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A base editing approach for Shwachman-Diamond-Syndrome
A base editing approach for Shwachman-Diamond-Syndrome
Background
Shwachman-Diamond-Syndrome (SDS), is life threatening genetic condition caused in 90% of cases by a mutation in the SBDS-gene, which leads to defective splicing and loss of protein expression. Patients may develop many symptoms such as frequent infections due to low white blood cell...
Published: 3/26/2026
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Updated: 12/8/2025
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Inventor(s): Christian Brendel
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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