The Role of Nemo like Kinase in the Pathogenesis and Treatment of Diamond Blackfan Anemia

Abstract

Diamond Blackfan Anemia (DBA) is a genetic disorder that results in pure red cell aplasia, congenital abnormalities, and predisposition to cancer. The current treatment with steroids, chronic transfusions, and stem cell transplantation leads to significant morbidity and even mortality. Approximately 25% of patients with DBA have mutations in RPS19. We identified Nemo-Like Kinase (NLK) as being hyperactive in RPS19-insufficient human cord blood CD34+hematopoietic stem cells. Inhibition of NLK by knockdown or small molecules rescues the erythroid defect in RPS19-insufficient HPSCs.

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Document Details

Document Type
Technical Report
Publication Date
Aug 01, 2021
Accession Number
AD1150353

Entities

People

  • Kathleen Sakamoto

Organizations

  • Stanford University

Tags

DTIC Thesaurus Topics

  • Antineoplastic Agents
  • Blood
  • Bone Marrow Cells
  • Cell Physiological Processes
  • Cells
  • Chemical Synthesis
  • Chemistry
  • Colon Cancer
  • Gene Expression
  • Genetics
  • Health Services
  • Medical Personnel
  • Myeloid Cells
  • Oncology
  • Peptide Growth Factors
  • Proteins
  • Stem Cells

Fields of Study

  • Medicine

Readers

  • Immunology and Pathology
  • Molecular and Cellular Biology

Technology Areas

  • Biotechnology