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Hemophilia CRISPR Gene Editing

Challenges and opportunities when transitioning
from in vivo gene replacement to in vivo CRISPR/
Cas9 therapies – a spotlight on hemophilia

June 28, 2022June 21, 2022 by admin

In vivo gene editing in liver tissues may be able to rescue patients younger than 18 years who are not eligible for gene replacement therapies, with hemophilia as a prime example

Categories Hemophilia CRISPR Gene Editing, Publications Leave a comment

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

February 18, 2022November 14, 2019 by admin
Categories Hemophilia CRISPR Gene Editing, Publications Leave a comment

Recent Posts

  • Director, Gene and Cell Therapy
  • Challenges and opportunities when transitioning
    from in vivo gene replacement to in vivo CRISPR/
    Cas9 therapies – a spotlight on hemophilia
  • New Agreement Set to Scale Up Production of Hem A Gene Therapy
  • Charles River and ASC Therapeutics to Scale Manufacturing of Second-Generation Gene Therapy for Hemophilia A
  • ASC Therapeutics Joins U.S. Consortium of Gene Therapy Partners Including the NIH and FDA to Accelerate Treatments for Rare Diseases

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