natal retroviral vector mediated gene therapy for mucopolysaccharidosis vii in dogs results in stable hepatocyte transduction and marked improvement in bone and joint cardiac and corneal manifestations for up to 14

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927. Adeno-Associated Virus Display: A Combinatorial
Library for the Generation of Retargeted V
ectors
Vectors
Luca Perabo,1 Hildegard Buning,1 Anne Girod,1 Martin Ried,1 David
Kofler,2 Jorg Enssle,1 Michael Hallek.1,2,3
1
Gene Center, LMU-Munich, Munich, Germany; 2GSF, National
Research Center for Environment and Health, Munich, Germany;
3
Medical Department III, Klinikum Grosshadern, Munich,
Germany.
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928. Efficient Site-Specific Genomic Integration of hα-1-A
T and hFIX in Mice
-1-AT
Eric C. Olivares, Roger P. Hollis, Leonard Meuse, Mark Kay,
Michele P. Calos.
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Molecular Therapy Vol. 5, No. 5, May 2002, Part 2 of 2 Parts
Copyright © The American Society of Gene Therapy

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929. Neonatal Retroviral V
ector-Mediated Gene
Vector-Mediated
Therapy for Mucopolysaccharidosis VII in Dogs Results
in Stable Hepatocyte T
ransduction and Marked
Transduction
Improvement in Bone and Joint, Cardiac, and Corneal
Manifestations for up to 14 Months
Mark Haskins,1 John Melniczek,1 Lingfei Xu,2 Margaret Weil,1
Thomas O′Malley,1 Patricia O′Donnell,1 Rafe Knox,1 Gustavo
Aguirre,3 Meg Sleeper,1 Matthew Ellinwood,1 Susan Volk,1 Marina
D′Angelo,4 Hamutal Mazrier,1 Robert Mango,2 Albert Maguire,5 Jean
Zweigle,1 John Wolfe,1,6 Katherine P. Ponder.2
1
School of Veterinary Medicine, University of Pennsylvania,
Philadelphia, PA, United States; 2Departments of Internal
Medicine and Biochemistry and Molecular Biophysics,
Washington University School of Medicine, St. Louis, MO, United

States; 3College of Veterinary Medicine, Cornell University,
Ithaca, NY, United States; 4Anatomy, Philadelphia College of
Osteopathic Medicine, Philadelphia, PA, United States; 5Scheie
Eye Institute, University of Pennsylvania, Philadelphia, PA, United
States; 6Children′s Hospital of Philadelphia, Philadelphia, PA,
United States.
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930. In Vivo Functional Correction of Hemophilia a
Mice Via a Novel Spliceosome-Mediated RNA TransSplicing Repair Mechanism
Hengjun Chao,1 S. Gary Mansfield,2 Lloyd G. Mitchell,2 Mariano A.
Garcia-Blanco,2 Christopher E. Walsh.1
1
Gene Therapy Center, Dept. of Medicine, UNC, Chapel Hill, NC,
United States; 2Intronn Inc., Raleigh, NC, United States.
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931. Correction of ADA-SCID by Stem Cell Gene
Therapy Combined with a Non-Myeloablative
Conditioning
Alessandro Aiuti,1 Shimon Slavin,2 Memet Aker,2 Francesca
Ficara,1 Sara Deola,1 Alessandra Mortellaro,1 Grazia Andolfi,1
Shoshana Morecki,2 Antonella Tabucchi,3 Filippo Carlucci,3 Enrico
Marinello,3 Federica Cattaneo,1 Sergio Vai,1 Paolo Servida,4 Roberto
Miniero,5 Maria Grazia Roncarolo§,1 Claudio Bordignon§.1,4
1
San Raffaele - Telethon Institute for Gene Therapy, Scientific
Institute HS Raffaele, Milan, Italy; 2BM Transplantation and
Pediatrics, Hadassah University Hospital, Jerusalem, Israel;
3
Inst. of Biochemistry and Enzimology, University of Siena, Siena,
Italy; 4BMT and Gene Therapy Program, Scientific Institute HS
Raffaele, Milan, Italy; 5Dep. of Biological and Clinical Sciences,
University of Turin, Turin, Italy.
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Molecular Therapy Vol. 5, No. 5, May 2002, Part 2 of 2 Parts
Copyright © The American Society of Gene Therapy