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  1. Book: Antisense drug technology

    Crooke, Stanley T.

    principles, strategies, and applications

    2008  

    Author's details ed. by Stanley T. Crooke
    Keywords Oligonucleotides, Antisense / therapeutic use
    Language English
    Size XVII, 825 S. : Ill., graph. Darst.
    Edition 2. ed.
    Publisher CRC Press
    Publishing place Boca Raton, Fla. u.a.
    Publishing country United States
    Document type Book
    HBZ-ID HT015060692
    ISBN 0-84938-796-5 ; 0-8493-8796-5 ; 978-0-8493-8796-8 ; 978-0-84938-796-8
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    2007 A 4498 order for loan Magazin

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  2. Article ; Online: Establishing an environment in which rigorous scientific inquiry is practiced: a personal journey.

    Crooke, Stanley T

    Nucleic acids research

    2022  Volume 50, Issue 13, Page(s) 7216–7223

    Abstract: For more than three decades, Ionis Pharmaceutics has pursued the challenging mission of creating a new platform for drug discovery. To overcome the numerous challenges faced required the integration of innovation across many scientific areas, despite ... ...

    Abstract For more than three decades, Ionis Pharmaceutics has pursued the challenging mission of creating a new platform for drug discovery. To overcome the numerous challenges faced required the integration of innovation across many scientific areas, despite many disappointments and failures. The approaches implemented to create and maintain a scientific environment to achieve the mission demanded the rigorous practice of science over three decades. The approaches taken are discussed in this perspective.
    MeSH term(s) Drug Discovery ; Drug Industry ; Research ; Science
    Language English
    Publishing date 2022-07-08
    Publishing country England
    Document type Journal Article
    ZDB-ID 186809-3
    ISSN 1362-4962 ; 1362-4954 ; 0301-5610 ; 0305-1048
    ISSN (online) 1362-4962 ; 1362-4954
    ISSN 0301-5610 ; 0305-1048
    DOI 10.1093/nar/gkac526
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 1067: Show issues Location:
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    bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular
    Jg. 1995 - 2021: Lesesall (1.OG)
    ab Jg. 2022: Lesesaal (EG)

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  3. Article ; Online: Meeting the needs of patients with ultrarare diseases.

    Crooke, Stanley T

    Trends in molecular medicine

    2022  Volume 28, Issue 2, Page(s) 87–96

    Abstract: Patients with ultrarare diseases present unique challenges to the health care systems of developed economies that demand novel approaches, beginning with achieving a diagnosis and concluding with long-term treatment. The challenges derive from numbers. ... ...

    Abstract Patients with ultrarare diseases present unique challenges to the health care systems of developed economies that demand novel approaches, beginning with achieving a diagnosis and concluding with long-term treatment. The challenges derive from numbers. On the one hand, the rarity of the disease phenotypes means that the vast majority of ultrarare patients are never diagnosed, and for the fortunate few who are diagnosed, the journey to a genetic diagnosis is long and perilous. On the other hand, as more human genomes are sequenced, the number of these patients identified is growing logarithmically. Once patients are diagnosed, personalized medicines must be rapidly developed and delivered. Here I define the problems and propose a nonprofit model to meet the needs of some of these patients.
    MeSH term(s) Delivery of Health Care ; Genome, Human ; Humans ; Phenotype ; Precision Medicine
    Language English
    Publishing date 2022-01-07
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 2036490-8
    ISSN 1471-499X ; 1471-4914
    ISSN (online) 1471-499X
    ISSN 1471-4914
    DOI 10.1016/j.molmed.2021.12.002
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 4345: Show issues Location:
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  4. Article ; Online: Progress in molecular biology and translational science addressing the needs of nano-rare patients.

    Crooke, Stanley T

    Progress in molecular biology and translational science

    2022  Volume 190, Issue 1, Page(s) 127–146

    Abstract: The healthcare systems in the developed economies were established primarily to address the more prevalent diseases and have been customized to support the provision of therapeutics to rare patients. However, with the ever-broader implementation of ... ...

    Abstract The healthcare systems in the developed economies were established primarily to address the more prevalent diseases and have been customized to support the provision of therapeutics to rare patients. However, with the ever-broader implementation of genomic sequencing, it is clear that there are significantly more disease-causing mutations in the human genome than realized and that many mutations are much rarer than current definitions of rare disease populations. Given this, I propose parsing patient populations and defining patient populations more precisely. Nano-rare patients are defined as patients having disease-causing mutations that are unique to a single patient or having a known worldwide prevalence of less than 30. These patient populations present unique challenges to healthcare systems that demand the development of novel models for delivery of therapeutics and novel, more efficient drug discovery technologies, such as antisense technology. The challenges presented by nano-rare patients, a novel non-profit model as a means of providing experimental treatments rather than the traditional commercial model, and progress in establishing a non-profit solution are discussed.
    MeSH term(s) Drug Discovery ; Humans ; Molecular Biology ; Rare Diseases/genetics ; Rare Diseases/therapy ; Translational Science, Biomedical
    Language English
    Publishing date 2022-06-03
    Publishing country Netherlands
    Document type Journal Article
    ZDB-ID 2471995-X
    ISSN 1878-0814 ; 0079-6603 ; 1877-1173
    ISSN (online) 1878-0814
    ISSN 0079-6603 ; 1877-1173
    DOI 10.1016/bs.pmbts.2022.04.002
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: A call to arms against ultra-rare diseases.

    Crooke, Stanley T

    Nature biotechnology

    2021  Volume 39, Issue 6, Page(s) 671–677

    MeSH term(s) Humans ; Oligonucleotides, Antisense/economics ; Oligonucleotides, Antisense/therapeutic use ; Organizations, Nonprofit ; Rare Diseases/drug therapy
    Chemical Substances Oligonucleotides, Antisense
    Language English
    Publishing date 2021-05-10
    Publishing country United States
    Document type Journal Article
    ZDB-ID 1311932-1
    ISSN 1546-1696 ; 1087-0156
    ISSN (online) 1546-1696
    ISSN 1087-0156
    DOI 10.1038/s41587-021-00945-0
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 2167: Show issues Location:
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    Zs.MO 137: Show issues
    Zs.MG 43: Show issues

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  6. Article ; Online: Addressing the Needs of Patients with Ultra-Rare Mutations One Patient at a Time: The n-Lorem Approach.

    Crooke, Stanley T

    Nucleic acid therapeutics

    2021  Volume 32, Issue 2, Page(s) 95–100

    Abstract: Thanks to the advent of genomic sequencing and numerous personalized medicine initiatives in various medical centers, it is now known that there are many patients who have heretofore never been diagnosed who have mutations that are unique to them and ... ...

    Abstract Thanks to the advent of genomic sequencing and numerous personalized medicine initiatives in various medical centers, it is now known that there are many patients who have heretofore never been diagnosed who have mutations that are unique to them and them only and others that may be members of an extremely rare mutation (<30 patients in the world). Although each mutation may be unique it is now estimated that there are millions of these unique or vanishingly small patient groups. Patients with diseases caused by ultra-rare mutations present challenges to the health care system that are as unique as their mutation. n-Lorem was founded to take advantage of the antisense technology that we created at Ionis to discover and develop personalized antisense oligonucleotides (ASOs) one patient at a time and provide those experimental ASO treatments for free for life. In our first 18 months of operation, we have demonstrated this goal is achievable and worked with the FDA to develop guidance for ASO treatment of patients with ultra-rare diseases. In this article, I define the problem, discuss the ASO solution, and our progress at n-Lorem to date. I then focus on important steps that we have taken to assure that these complex risk/benefit judgments are made with high quality and that each patient receives the highest quality ASO possible. I then describe the processes we have created to assure that the opportunity to learn from each patient and our aggregate experience are maximized and shared with all stakeholders.
    MeSH term(s) Humans ; Mutation ; Oligonucleotides, Antisense/genetics ; Oligonucleotides, Antisense/therapeutic use ; Precision Medicine
    Chemical Substances Oligonucleotides, Antisense
    Language English
    Publishing date 2021-09-13
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2639888-6
    ISSN 2159-3345 ; 2159-3337
    ISSN (online) 2159-3345
    ISSN 2159-3337
    DOI 10.1089/nat.2021.0046
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article: Characterization of cooperative PS-oligo activation of human TLR9.

    Pollak, Adam J / Zhao, Luyi / Crooke, Stanley T

    Molecular therapy. Nucleic acids

    2023  Volume 33, Page(s) 832–844

    Abstract: Single-stranded phosphorothioate oligonucleotides (PS-oligos) can activate TLR9, leading to an innate immune response. This can occur with PS-oligos containing unmethylated CpG sites, the canonical motif, or PS-oligos that do not contain those motifs ( ... ...

    Abstract Single-stranded phosphorothioate oligonucleotides (PS-oligos) can activate TLR9, leading to an innate immune response. This can occur with PS-oligos containing unmethylated CpG sites, the canonical motif, or PS-oligos that do not contain those motifs (non-CpG). Structural evidence shows that TLR9 contains two PS-oligo binding sites, and recent data suggest that synergistic cooperative activation of TLR9 can be achieved by adding two separate PS-oligos to cells, each engaging with a separate site on TLR9 to enhance TLR9 activation as a pair. Here, we demonstrate and characterize this cooperativity phenomenon using PS-oligos in human cell lines, and we introduce several novel PS-oligo pairs (CpG and non-CpG pairs) that show cooperative activation. Indeed, we find that cooperative PS-oligos likely bind at different sites on TLR9. Interestingly, we find that PS-oligos that generate little TLR9 activation on their own can prime TLR9 to be activated by other PS-oligos. Finally, we determine that previous models of TLR9 activation cannot be used to fully explain data from systems using human TLR9 and PS-oligos. Overall, we reveal new details of TLR9 activation, but we also find that more work needs to be done to determine where certain PS-oligos are binding to TLR9.
    Language English
    Publishing date 2023-08-15
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2662631-7
    ISSN 2162-2531
    ISSN 2162-2531
    DOI 10.1016/j.omtn.2023.08.011
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Systematic Analysis of Chemical Modifications of Phosphorothioate Antisense Oligonucleotides that Modulate Their Innate Immune Response.

    Pollak, Adam J / Zhao, Luyi / Crooke, Stanley T

    Nucleic acid therapeutics

    2023  Volume 33, Issue 2, Page(s) 95–107

    Abstract: While rare, some gapmer phosphorothioate (PS) antisense oligonucleotides (ASOs) can induce a noncanonical TLR9-dependent innate immune response. In this study, we performed systematic analyses of the roles of PS ASO backbone chemistry, 2' modifications, ... ...

    Abstract While rare, some gapmer phosphorothioate (PS) antisense oligonucleotides (ASOs) can induce a noncanonical TLR9-dependent innate immune response. In this study, we performed systematic analyses of the roles of PS ASO backbone chemistry, 2' modifications, and sequence in PS ASO induced TLR9 signaling. We found that each of these factors can contribute to altering PS ASO induced TLR9 signaling, and in some cases the effects are quite dramatic. We also found that the positioning (5' vs. 3') of a particular backbone or 2' modification within a PS ASO can affect its TLR9 signaling. Interestingly, medicinal chemical strategies that decrease TLR9 signaling for one sequence can have opposing effects on another sequence. Our results demonstrate that TLR9 signaling is highly PS ASO sequence dependent, the mechanism of which remains unknown. Despite this, we determined that placement of two mesyl phosphoramidate linkages within the PS ASO gap is the most promising strategy to mitigate PS ASO dependent TLR9 activation to enhance the therapeutic index and, therefore, further streamline PS ASO drug development.
    MeSH term(s) Oligonucleotides, Antisense/genetics ; Toll-Like Receptor 9/genetics ; Phosphorothioate Oligonucleotides/genetics
    Chemical Substances Oligonucleotides, Antisense ; Toll-Like Receptor 9 ; Phosphorothioate Oligonucleotides
    Language English
    Publishing date 2023-02-07
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2639888-6
    ISSN 2159-3345 ; 2159-3337
    ISSN (online) 2159-3345
    ISSN 2159-3337
    DOI 10.1089/nat.2022.0067
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Book: Antisense research and application

    Agrawal, Sudhir / Crooke, Stanley T.

    (Handbook of experimental pharmacology ; 131)

    1998  

    Author's details contributors S. Agrawal ... Ed. Stanley T. Crooke
    Series title Handbook of experimental pharmacology ; 131
    Collection
    Keywords Antisense-Oligonucleotide
    Subject Triplex-Bildner ; Antisense-Pharmakon ; Antisense-Oligomere ; Antisense-Oligodeoxynucleotide ; Phosphorothioat-Oligonucleotide ; S-Oligos ; Antisense-Oligonukleotide
    Language English
    Size XXVI, 630 S. : graph. Darst.
    Publisher Springer
    Publishing place Berlin u.a.
    Document type Book
    HBZ-ID HT008399482
    ISBN 3-540-63833-4 ; 978-3-540-63833-9
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    1998 A 4653 order for loan Magazin
    2004 A 6648 order for loan Magazin

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  10. Book: Antisense research and applications

    Crooke, Stanley T.

    1993  

    Author's details ed. by Stanley T. Crooke
    Keywords Antisense Elements (Genetics) ; DNA, Antisense / therapeutic use ; Oligonucleotides, Antisense / therapeutic use ; Oligonucleotide ; Heilmittel
    Subject Therapeutikum ; Therapeutische Leistung ; Oligonukleotide
    Language English
    Size [12], 579 S. : Ill., graph. Darst.
    Publisher CRC Press
    Publishing place Boca Raton u.a.
    Publishing country United States
    Document type Book
    HBZ-ID HT006128353
    ISBN 0-8493-4705-X ; 978-0-8493-4705-4
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    1994 A 416 order for loan Magazin

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