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Advancing Hemoglobinopathy Care: Innovative BCL11A Inhibition for Sustained Fetal Hemoglobin Production
Advancing Hemoglobinopathy Care: Innovative BCL11A Inhibition for Sustained Fetal Hemoglobin Production
Background
Hemoglobinopathies, including sickle cell anemia and β-thalassemias, are genetic disorders caused by mutations that impair the production or structure of of β-globin chains in hemoglobin. These conditions result in patients...
Published: 3/25/2026
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Updated: 12/5/2025
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Inventor(s): Daniel Bauer, Yuxuan Wu, Jing Zeng, Scot Wolfe, Mehmet Bolukbasi, Benjamin Roscoe, Pengpeng Liu, Luk.Kevin
Keywords(s): Sickle Cell Anemia
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Enhanced BCL11A RNP / CRISPR delivery & editing using a 3xNLS-Cas9
Advancing Gene Therapy for Hemoglobinopathies: Enhanced CRISPR-Cas9 with Multi-NLS Integration
Background
Sickle cell disease (SCD) and β-thalassemia are severe β-globin disorders affecting millions globally, characterized by defective adult hemoglobin that fails to transport oxygen effectively, leading to significant morbidity. Existing...
Published: 3/25/2026
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Updated: 10/13/2025
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Inventor(s): Daniel Bauer, Pengpeng Liu, Scot Wolfe, Yuxuan Wu, Jing Zeng
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Temp: Repair of ELANE therapeutic gene editing for congenital neutropenia
Therapeutic Gene Editing for Severe Congenital Neutropenia
Background
Severe congenital neutropenia (SCN) is a rare inherited disorder characterized by low absolute neutrophil counts which increases the risk of life-threatening infections. Occurrence of SCN is attributed to changes in various genes, but an estimated 50% of severe congenital...
Published: 3/26/2026
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Updated: 4/11/2024
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Inventor(s): Daniel Bauer, Scot Wolfe, Peter Newburger, Josias Frazão, Shuquan Rao
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Rare Diseases
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