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  1. Article ; Online: Playing the genetic lottery: an interview with Kiran Musunuru.

    Musunuru, Kiran

    Disease models & mechanisms

    2023  Volume 16, Issue 10

    Language English
    Publishing date 2023-10-10
    Publishing country England
    Document type Interview
    ZDB-ID 2451104-3
    ISSN 1754-8411 ; 1754-8403
    ISSN (online) 1754-8411
    ISSN 1754-8403
    DOI 10.1242/dmm.050508
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Book ; Online ; E-Book: Genome editing

    Musunuru, Kiran

    a practical guide to research and clinical applications

    2021  

    Abstract: Genome Editing: A Practical Guide to Research and Clinical Applications is geared towards investigators interested in learning how to use CRISPR-Cas9-based technologies, with a focus on cardiovascular research and clinical applications. Covering a range ... ...

    Author's details Kiran Musunuru
    Abstract Genome Editing: A Practical Guide to Research and Clinical Applications is geared towards investigators interested in learning how to use CRISPR-Cas9-based technologies, with a focus on cardiovascular research and clinical applications. Covering a range of topics from the basics of genome editing to design considerations, to assessments and applications, this reference allows readers to get started and establish a full workflow from the beginning of the project to its full completion. With worked examples drawn from real-life experiments, as well as troubleshooting and pitfalls to avoid, the book serves as an essential reference for researchers and investigators in both cardiovascular and biomedical research.
    Keywords Gene editing/Methodology
    Subject code 572.86
    Language English
    Size 1 online resource :, illustrations (some colour)
    Publisher Academic Press
    Publishing place London, England
    Document type Book ; Online ; E-Book
    Remark Zugriff für angemeldete ZB MED-Nutzerinnen und -Nutzer
    ISBN 978-0-12-823647-5 ; 0-12-823647-7 ; 978-0-12-823484-6 ; 0-12-823484-9
    Database ZB MED Catalogue: Medicine, Health, Nutrition, Environment, Agriculture

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  3. Article ; Online: Playing the genetic lottery

    Kiran Musunuru

    Disease Models & Mechanisms, Vol 16, Iss

    an interview with Kiran Musunuru

    2023  Volume 10

    Keywords Medicine ; R ; Pathology ; RB1-214
    Language English
    Publishing date 2023-10-01T00:00:00Z
    Publisher The Company of Biologists
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

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  4. Article: An Overview of Genome Editing in Cardiovascular and Metabolic Diseases.

    Musunuru, Kiran

    Advances in experimental medicine and biology

    2022  Volume 1396, Page(s) 3–16

    Abstract: This chapter summarizes the definition, classification, and function of genome editing and highlights the breakthroughs of genome editing in cardiovascular and metabolic diseases for disease modeling, diagnostics, and therapeutics, with a particular ... ...

    Abstract This chapter summarizes the definition, classification, and function of genome editing and highlights the breakthroughs of genome editing in cardiovascular and metabolic diseases for disease modeling, diagnostics, and therapeutics, with a particular focus on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated 9 (Cas9) technology as applied to nuclease editing, base editing, and epigenome editing.
    MeSH term(s) Humans ; Gene Editing ; Cardiovascular System ; Metabolic Diseases/genetics ; Metabolic Diseases/therapy ; Endonucleases ; Epigenome
    Chemical Substances Endonucleases (EC 3.1.-)
    Language English
    Publishing date 2022-12-01
    Publishing country United States
    Document type Journal Article
    ISSN 2214-8019 ; 0065-2598
    ISSN (online) 2214-8019
    ISSN 0065-2598
    DOI 10.1007/978-981-19-5642-3_1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Moving toward genome-editing therapies for cardiovascular diseases.

    Musunuru, Kiran

    The Journal of clinical investigation

    2022  Volume 132, Issue 1

    Abstract: The rapid invention of genome-editing technologies over the past decade, which has already been transformative for biomedical research, has raised the tantalizing prospect of an entirely new therapeutic modality. Whereas the treatment of chronic ... ...

    Abstract The rapid invention of genome-editing technologies over the past decade, which has already been transformative for biomedical research, has raised the tantalizing prospect of an entirely new therapeutic modality. Whereas the treatment of chronic cardiovascular diseases has heretofore entailed the use of chronic therapies that typically must be taken repeatedly and frequently for the remainder of the lifetime, genome editing will enable the development of "one-and-done" therapies with durable effects. This Review summarizes the variety of available genome-editing approaches, including nuclease editing, base editing, epigenome editing, and prime editing; illustrates how these various approaches could be implemented as novel therapies for cardiovascular diseases; and outlines a path from technology development to preclinical studies to clinical trials. Although this Review focuses on PCSK9 as an instructive example of the various genome-editing approaches under active investigation, the lessons learned will be broadly applicable to the treatment of a variety of diseases.
    MeSH term(s) Animals ; Cardiovascular Diseases/genetics ; Cardiovascular Diseases/metabolism ; Cardiovascular Diseases/therapy ; Clinical Trials as Topic ; Epigenome ; Gene Editing ; Humans ; Proprotein Convertase 9/genetics ; Proprotein Convertase 9/metabolism
    Chemical Substances PCSK9 protein, human (EC 3.4.21.-) ; Proprotein Convertase 9 (EC 3.4.21.-)
    Language English
    Publishing date 2022-01-27
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Review
    ZDB-ID 3067-3
    ISSN 1558-8238 ; 0021-9738
    ISSN (online) 1558-8238
    ISSN 0021-9738
    DOI 10.1172/JCI148555
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Ua VI Zs.184: Show issues Location:
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    bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular
    ab Jg. 2022: Lesesaal (EG)

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  6. Article ; Online: CRISPR and cardiovascular diseases.

    Musunuru, Kiran

    Cardiovascular research

    2022  Volume 119, Issue 1, Page(s) 79–93

    Abstract: CRISPR technologies have progressed by leaps and bounds over the past decade, not only having a transformative effect on biomedical research but also yielding new therapies that are poised to enter the clinic. In this review, I give an overview of (i) ... ...

    Abstract CRISPR technologies have progressed by leaps and bounds over the past decade, not only having a transformative effect on biomedical research but also yielding new therapies that are poised to enter the clinic. In this review, I give an overview of (i) the various CRISPR DNA-editing technologies, including standard nuclease gene editing, base editing, prime editing, and epigenome editing, (ii) their impact on cardiovascular basic science research, including animal models, human pluripotent stem cell models, and functional screens, and (iii) emerging therapeutic applications for patients with cardiovascular diseases, focusing on the examples of hypercholesterolaemia, transthyretin amyloidosis, and Duchenne muscular dystrophy.
    MeSH term(s) Animals ; Humans ; Clustered Regularly Interspaced Short Palindromic Repeats ; Cardiovascular Diseases/diagnosis ; Cardiovascular Diseases/genetics ; Cardiovascular Diseases/therapy ; Genetic Therapy ; Gene Editing ; Muscular Dystrophy, Duchenne/genetics ; CRISPR-Cas Systems
    Language English
    Publishing date 2022-04-06
    Publishing country England
    Document type Review ; Journal Article
    ZDB-ID 80340-6
    ISSN 1755-3245 ; 0008-6363
    ISSN (online) 1755-3245
    ISSN 0008-6363
    DOI 10.1093/cvr/cvac048
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 565: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
    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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  7. Article ; Online: Moving toward genome-editing therapies for cardiovascular diseases

    Kiran Musunuru

    The Journal of Clinical Investigation, Vol 132, Iss

    2022  Volume 1

    Abstract: The rapid invention of genome-editing technologies over the past decade, which has already been transformative for biomedical research, has raised the tantalizing prospect of an entirely new therapeutic modality. Whereas the treatment of chronic ... ...

    Abstract The rapid invention of genome-editing technologies over the past decade, which has already been transformative for biomedical research, has raised the tantalizing prospect of an entirely new therapeutic modality. Whereas the treatment of chronic cardiovascular diseases has heretofore entailed the use of chronic therapies that typically must be taken repeatedly and frequently for the remainder of the lifetime, genome editing will enable the development of “one-and-done” therapies with durable effects. This Review summarizes the variety of available genome-editing approaches, including nuclease editing, base editing, epigenome editing, and prime editing; illustrates how these various approaches could be implemented as novel therapies for cardiovascular diseases; and outlines a path from technology development to preclinical studies to clinical trials. Although this Review focuses on PCSK9 as an instructive example of the various genome-editing approaches under active investigation, the lessons learned will be broadly applicable to the treatment of a variety of diseases.
    Keywords Medicine ; R
    Language English
    Publishing date 2022-01-01T00:00:00Z
    Publisher American Society for Clinical Investigation
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

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  8. Article: Adenine base editing to treat progeria syndrome and extend the lifespan.

    Musunuru, Kiran

    The journal of cardiovascular aging

    2021  Volume 1

    Abstract: Hutchinson-Gilford progeria syndrome (HGPS) is an exceedingly rare and hitherto incurable and fatal disease marked by accelerated aging simultaneously affecting a number of organs. Most cases of HGPS are caused by a single copy of a specific single- ... ...

    Abstract Hutchinson-Gilford progeria syndrome (HGPS) is an exceedingly rare and hitherto incurable and fatal disease marked by accelerated aging simultaneously affecting a number of organs. Most cases of HGPS are caused by a single copy of a specific single-nucleotide mutation, c.C1824T, in the
    Language English
    Publishing date 2021-06-17
    Document type Journal Article
    DOI 10.20517/jca.2021.10
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article ; Online: CRISPR Hits Home in a First-in-Human Study.

    Musunuru, Kiran

    The CRISPR journal

    2021  Volume 4, Issue 4, Page(s) 460–461

    MeSH term(s) Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/therapy ; Animals ; CRISPR-Cas Systems ; Clinical Trials as Topic ; Clustered Regularly Interspaced Short Palindromic Repeats ; Gene Editing ; Genetic Therapy/methods ; Humans ; Treatment Outcome
    Language English
    Publishing date 2021-08-18
    Publishing country United States
    Document type Letter
    ZDB-ID 3017891-5
    ISSN 2573-1602 ; 2573-1599
    ISSN (online) 2573-1602
    ISSN 2573-1599
    DOI 10.1089/crispr.2021.29131.mus
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 7195: 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)
    Zs.MG 119: Show issues

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  10. Article ; Online: Correcting tyrosinaemia via a point mutation.

    Musunuru, Kiran

    Nature biomedical engineering

    2020  Volume 4, Issue 1, Page(s) 14–15

    MeSH term(s) Adenine ; Animals ; Mice ; Point Mutation ; Tyrosinemias
    Chemical Substances Adenine (JAC85A2161)
    Language English
    Publishing date 2020-01-14
    Publishing country England
    Document type Journal Article ; Comment
    ISSN 2157-846X
    ISSN (online) 2157-846X
    DOI 10.1038/s41551-019-0489-x
    Database MEDical Literature Analysis and Retrieval System OnLINE

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