Blood Disorders – Page 6

Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome

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Post published:July 3, 2025
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Translational Candidate Human hematopoietic stem cells that have been modified to express a functional WAS gene to treat patients with Wiskott Aldrich Syndrome (WAS) Area of Impact These studies will…

Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome

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Post published:July 3, 2025
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Translational Candidate Human hematopoietic stem cells that have been modified to express a functional FOXP3 gene to treat patients with IPEX Syndrome Area of Impact These studies will bring stem…

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

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Post published:July 3, 2025
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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…

Curing Sickle cell Disease with CRISPR-Cas9 genome editing

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Post published:July 3, 2025
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Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The…

Curing Sickle cell Disease with CRISPR-Cas9 genome editing

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Post published:July 3, 2025
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Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The…