

Design of a triple-helix-specific cleaving reagent.

Zain R; Marchand C; Sun J; 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 & 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 (31)P 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 & 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 & 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

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; Österborg A; Palma M; Buggert M; Bergman P; Bryceson Y
Review: FRONTIERS IN IMMUNOLOGY. 2021;12:694853

Structure-Function Relationships of Covalent and Non-Covalent BTK Inhibitors.

Zain R; Vihinen M
Editorial comment: FRONTIERS IN IMMUNOLOGY. 2021;12:804735

Editorial: New Insights on Bruton's Tyrosine Kinase Inhibitors.

Smith CIE; Brown JR; Zain R
Review: FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY. 2021;9:630942

Comparative Analysis of BTK Inhibitors and Mechanisms Underlying Adverse Effects.

Estupiñán HY; Berglöf 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 J-S
Published conference paper: NUCLEOSIDES NUCLEOTIDES & 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 & 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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