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Updated: Aug 9, 2026

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells
09:16

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells

Published on: September 1, 2019

Molecular therapeutic approaches to acute myeloid leukemia: targeting aberrant chromatin dynamics and signal

Francesco Piazza1, Gianpietro Semenzato

  • 1Padova University School of Medicine, Venetian Institute of Molecular Medicine, Unit of Hematological Malignancies, via Orus 2 35129 Padova, Italy. francesco.piazza@unipd.it

Expert Review of Anticancer Therapy
|May 27, 2004
PubMed

Insights

Molecular biology advances have identified key targets in acute myeloid leukemia (AML), including aberrant transcription factors and signaling pathways. New drugs targeting these mechanisms offer potential therapeutic benefits for AML patients.

Area of Science:

  • Hematology
  • Molecular Biology
  • Oncology

Background:

  • Molecular biology has advanced acute myeloid leukemia (AML) research by identifying key pathogenetic mechanisms.
  • Aberrant transcription/chromatin-remodeling factors and deregulated signal transduction pathways are crucial in leukemic cell development and therapy resistance.

Purpose of the Study:

  • To highlight the role of molecular alterations in AML pathogenesis and therapy.
  • To discuss the potential of novel molecularly targeted drugs for AML treatment.

Main Methods:

  • Review of molecular biology findings in AML.
  • Identification of key molecular lesions and their pathogenetic roles.
  • Analysis of therapeutic targets and emerging drug classes.

Main Results:

  • Specific molecular alterations, including transcription factor activity and signaling pathways, are pivotal in AML.
  • These molecular lesions are validated therapeutic targets in certain leukemias and increasingly in AML.
  • Novel drugs like HDAC inhibitors, demethylating agents, and TKIs show promise.

Conclusions:

  • Understanding molecular mechanisms in AML is critical for effective clinical management.
  • Targeting aberrant molecular pathways offers a promising strategy for novel AML therapies.
  • Emerging targeted therapies have the potential to significantly benefit AML patients.

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