Related Experiment Video

Updated: Aug 9, 2025

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
09:37

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development

Published on: March 5, 2017

13.2K

Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease

Alex Philippidis

    Human Gene Therapy
    |February 16, 2023
    PubMed
    Summary

    No abstract available in PubMed .

    More Related Videos

    CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
    08:32

    CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

    Published on: August 9, 2022

    3.7K
    Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
    07:31

    Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

    Published on: February 17, 2023

    1.2K

    Related Experiment Videos

    Last Updated: Aug 9, 2025

    Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
    09:37

    Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development

    Published on: March 5, 2017

    13.2K
    CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
    08:32

    CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

    Published on: August 9, 2022

    3.7K
    Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
    07:31

    Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

    Published on: February 17, 2023

    1.2K

    Related Concept Videos

    CRISPR01:59

    CRISPR

    52.6K
    Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
    52.6K
    What is Genetic Engineering?00:49

    What is Genetic Engineering?

    74.5K
    Overview
    74.5K
    iPS Cell Differentiation01:22

    iPS Cell Differentiation

    2.8K
    The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
    2.8K

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

    Five Years of Progress in CRISPR Clinical Trials (2019-2024).

    The CRISPR journal·2024

    Dichotomies in Ex Vivo and In Vivo Performance of Receptor-Binding Mutants of Adeno-Associated Virus Vectors.

    Human gene therapy·2026

    Celyvir: Oncolytic Virotherapy with Mesenchymal Stromal Cells. From Concept to Clinical Translation.

    Human gene therapy·2026

    CAR-T Cell Therapy: Manufacturing Platforms and Clinical Consequences.

    Human gene therapy·2026

    AAV.hBAG3 Gene Therapy Improves Phenotype in a Valosin Containing Protein Mouse Model of Hereditary Inclusion Body Myositis.

    Human gene therapy·2026

    Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing BEST1 Delivered by Subretinal Injection in a Canine Model of Human Bestrophinopathy.

    Human gene therapy·2026

    The German National Strategy for Gene- and Cell-Based Therapies: Generating Impact by Employing a Novel Multi-Stakeholder Approach.

    Human gene therapy·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