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Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome

  • Post author:
  • Post published:May 20, 2025
  • Post category:

Research Objective We are seeking to develop site-specific hematopoietic stem cell gene therapy with autologous transplant as a definitive treatment option for X-linked Hyper-IgM Syndrome. Impact These studies would bring…

Continue ReadingTargeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome

Ex Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome

  • Post author:
  • Post published:May 20, 2025
  • Post category:

Translational Candidate Human hematopoietic stem cells that have been gene-corrected at the CD40L gene to treat patients with X-Linked Hyper-IgM Syndrome Area of Impact These studies will bring stem cell…

Continue ReadingEx Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome
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