Related Experiment Video

Updated: Jul 27, 2025

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

Duchenne muscular dystrophy gene therapy approval rests on surrogate biomarker data

Thiago Carvalho

    Nature Medicine
    |June 5, 2023
    PubMed
    Abstract

    No abstract available in PubMed .

    More Related Videos

    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
    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

    Related Experiment Videos

    Last Updated: Jul 27, 2025

    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
    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

    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

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

    Barriers, Adoption, and Use of a Bike-Sharing System: A Market-Segment Approach to Current and Potential Users in Montréal, Canada.

    Transportation research record·2026

    Should Soccer Players Re-Warm-up at Half-Time? An Exploratory Randomized Controlled Trial in a Professional Women's Team.

    International journal of sports physiology and performance·2026

    Everything has changed: the impacts of the COVID-19 pandemic on the transit market in Montréal, Canada.

    Transportation·2026

    Activation-induced cytidine deaminase: The missing piece of many puzzles.

    Cell·2025

    Towards out-of-distribution detection using gradient vectors.

    Neural networks : the official journal of the International Neural Network Society·2025

    Warm-Up Strategies at Halftime: A Pilot Randomized Controlled Trial in a Professional Women's Soccer Team.

    Journal of functional morphology and kinesiology·2025

    Induced pluripotent stem cells from discovery to translation.

    Nature medicine·2026

    CD19 CAR T cell therapy for treatment-refractory seropositive rheumatoid arthritis: a phase 1 trial.

    Nature medicine·2026

    A multi-faceted hospital-based intervention for intrapartum care in sub-Saharan Africa: a stepped-wedge cluster-randomized trial.

    Nature medicine·2026

    Recombinant shingles vaccination and the risk of cardiovascular events.

    Nature medicine·2026

    Oligodendrocyte dysfunction in human age-related cognitive decline.

    Nature medicine·2026

    Citywide implementation of a rapid whole-genome sequencing program for critically ill pediatric patients.

    Nature medicine·2026

    Can Telehealth Improve Access to Amyloid-Targeting Therapies for Veterans Living With Alzheimer Disease?

    Federal practitioner : for the health care professionals of the VA, DoD, and PHS·2026

    Correction to: Sustained release microneedle patch for pronounced systemic delivery of doxazosin mesylate.

    BioImpacts : BI·2026

    A Nomogram Integrating Clinical and Cardiac Imaging for Predicting Short-Term Left Ventricular Ejection Fraction Decline in Patients with Duchenne Muscular Dystrophy.

    International journal of general medicine·2026

    Metformin modulates the phenotypes of tumor-associated macrophages in glioblastoma in a context-dependent manner by promoting the homeostasis of macrophages.

    Frontiers in oncology·2026

    OAF Blocks SIAH1-Mediated Degradation of SCPX, a Therapeutic Strategy for MASLD.

    Advanced science (Weinheim, Baden-Wurttemberg, Germany)·2026

    Real World Data and Its Impact on the Management of Plasma Cell Dyscrasias.

    Current hematologic malignancy reports·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