       Document 0356
 DOCN  M9620356
 TI    Gene therapy against retroviral diseases.
 DT    9602
 AU    Essex M; Matsuda Z; Yu X; Lee TH; Department of Cancer Biology, Harvard
       School of Public Health,; Boston, Massachusetts 02115, USA.
 SO    Leukemia. 1995 Oct;9 Suppl 1:S71-4. Unique Identifier : AIDSLINE
       MED/96022225
 AB    Eventually, gene therapy may be a valid option for chronic viral
       infections, including retroviral infections. Human retroviral diseases
       fit two categories: (1) those that result from a monoclonal outgrowth of
       a human T-cell leukemia virus type I (HTLV-I)-infected cell, as in the
       case of adult T cell leukemia (ATL); and (2) those that appear to result
       directly from virus load rather than monoclonal outgrowth--such as
       tropical spastic paraparesis/HTLV-I associated myelopathy (TSP/HAM) and
       human immunodeficiency virus (HIV)-associated acquired immune deficiency
       syndrome (AIDS). For ATL gene therapy, corrective mechanisms directed at
       regulatory sequences rather than viral sequences may be most important,
       though perhaps anti-tax therapy would be useful. For TSP/HAM and AIDS,
       gene therapy directed to control virus replication may be most useful.
       For anti-retroviral therapy, one may use dominant negative mutants and a
       variety of other approaches that direct toxins or compete out viral
       regulatory gene signal sequences. For maximum benefit, such therapy
       should be directed to different essential genes (eg gag, pol, env, tat
       or rev) involved in the virus replication cycle and utilize different
       toxic approaches. A major impediment to the use of gene therapy for AIDS
       is our inability to transfect a significant fraction of target cells in
       vivo. Except for reconstituted mice, retroviral systems of animals have
       been under-utilized as models for gene therapy. Naturally occurring
       retroviral diseases of cats, goats, horses, and other species provide
       models for future development.
 DE    Acquired Immunodeficiency Syndrome/*THERAPY  Adult  Animal  Cats  Gene
       Deletion  *Gene Therapy  Genes, Viral  Goats  Horses  Human  HIV  HTLV-I
       Infections/*THERAPY  Leukemia, T-Cell, Acute/*THERAPY  Mice  Regulatory
       Sequences, Nucleic Acid  Retroviridae/*GENETICS/PATHOGENICITY  Support,
       U.S. Gov't, P.H.S.  JOURNAL ARTICLE  REVIEW  REVIEW, TUTORIAL

       SOURCE: National Library of Medicine.  NOTICE: This material may be
       protected by Copyright Law (Title 17, U.S.Code).

