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Gene Therapy: Some History, Applications, Problems, and Prospects

From the Gene Therapy and Therapeutics Branch, National Institute of Dental and Craniofacial Research, NIH, DHHS, Bethesda, Maryland, USA

Correspondence: Address correspondence to: Dr. Ana Cotrim, GTTB, NIDCR, NIH, 10 Center Drive, Bethesda, MD 20892-1190, e-mail: acotrim{at}nidcr.nih.gov.

The concept of transferring genes to tissues for clinical applications has been discussed for nearly half a century, but our ability to manipulate genetic material via recombinant DNA technology has brought this goal to reality. While originally conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, gene therapy now is considered for many non–life-threatening conditions, including those adversely affecting a patient’s quality of life. The lack of suitable treatment has become a rational basis for extending the scope of gene therapy. This manuscript reviews the general methods by which genes are transferred as well as diverse examples of clinical applications (acquired tissue damage, upper gastrointestinal tract infection, autoimmune disease, systemic protein deficiency). Despite some well-publicized problems, gene therapy has made substantive progress, including tangible success, albeit much slower than was initially predicted. Although gene therapy is still at a fairly primitive stage, it is firmly science based. There is justifiable optimism that with increased pathobiological understanding and biotechnological improvements, gene therapy will become a standard part of clinical practice within 20 years.

Key Words: Salivary gland • animal models • cell(ular) pathology

Abbreviations: Ad5, serotype 5 adenovirus • MoMLV, Moloney murine leukemia virus • SG, salivary gland • GI, gastrointestinal • AAV2, serotype 2 adeno-associated virus • IR, irradiation • MnSOD-PL, manganese superoxide dismutase-plasmid liposome • AQP1, aquaporin-1 • AdhAQP1, Ad5 vector encoding human AQP1 • SS, Sjogren’s syndrome • VIP, vasoactive intestinal peptide • IL-10, interleukin-10 • NOD, nonobese diabetic • h, human • Epo, erythropoietin • FDA, Food and Drug Administration • NIH, National Institutes of Health • SCID, severe combined immunodeficiency disorder

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