Related Experiment Video

Updated: Sep 12, 2025

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
07:44

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

8.5K

Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: The Ethics and Financial Toxicity of Exuberant Hope

A Gordon Smith

    Neurology
    |August 8, 2025
    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
    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: Sep 12, 2025

    CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
    07:44

    CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

    Published on: September 14, 2019

    8.5K
    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
    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
    What is Genetic Engineering?00:49

    What is Genetic Engineering?

    75.5K
    Overview
    75.5K

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

    Biosimilar Therapeutics Substitution: American Academy of Neurology Position Statement.

    Neurology. Clinical practice·2026

    Efgartigimod and Ravulizumab for Treating Acetylcholine Receptor Auto-antibody-Positive (AChR-Ab+) Generalized Myasthenia Gravis: Indirect Treatment Comparison.

    Advances in therapy·2024

    Association of Skin Denervation With Brain Atrophy and Cognitive Impairment in Patients With Parkinson Disease.

    Neurology·2026

    The Mechanical Penumbra in Intracerebral Hemorrhage.

    Neurology·2026

    Trends in Withdrawal of Life-Sustaining Treatment in Patients With Traumatic Brain Injury, 2013-2023.

    Neurology·2026

    Traumatic Spinal Cord Injury Mortality in the United States, 1999-2024: A Population-Based Analysis of Temporal and Demographic Trends.

    Neurology·2026

    Clinical Factors Associated With Discordant Plasma P-Tau217 and Established Alzheimer Disease Biomarkers.

    Neurology·2026

    Clinical Reasoning: A 13-Year-Old Boy With Acute-Onset Generalized Dystonia, Rostrocaudal Gradient, and Severe Bulbar Dysfunction.

    Neurology·2026

    Molecular epidemiology and pathological burden of Mycobacterium avium subsp. paratuberculosis in staple livestock from Pakistan: implications for zoonotic surveillance and livestock productivity.

    Microbiology spectrum·2026

    From misdiagnosis to precision management: Thoracic vertebral infection caused by Scedosporium boydii diagnosed using a multi-platform approach - A case report and literature review.

    Medical mycology case reports·2026

    Bioactive Constituents of Melipona quadrifasciata anthidioides Geopropolis with Antifungal Activity against Sporothrix brasiliensis.

    ACS omega·2026

    Incidental intestinal parasitic infections detected during colonoscopy: A case series of hookworm in a Cambodian immigrant and whipworm in Korean patients.

    Parasites, hosts and diseases·2026

    Cerebral Schistosoma japonicum infection confirmed by histopathology and PCR: Diagnostic and therapeutic challenges in multiple parasitic coinfections.

    Parasitology international·2026

    Skeletal muscle-specific markers for paraspinal injury in full-endoscopic versus open microscopic lumbar spine surgery: a prospective observational study.

    Neurosurgical focus·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