       Document 0164
 DOCN  M9580164
 TI    The ENCEL system: a somatic cell protein delivery system.
 DT    9506
 AU    Squinto SP; Madri JA; Kennedy S; Springhorn J; Alexion Pharmaceuticals,
       Inc, New Haven, CT 06511, USA.
 SO    In Vivo. 1994 Nov-Dec;8(5):771-80. Unique Identifier : AIDSLINE
       MED/95244808
 AB    A wide variety of somatic cells are being explored for the introduction
       of foreign genes with a view toward gene therapy. A prime requirement
       for successful gene therapy is the sustained expression, effective
       dosing, and systemic delivery of the therapeutic gene product.
       Microvascular endothelial cells offer several advantages over other cell
       types as a somatic cell gene delivery vehicle in that they provide
       direct secretion of protein into the blood stream and they are amendable
       to highly stable retroviral-based protein expression. Importantly, they
       also offer a large surface volume to size ratio in that they can be
       induced with angiogenic factors to form organized capillary-like
       structures in vitro when grown in a three dimensional culture system
       using collagen gels. These genetically-modified capillary endothelial
       cells (the ENCEL system) maintained in collagen gels can be stably
       transplanted and removed. The unique biological properties of
       microvascular capillary endothelial cells allows the ENCEL system to
       provide large numbers of cells in a small volume which offers the highly
       desired opportunity for providing a sustained and effective dose of a
       therapeutic protein. Alexion is currently applying its Unigraft
       immunotherapeutic and engineering technologies to commercialize a
       non-human ENCEL system acceptable for implantation into any patient.
 DE    Acquired Immunodeficiency Syndrome/THERAPY  Animal  Cytomegalovirus
       Infections/THERAPY  *Drug Carriers  Gene Therapy/*METHODS  Hereditary
       Diseases/*THERAPY  Human  Immunologic Deficiency Syndromes/*THERAPY
       Neoplasms/*THERAPY  Proteins/*ADMINISTRATION & DOSAGE
       Vaccines/ADMINISTRATION & DOSAGE  JOURNAL ARTICLE  REVIEW  REVIEW,
       TUTORIAL

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

