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Hematopoietic Stem Cell Transplantation: Enhanced Engraftment without Chemotherapy for Genetic and Acquired Disorders
Hematopoietic Stem Cell Transplantation: Enhanced Engraftment without Chemotherapy for Genetic and Acquired Disorders
Background
There is a crucial unmet medical need for improved methods to treat inherited and acquired diseases via hematopoietic stem cell transplantation. Currently, patients must undergo high-dose alkylating chemotherapy as part...
Published: 3/26/2026
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Updated: 10/9/2025
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Inventor(s): Daniel Bauer, Vivien Schoonenberg, Sabine Studer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Cell Therapy, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area > Hematology
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Therapeutic twin prime editing of hematopoietic stem cells for TINF2 dyskeratosis congenita
Therapeutic twin prime editing of hematopoietic stem cells for TINF2 dyskeratosis congenita
Background
Dyskeratosis congenita (DC) is a rare, genetic form of bone marrow (BM) failure. It is estimated that one in one million people has this condition. About 50% of the people who have DC have changes in the genes that are important in telomere maintenance,...
Published: 3/26/2026
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Updated: 11/20/2024
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Inventor(s): Daniel Bauer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Improving gene editing by modulating nucleotide metabolism
Improving gene editing by modulating nucleotide metabolism
Background
Currently, most treatments for genetic blood-related disorders (both non-cancerous and cancerous) aim to alleviate symptoms, manage complications, and slow disease progression, rather than completely correcting the underlying genetic defect. In some cases, patients may qualify...
Published: 3/26/2026
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Updated: 11/20/2024
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Inventor(s): Daniel Bauer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Editing, Technology Classifications > Disease Area > Rare Diseases
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