Related Experiment Video

Updated: Jul 25, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
09:00

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

8.2K

FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections

Asher Mullard

    Nature Reviews. Drug Discovery
    |June 23, 2023
    PubMed
    Abstract

    No abstract available in PubMed .

    More Related Videos

    Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
    05:16

    Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

    Published on: May 7, 2020

    6.9K
    Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells
    06:20

    Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells

    Published on: May 7, 2020

    7.1K

    Related Experiment Videos

    Last Updated: Jul 25, 2025

    Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
    09:00

    Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

    Published on: August 2, 2018

    8.2K
    Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
    05:16

    Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

    Published on: May 7, 2020

    6.9K
    Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells
    06:20

    Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells

    Published on: May 7, 2020

    7.1K

    Related Concept Videos

    Satellite Stem Cells and Muscular Dystrophy01:21

    Satellite Stem Cells and Muscular Dystrophy

    2.0K
    Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
    2.0K
    What is Genetic Engineering?00:49

    What is Genetic Engineering?

    74.4K
    Overview
    74.4K

    Articles linked to this work by shared authors, journal, and citation graph.

    Towards precision psychiatry: initial foundations and future directions.

    Nature reviews. Drug discovery·2026

    Targeting CDKs in the RNAPII transcription cycle.

    Nature reviews. Drug discovery·2026

    Artificial intelligence in drug discovery - what it is, where we stand and the path forward.

    Nature reviews. Drug discovery·2026

    Targeting T follicular helper cells for lifelong health.

    Nature reviews. Drug discovery·2026

    Design, simulate, refine: simulation-guided clinical trials for accelerated drug development.

    Nature reviews. Drug discovery·2026

    Intranasal essential oils in therapy: advances in clinical applications and delivery mechanisms.

    Journal of traditional Chinese medicine = Chung i tsa chih ying wen pan·2026

    Immunobridging Analysis of Pemivibart for the Treatment of COVID-19: A Therapeutic Gap for the Immune-Compromised Population Remains.

    Open forum infectious diseases·2026

    Desferrioxamine attenuating high glucose-induced ferroptosis and inflammatory responses in Müller glial cells via Nrf2/TXNRD1 pathway.

    International journal of ophthalmology·2026

    Safety and effectiveness of Bian-stone and moxibustion in treatment of meibomian gland dysfunction.

    International journal of ophthalmology·2026

    Rezvilutamide combined with ADT in an HIV-positive patient with high-volume mHSPC on efavirenz-based antiretroviral therapy: a case report.

    Frontiers in oncology·2026

    A rhesus macaque model of α-dystroglycanopathy caused by a POMT1 splice altering variant.

    Animal models and experimental medicine·2026
    See all related articles
    JoVE
    x logofacebook logolinkedin logoyoutube logo
    ABOUT JoVE
    OverviewLeadershipBlogJoVE Help Center
    AUTHORS
    Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
    LIBRARIANS
    TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
    RESEARCH
    JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
    EDUCATION
    JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
    Terms & Conditions of Use
    Privacy Policy
    Policies
    Jove
    Visualize
    Contact Us