Extracellular vesicle in vivo biodistribution is determined by cell source, route of administration and targeting von Wiklander, Oscar P. B., Nordin, Joel Z., O'Loughlin, Aisling, Gustafsson, Ylva, Corso, Giulia, Mäger, Imre, Vader, Pieter, Lee, Yi, Sork, Helena, Seow, Yiqi, Heldring, Nina, Alvarez-Erviti, Lydia, Smith, CI Edvard, Le Blanc, Katarina, Macchiarini, Paolo, Jungebluth, Philipp, Wood, Matthew J. A., Andaloussi, Samir EL

Volltext

Therapeutic Oligonucleotides: State of the Art von Smith, C.I. Edvard, Zain, Rula

Volltext

Diagnostics of primary immunodeficiency diseases: a sequencing capture approach von Moens, Lotte N, Falk-Sörqvist, Elin, Asplund, A Charlotta, Bernatowska, Ewa, Smith, C I Edvard, Nilsson, Mats

Volltext

Bruton's tyrosine kinase (Btk): function, regulation, and transformation with special emphasis on the PH domain von Mohamed, Abdalla J, Yu, Liang, Bäckesjö, Carl-Magnus, Vargas, Leonardo, Faryal, Rani, Aints, Alar, Christensson, Birger, Berglöf, Anna, Vihinen, Mauno, Nore, Beston F, Edvard Smith, C.I

Volltext

The ability of locked nucleic acid oligonucleotides to pre-structure the double helix: A molecular simulation and binding study von Xu, You, Gissberg, Olof, Pabon-Martinez, Y Vladimir, Wengel, Jesper, Lundin, Karin E, Smith, C I Edvard, Zain, Rula, Nilsson, Lennart, Villa, Alessandra

Volltext

Oligonucleotide⁻Palladacycle Conjugates as Splice-Correcting Agents von Hande, Madhuri, Saher, Osama, Lundin, Karin E, Smith, C I Edvard, Zain, Rula, Lönnberg, Tuomas

Volltext

Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration von Roberts, Thomas C, Johansson, Henrik J, McClorey, Graham, Godfrey, Caroline, Blomberg, K Emelie M, Coursindel, Thibault, Gait, Michael J, Smith, C I Edvard, Lehtiö, Janne, El Andaloussi, Samir, Wood, Matthew J A

Volltext

Oligonucleotide Binding to Non-B-DNA in MYC von Umek, Tea, Sollander, Karin, Bergquist, Helen, Wengel, Jesper, Lundin, Karin E, Smith, C I Edvard, Zain, Rula

Volltext

Comparative Analysis of BTK Inhibitors and Mechanisms Underlying Adverse Effects von Estupiñán, H Yesid, Berglöf, Anna, Zain, Rula, Smith, C I Edvard

Volltext

Dual Phosphorylation of Btk by Akt/Protein Kinase B Provides Docking for 14-3-3ζ, Regulates Shuttling, and Attenuates both Tonic and Induced Signaling in B Cells von Mohammad, Dara K., Nore, Beston F., Hussain, Alamdar, Gustafsson, Manuela O., Mohamed, Abdalla J., Smith, C. I. Edvard

Volltext

A Peptide-based Vector for Efficient Gene Transfer In Vitro and In Vivo von Lehto, Taavi, Simonson, Oscar E, Mäger, Imre, Ezzat, Kariem, Sork, Helena, Copolovici, Dana-Maria, Viola, Joana R, Zaghloul, Eman M, Lundin, Per, Moreno, Pedro MD, Mäe, Maarja, Oskolkov, Nikita, Suhorutšenko, Julia, Smith, CI Edvard, Andaloussi, Samir EL

Volltext

Disruption of Higher Order DNA Structures in Friedreich's Ataxia (GAA)n Repeats by PNA or LNA Targeting von Bergquist, Helen, Rocha, Cristina S J, Álvarez-Asencio, Rubén, Nguyen, Chi-Hung, Rutland, Mark W, Smith, C I Edvard, Good, Liam, Nielsen, Peter E, Zain, Rula

Volltext

Resistance Mutations to BTK Inhibitors Originate From the NF-κB but Not From the PI3K-RAS-MAPK Arm of the B Cell Receptor Signaling Pathway von Smith, C I Edvard, Burger, Jan A

Volltext

BTK gatekeeper residue variation combined with cysteine 481 substitution causes super-resistance to irreversible inhibitors acalabrutinib, ibrutinib and zanubrutinib von Estupiñán, H. Yesid, Wang, Qing, Berglöf, Anna, Schaafsma, Gerard C. P., Shi, Yuye, Zhou, Litao, Mohammad, Dara K., Yu, Liang, Vihinen, Mauno, Zain, Rula, Smith, C. I. Edvard

Volltext

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... von Rastokina, Anastasia, Cebrián, Jorge, Mozafari, Negin, Mandel, Nicholas H, Smith, C I Edvard, Lopes, Massimo, Zain, Rula, Mirkin, Sergei M

Volltext

Nuclease resistant oligonucleotides with cell penetrating properties von Milton, Stefan, Honcharenko, Dmytro, Rocha, Cristina S J, Moreno, Pedro M D, Smith, C I Edvard, Strömberg, Roger

Volltext

Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy von Roberts, Thomas C, Blomberg, K Emelie M, McClorey, Graham, Andaloussi, Samir EL, Godfrey, Caroline, Betts, Corinne, Coursindel, Thibault, Gait, Michael J, Edvard Smith, CI, Wood, Matthew JA

Volltext

ANKRD54 preferentially selects Bruton's Tyrosine Kinase (BTK) from a Human Src-Homology 3 (SH3) domain library von Gustafsson, Manuela O, Mohammad, Dara K, Ylösmäki, Erkko, Choi, Hyunseok, Shrestha, Subhash, Wang, Qing, Nore, Beston F, Saksela, Kalle, Smith, C I Edvard

Volltext

Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides von Bost, Jeremy P., Ojansivu, Miina, Munson, Michael J., Wesén, Emelie, Gallud, Audrey, Gupta, Dhanu, Gustafsson, Oskar, Saher, Osama, Rädler, Julia, Higgins, Stuart G., Lehto, Taavi, Holme, Margaret N., Dahlén, Anders, Engkvist, Ola, Strömstedt, Per-Erik, Andersson, Shalini, Edvard Smith, C. I., Stevens, Molly M., Esbjörner, Elin K., Collén, Anna, El Andaloussi, Samir

Volltext

Single-stranded DNA oligonucleotides inhibit TLR3-mediated responses in human monocyte-derived dendritic cells and in vivo in cynomolgus macaques von Sköld, Annette E., Hasan, Maroof, Vargas, Leonardo, Saidi, Hela, Bosquet, Nathalie, Le Grand, Roger, Smith, C. I. Edvard, Spetz, Anna-Lena

Volltext