title: Exploiting genetic alterations to design novel therapies for cancer.
abstract: In the 3 decades since the signing of the National Cancer Act, there has been tremendous progress in the elucidation of the molecular underpinnings of cancer. Molecular genetic studies have been particularly insightful, revealing genetic rearrangements, such as chromosomal translocations, which may be the seminal event leading to deregulated cell growth for many childhood and adult cancers. These findings have led to new diagnostic and prognostic tools but have been slow to be translated into new therapeutic modalities. This article reviews a variety of methods now under development to exploit genetic changes in cancer to develop specific anticancer agents using gene therapy, viral therapy, and immunotherapy approaches. As many of these strategies inevitably enter the clinic, it will be imperative for health care professionals to be familiar with these novel approaches as they help patients navigate the likely broad array of treatment options.
authors.affiliation: Department of Pediatrics, Division of Hematology/Oncology, Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA. t.cripe@chmcc.org
authors.forename: T P
authors.initials: TP
authors.lastname: Cripe
authors.forename: C L
authors.initials: CL
authors.lastname: Mackall
mesh_terms: D000260:Adenoviruses, Human / Q000235:genetics / Q000502:physiology; D000818:Animals; D017951:Antigen Presentation; D002648:Child; D002869:Chromosome Aberrations; D002986:Clinical Trials as Topic; D003673:Defective Viruses / Q000235:genetics / Q000502:physiology; D015195:Drug Design; D005544:Forecasting; D017353:Gene Deletion; D015972:Gene Expression Regulation, Neoplastic / Q000187:drug effects; D016147:Genes, Tumor Suppressor; D015316:Genetic Therapy; D006801:Humans; D007108:Immune Tolerance; D007167:Immunotherapy; D051379:Mice; D023421:Models, Animal; D018716:Molecular Mimicry; D009363:Neoplasm Proteins / Q000172:deficiency / Q000235:genetics; D009369:Neoplasms / Q000235:genetics / Q000628:therapy*; D009690:Nucleic Acid Conformation; D009838:Oligodeoxyribonucleotides / Q000737:chemistry / Q000627:therapeutic use; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use; D009857:Oncogenes; D010446:Peptide Fragments / Q000276:immunology / Q000627:therapeutic use; D016337:RNA, Catalytic / Q000627:therapeutic use; D011993:Recombinant Fusion Proteins / Q000235:genetics / Q000502:physiology; D016176:T-Lymphocyte Subsets / Q000276:immunology; D014157:Transcription Factors / Q000235:genetics / Q000502:physiology
chemical_list: D009363:Neoplasm Proteins; D009838:Oligodeoxyribonucleotides; D016376:Oligonucleotides, Antisense; D010446:Peptide Fragments; D016337:RNA, Catalytic; D011993:Recombinant Fusion Proteins; D014157:Transcription Factors