RNA targeted therapeutics for hematologic malignancies.
In a variety of experimental systems, antisense nucleic acids (ASNA) of various composition, including antisense oligodeoxynucleotides (ODN) and siRNA, have been shown to have the ability to variably perturb gene expression in a sequence specific manner. Pilot clinical studies from our group, and others, have demonstrated that gene silencing is a therapeutic strategy that is starting to make a real contribution to the treatment of various diseases. The development of this field, with specific reference to hematologic malignancies, is reviewed very briefly below.
Gewirtz
Alan M
AM
Division of Hematology/Oncology, Department of Medicine and Abramson Family Cancer Research Institute, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA. gewirtz@mail.med.upenn.edu
D020868:Gene Silencing; D019337:Hematologic Neoplasms / Q000628:therapy*; D006801:Humans; D020319:Oligodeoxyribonucleotides, Antisense / Q000627:therapeutic use; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use; D034741:RNA, Small Interfering / Q000627:therapeutic use
D020319:Oligodeoxyribonucleotides, Antisense; D016376:Oligonucleotides, Antisense; D034741:RNA, Small Interfering