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  1. CRISPR Off-Target Analysis Platforms

    The clustered regularly interspaced short palindromic repeats (CRISPR)-Caspase9 (Cas9) system provides a programmable technology that may be used to...
    Christine L. Xu, Merry Zhechao Ruan, ... Stephen H. Tsang in Retinitis Pigmentosa
    Protocol 2023
  2. Human genetic diversity alters off-target outcomes of therapeutic gene editing

    CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat disease. However, standard computational and biochemical...

    Samuele Cancellieri, **g Zeng, ... Luca Pinello in Nature Genetics
    Article 15 December 2022
  3. AttSiOff: a self-attention-based approach on siRNA design with inhibition and off-target effect prediction

    Small interfering RNA (siRNA) is often used for function study and expression regulation of specific genes, as well as the development of small...

    Bin Liu, Ye Yuan, ... Cheng ** in Med-X
    Article Open access 23 April 2024
  4. Affinity fine-tuning anti-CAIX CAR-T cells mitigate on-target off-tumor side effects

    One of the major hurdles that has hindered the success of chimeric antigen receptor (CAR) T cell therapies against solid tumors is on-target...

    Yufei Wang, Alicia Buck, ... Wayne A. Marasco in Molecular Cancer
    Article Open access 16 March 2024
  5. Off-target activity of NBOMes and NBOMe analogs at the µ opioid receptor

    New psychoactive substances (NPS) are introduced on the illicit drug market at a rapid pace. Their molecular targets are often inadequately...

    Marie H. Deventer, Mattias Persson, ... Christophe P. Stove in Archives of Toxicology
    Article 28 February 2023
  6. Reduction of Off-Target Effects of Gapmer Antisense Oligonucleotides by Oligonucleotide Extension

    Aim

    Antisense oligonucleotide (ASO) has the potential to induce off-target effects by inadvertent binding of ASOs to unintended RNAs that have a...

    Hidenori Yasuhara, Tokuyuki Yoshida, ... Takao Inoue in Molecular Diagnosis & Therapy
    Article Open access 07 January 2022
  7. Fcγ Receptor-Dependent Internalization and Off-Target Cytotoxicity of Antibody-Drug Conjugate Aggregates

    Purpose

    Antibody-drug conjugates (ADCs), which are monoclonal antibodies (mAbs) conjugated with highly toxic payloads, achieve high tumor killing...

    Michihiko Aoyama, Minoru Tada, ... Akiko Ishii-Watabe in Pharmaceutical Research
    Article Open access 27 December 2021
  8. Pharmacological inhibitors of the cystic fibrosis transmembrane conductance regulator exert off-target effects on epithelial cation channels

    The cystic fibrosis transmembrane conductance regulator (CFTR) anion channel and the epithelial Na + channel (ENaC) play essential roles in...

    **Heng Lin, Sean M. Gettings, ... Michael A. Gray in Pflügers Archiv - European Journal of Physiology
    Article Open access 07 October 2022
  9. Disrupting off-target Cas9 activity in the liver

    Sean A. Dilliard, Daniel J. Siegwart in Nature Biomedical Engineering
    Article 21 February 2022
  10. Characterizing off-target corticospinal responses to double-cone transcranial magnetic stimulation

    Introduction

    The double-cone coil (D-CONE) is frequently used in transcranial magnetic stimulation (TMS) experiments that target the motor cortex (M1)...

    F. Proessl, M. C. Canino, ... S. D. Flanagan in Experimental Brain Research
    Article 06 February 2021
  11. Organ-specific off-target effects of Pim/ZIP kinase inhibitors suggest lack of contractile Pim kinase activity in prostate, bladder, and vascular smooth muscle

    Smooth muscle contraction by Pim kinases and ZIPK has been suggested, but evidence for lower urinary tract organs or using Pim-selective inhibitor...

    Sheng Hu, Moritz Trieb, ... Martin Hennenberg in Naunyn-Schmiedeberg's Archives of Pharmacology
    Article Open access 01 September 2023
  12. In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

    Base editors are RNA-programmable deaminases that enable precise single-base conversions in genomic DNA. However, off-target activity is a concern in...

    Lukas Villiger, Tanja Rothgangl, ... Gerald Schwank in Nature Biomedical Engineering
    Article 25 January 2021
  13. Using RNA-seq to Assess Off-Target Effects of Antisense Oligonucleotides in Human Cell Lines

    Background

    The field of antisense oligonucleotide therapeutics is rapidly growing and in addition to small molecules and therapeutic antibodies,...

    Sven Michel, Ksenija Schirduan, ... Frank Jaschinski in Molecular Diagnosis & Therapy
    Article 12 December 2020
  14. Selection of Chemical Modifications in the siRNA Seed Region That Repress Off-Target Effect

    RNA interference mediated by small interfering RNA (siRNA) has been widely used as a procedure to knock down the expression of an intended target...
    Yoshiaki Kobayashi, Kengo Miyamoto, ... Kumiko Ui-Tei in Design and Delivery of SiRNA Therapeutics
    Protocol 2021
  15. Much ado about nothing? Off-target amplification can lead to false-positive bacterial brain microbiome detection in healthy and Parkinson’s disease individuals

    Background

    Recent studies suggested the existence of (poly-)microbial infections in human brains. These have been described either as putative...

    Janis R. Bedarf, Naiara Beraza, ... Falk Hildebrand in Microbiome
    Article Open access 26 March 2021
  16. Improved analysis of CRISPR fitness screens and reduced off-target effects with the BAGEL2 gene essentiality classifier

    Background

    Identifying essential genes in genome-wide loss-of-function screens is a critical step in functional genomics and cancer target finding. We...

    Eiru Kim, Traver Hart in Genome Medicine
    Article Open access 06 January 2021
  17. Mitigating off-target distribution and enhancing cytotoxicity in breast cancer cells with alpha-ketoglutaric acid-modified Fe/Mg-CA nanoparticles

    Purpose

    In this work, pH-sensitive alpha-ketoglutaric acid-modified Fe/Mg-carbonate apatite (α-KAM-Fe/Mg-CA) NPs were introduced and found to be...

    Sheikh Tanzina Haque, Md. Emranul Karim, ... Ezharul Hoque Chowdhury in Journal of Pharmaceutical Investigation
    Article Open access 05 April 2022
  18. Mitigating off-target effects in CRISPR/Cas9-mediated in vivo gene editing

    The rapid advancement of genome editing technologies has opened up new possibilities in the field of medicine. Nuclease-based techniques such as the...

    Hua Alexander Han, Jeremy Kah Sheng Pang, Boon-Seng Soh in Journal of Molecular Medicine
    Article Open access 20 March 2020
  19. Do Fasting, CR and FMD Improve the Chemotherapy Response, Reduce Off-Target Toxicities and Enhance Antitumour Immunity? Illusion or Clinical Reality?

    Besides the great strides of efforts made towards understanding the effect of fasting, calorie restriction (CR) and fasting-mimicking diet (FMD), yet...
    Rajesh N. Gacche in Dietary Research and Cancer
    Chapter 2021
  20. Structure-guided discovery of highly efficient cytidine deaminases with sequence-context independence

    The applicability of cytosine base editors is hindered by their dependence on sequence context and by off-target effects. Here, by using AlphaFold2...

    Kui Xu, Hu Feng, ... Erwei Zuo in Nature Biomedical Engineering
    Article Open access 03 June 2024
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