Oligonucleotide therapeutics for hematologic disorders

Biochim Biophys Acta. 1999 Dec 10;1489(1):85-96. doi: 10.1016/s0167-4781(99)00142-6.


During the last decade, the catalogue of known genes responsible for cell growth, development, and neoplastic transformation has expanded dramatically. Attempts to translate this information into new therapeutic strategies for both hematologic and non-hematologic diseases have accelerated at a rapid pace as well. Inserting genes into cells which either replace, or counter the effects of disease causing genes has been one of the primary ways in which scientists have tried to exploit this new knowledge. Strategies to directly downregulate gene expression have developed in parallel with this approach. The latter include triple helix forming oligonucleotides (ODN) and 'antisense' ODN. The latter have already entered clinical trials for a variety of disorders. In this monograph, we review the use of these materials in the treatment of hematologic diseases, particularly myelogenous leukemias. Problems and possible solutions associated with the use of ODN will be discussed as well.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Blood Coagulation Disorders / drug therapy
  • Fusion Proteins, bcr-abl / antagonists & inhibitors
  • Fusion Proteins, bcr-abl / genetics
  • Hematologic Diseases / drug therapy*
  • Humans
  • Leukemia, Experimental / drug therapy
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy
  • Leukemia, Myeloid, Acute / drug therapy
  • Lymphoma, Non-Hodgkin / drug therapy
  • Mice
  • Mice, SCID
  • Oligonucleotides / therapeutic use*
  • Oligonucleotides, Antisense / therapeutic use*
  • Proto-Oncogene Proteins c-bcl-2 / antagonists & inhibitors
  • Proto-Oncogene Proteins c-bcl-2 / genetics
  • Proto-Oncogene Proteins c-myb / antagonists & inhibitors
  • Proto-Oncogene Proteins c-myb / genetics
  • Proto-Oncogene Proteins c-myc / antagonists & inhibitors
  • Proto-Oncogene Proteins c-myc / genetics
  • RNA, Messenger / antagonists & inhibitors
  • Signal Transduction / drug effects
  • Tumor Suppressor Protein p53 / antagonists & inhibitors
  • Tumor Suppressor Protein p53 / genetics


  • Oligonucleotides
  • Oligonucleotides, Antisense
  • Proto-Oncogene Proteins c-bcl-2
  • Proto-Oncogene Proteins c-myb
  • Proto-Oncogene Proteins c-myc
  • RNA, Messenger
  • Tumor Suppressor Protein p53
  • Fusion Proteins, bcr-abl