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  1. Book: Muscular dystrophy therapeutics

    Maruyama, Rika / Yokota, Toshifumi

    methods and protocols

    (Methods in molecular biology ; 2587 ; Springer protocols)

    2023  

    Author's details edited by Rika Maruyama and Toshifumi Yokota
    Series title Methods in molecular biology ; 2587
    Springer protocols
    Collection
    Keywords Muscular dystrophy/Treatment
    Subject code 616.74806
    Language English
    Size xv, 575 Seiten, Illustrationen, 26 cm
    Publisher Humana Press
    Publishing place New York, NY
    Publishing country United States
    Document type Book
    Note Includes bibliographical references and index
    HBZ-ID HT021687587
    ISBN 978-1-0716-2771-6 ; 9781071627723 ; 1-0716-2771-6 ; 1071627724
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    Zs A 7042 -2587- not available for loan Lesesaal EG

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  2. Book: Gapmers

    Yokota, Toshifumi / Maruyama, Rika

    methods and protocols

    (Methods in molecular biology ; 2176 ; Springer protocols)

    2020  

    Author's details edited by Yokota Toshifumi, Rika Maruyama
    Series title Methods in molecular biology ; 2176
    Springer protocols
    Collection
    Language English
    Size xi, 247 Seiten, Illustrationen
    Publisher Humana Press
    Publishing place New York, NY
    Publishing country United States
    Document type Book
    HBZ-ID HT020579125
    ISBN 978-1-0716-0770-1 ; 9781071607718 ; 1-0716-0770-7 ; 1071607715
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    Zs A 7042 -2176- verfügbar in Königswinter Königswinter

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  3. Book: Exon skipping and inclusion therapies

    Yokota, Toshifumi / Maruyama, Rika

    methods and protocols

    (Methods in molecular biology ; 1828 ; Springer protocols)

    2018  

    Author's details edited by Toshifumi Yokota, Rika Maruyama
    Series title Methods in molecular biology ; 1828
    Springer protocols
    Collection
    Language English
    Size xv, 569 Seiten, Illustrationen
    Publisher Humana Press
    Publishing place New York, NY
    Publishing country United States
    Document type Book
    HBZ-ID HT019807841
    ISBN 978-1-4939-8650-7 ; 9781493986514 ; 1-4939-8650-3 ; 1493986511
    Database Catalogue ZB MED Medicine, Health

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    In stock of ZB MED Cologne/Königswinter

    Shelf markLoan statusLocation
    Zs A 7042 -1828- verfügbar in Königswinter Königswinter

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  4. Article ; Online: The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments.

    Anwar, Saeed / Yokota, Toshifumi

    Biomolecules

    2024  Volume 14, Issue 3

    Abstract: Dysferlinopathies refer to a spectrum of muscular dystrophies that cause progressive muscle weakness and degeneration. They are caused by mutations in ... ...

    Abstract Dysferlinopathies refer to a spectrum of muscular dystrophies that cause progressive muscle weakness and degeneration. They are caused by mutations in the
    MeSH term(s) Animals ; Muscle Proteins/genetics ; Membrane Proteins/genetics ; Muscular Dystrophies, Limb-Girdle/genetics ; Muscular Dystrophies, Limb-Girdle/therapy ; Muscular Dystrophies, Limb-Girdle/metabolism ; Muscular Dystrophies/genetics ; Mutation
    Chemical Substances Muscle Proteins ; Membrane Proteins
    Language English
    Publishing date 2024-02-21
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2701262-1
    ISSN 2218-273X ; 2218-273X
    ISSN (online) 2218-273X
    ISSN 2218-273X
    DOI 10.3390/biom14030256
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Duchenne muscular dystrophy: promising early-stage clinical trials to watch.

    Tang, Annie / Yokota, Toshifumi

    Expert opinion on investigational drugs

    2024  Volume 33, Issue 3, Page(s) 201–217

    Abstract: Introduction: Current therapies are unable to cure Duchenne muscular dystrophy (DMD), a severe and common form of muscular dystrophy, and instead aim to delay disease progression. Several treatments currently in phase I trials could increase the number ... ...

    Abstract Introduction: Current therapies are unable to cure Duchenne muscular dystrophy (DMD), a severe and common form of muscular dystrophy, and instead aim to delay disease progression. Several treatments currently in phase I trials could increase the number of therapeutic options available to patients.
    Areas covered: This review aims to provide an overview of current treatments undergoing or having recently undergone early-stage trials. Several exon-skipping and gene therapy approaches are currently being investigated at the clinical stage to address an unmet need for DMD treatments. This article also covers Phase I trials from the last 5 years that involve inhibitors, small molecules, a purified synthetic flavanol, a cell-based therapy, and repurposed cardiac or tumor medications.
    Expert opinion: With antisense oligonucleotide (AON) treatments making up the majority of conditionally approved DMD therapies, most of the clinical trials occurring within the last 5 years have also evaluated exon-skipping AONs. The approval of Elevidys, a micro-dystrophin therapy, is reflected in a recent trend toward gene transfer therapies in phase I DMD clinical trials, but their safety and efficacy are being established in this phase of development. Other Phase I clinical-stage approaches are diverse, but have a range in efficacy, safety, and endpoint measures.
    MeSH term(s) Humans ; Genetic Therapy ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/therapy ; Oligonucleotides, Antisense/therapeutic use ; RNA Splicing ; Clinical Trials as Topic
    Chemical Substances Oligonucleotides, Antisense
    Language English
    Publishing date 2024-02-06
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 1182884-5
    ISSN 1744-7658 ; 0967-8298 ; 1354-3784
    ISSN (online) 1744-7658
    ISSN 0967-8298 ; 1354-3784
    DOI 10.1080/13543784.2024.2313105
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 3739: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
    bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular
    Jg. 1995 - 2021: Lesesall (2.OG)
    ab Jg. 2022: Lesesaal (EG)

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  6. Article: CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy.

    Wilton-Clark, Harry / Yokota, Toshifumi

    Molecular therapy. Methods & clinical development

    2023  Volume 30, Page(s) 500–501

    Language English
    Publishing date 2023-09-04
    Publishing country United States
    Document type News
    ZDB-ID 2872938-9
    ISSN 2329-0501 ; 2329-0501
    ISSN (online) 2329-0501
    ISSN 2329-0501
    DOI 10.1016/j.omtm.2023.08.013
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 7107: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
    bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular
    ab Jg. 2022: Lesesaal (EG)

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  7. Article ; Online: Navigating the Complex Landscape of Fibrodysplasia Ossificans Progressiva: From Current Paradigms to Therapeutic Frontiers.

    Anwar, Saeed / Yokota, Toshifumi

    Genes

    2023  Volume 14, Issue 12

    Abstract: Fibrodysplasia ossificans progressiva (FOP) is an enigmatic, ultra-rare genetic disorder characterized by progressive heterotopic ossification, wherein soft connective tissues undergo pathological transformation into bone structures. This incapacitating ... ...

    Abstract Fibrodysplasia ossificans progressiva (FOP) is an enigmatic, ultra-rare genetic disorder characterized by progressive heterotopic ossification, wherein soft connective tissues undergo pathological transformation into bone structures. This incapacitating process severely limits patient mobility and poses formidable challenges for therapeutic intervention. Predominantly caused by missense mutations in the
    MeSH term(s) Humans ; Myositis Ossificans/genetics ; Myositis Ossificans/therapy ; Proteomics ; Rare Diseases ; Ossification, Heterotopic/genetics ; Ossification, Heterotopic/pathology ; Bone and Bones/pathology
    Language English
    Publishing date 2023-11-30
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2527218-4
    ISSN 2073-4425 ; 2073-4425
    ISSN (online) 2073-4425
    ISSN 2073-4425
    DOI 10.3390/genes14122162
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment.

    Wilton-Clark, Harry / Yokota, Toshifumi

    Med (New York, N.Y.)

    2023  Volume 4, Issue 12, Page(s) 855–856

    Abstract: Gene therapies, notably those leveraging CRISPR and adeno-associated virus vectors (AAVs), have risen to the forefront of potential treatments for neuromuscular disorders. The recent demise of a Duchenne muscular dystrophy (DMD) patient following a trial ...

    Abstract Gene therapies, notably those leveraging CRISPR and adeno-associated virus vectors (AAVs), have risen to the forefront of potential treatments for neuromuscular disorders. The recent demise of a Duchenne muscular dystrophy (DMD) patient following a trial utilizing CRISPR transactivation with AAV has cast a spotlight on the potential risks associated with these approaches.
    MeSH term(s) Humans ; Clustered Regularly Interspaced Short Palindromic Repeats/genetics ; Genetic Vectors/adverse effects ; Genetic Vectors/genetics ; Dependovirus/genetics ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/therapy ; Genetic Therapy/adverse effects
    Language English
    Publishing date 2023-12-01
    Publishing country United States
    Document type Journal Article
    ISSN 2666-6340
    ISSN (online) 2666-6340
    DOI 10.1016/j.medj.2023.11.008
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article: Cardiac therapies for Duchenne muscular dystrophy.

    Shah, Md Nur Ahad / Yokota, Toshifumi

    Therapeutic advances in neurological disorders

    2023  Volume 16, Page(s) 17562864231182934

    Abstract: Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting complications such as loss of skeletal muscle function as well as respiratory and cardiac complications. Advanced therapeutics in pulmonary care have significantly ... ...

    Abstract Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting complications such as loss of skeletal muscle function as well as respiratory and cardiac complications. Advanced therapeutics in pulmonary care have significantly reduced respiratory complication-related mortality, making cardiomyopathy the main determinant factor of survival. While there are multiple therapies such as the use of anti-inflammatory drugs, physical therapy, and ventilatory assistance targeted toward delaying the disease progression in DMD, a cure remains elusive. In the last decade, several therapeutic approaches have been developed to improve patient survival. These include small molecule-based therapy, micro-dystrophin gene delivery, CRISPR-mediated gene editing, nonsense readthrough, exon skipping, and cardiosphere-derived cell therapy. Associated with the specific benefits of each of these approaches are their individual risks and limitations. The variability in the genetic aberrations leading to DMD also limits the widespread use of these therapies. While numerous approaches have been explored to treat DMD pathophysiology, only a handful have successfully advanced through the preclinical stages. In this review, we summarize the currently approved as well as the most promising therapeutics undergoing clinical trials aimed toward treating DMD with a focus on its cardiac manifestations.
    Language English
    Publishing date 2023-07-03
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 2442245-9
    ISSN 1756-2864 ; 1756-2856
    ISSN (online) 1756-2864
    ISSN 1756-2856
    DOI 10.1177/17562864231182934
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article: Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy.

    Wilton-Clark, Harry / Yokota, Toshifumi

    Pharmaceutics

    2023  Volume 15, Issue 3

    Abstract: Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting 1/5000 boys globally, characterized by progressive muscle breakdown and eventual death, with an average lifespan in the mid-late twenties. While no cure yet exists ... ...

    Abstract Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting 1/5000 boys globally, characterized by progressive muscle breakdown and eventual death, with an average lifespan in the mid-late twenties. While no cure yet exists for DMD, gene and antisense therapies have been heavily explored in recent years to better treat this disease. Four antisense therapies have received conditional FDA approval, and many more exist in varying stages of clinical trials. These upcoming therapies often utilize novel drug chemistries to address limitations of existing therapies, and their development could herald the next generation of antisense therapy. This review article aims to summarize the current state of development for antisense-based therapies for the treatment of Duchenne muscular dystrophy, exploring candidates designed for both exon skipping and gene knockdown.
    Language English
    Publishing date 2023-02-26
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2527217-2
    ISSN 1999-4923
    ISSN 1999-4923
    DOI 10.3390/pharmaceutics15030778
    Database MEDical Literature Analysis and Retrieval System OnLINE

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