Molecular Therapy Vol.. Adeno-Associated Virus Display: A Combinatorial Library for the Generation of Retargeted V Library for the Generation of Retargeted Vectors ectors Luca Perabo,1 H
Trang 1Molecular Therapy Vol 5, No 5, May 2002, Part 2 of 2 Parts
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927 Adeno-Associated Virus Display: A Combinatorial
Library for the Generation of Retargeted V
Library for the Generation of Retargeted Vectors ectors
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; 2 GSF, National
Research Center for Environment and Health, Munich, Germany;
3 Medical Department III, Klinikum Grosshadern, Munich,
Germany
89:
928 Efficient Site-Specific Genomic Integration of
h-α
αα
αα-1-A -1-A -1-AT and hFIX in Mice T and hFIX in Mice
Eric C Olivares, Roger P Hollis, Leonard Meuse, Mark Kay,
Michele P Calos
α"8" - '' / F - ?F/
929 Neonatal Retroviral V
929 Neonatal Retroviral Vector-Mediated Gene ector-Mediated Gene Therapy for Mucopolysaccharidosis VII in Dogs Results
in Stable Hepatocyte T
in Stable Hepatocyte Transduction and Marked ransduction and Marked 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; 2 Departments of Internal Medicine and Biochemistry and Molecular Biophysics, Washington University School of Medicine, St Louis, MO, United States; 3 College of Veterinary Medicine, Cornell University, Ithaca, NY, United States; 4 Anatomy, Philadelphia College of Osteopathic Medicine, Philadelphia, PA, United States; 5 Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, United States; 6 Children ′s Hospital of Philadelphia, Philadelphia, PA, United States
Trang 2Molecular Therapy Vol 5, No 5, May 2002, Part 2 of 2 Parts Copyright © The American Society of Gene Therapy
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930. In Vivo In Vivo Functional Correction of Hemophilia a Functional Correction of Hemophilia a
Mice Via a Novel Spliceosome-Mediated RNA Trans
Trans -Splicing 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; 2 Intronn Inc., Raleigh, NC, United States
88
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; 2 BM Transplantation and Pediatrics, Hadassah University Hospital, Jerusalem, Israel;
3 Inst of Biochemistry and Enzimology, University of Siena, Siena, Italy; 4 BMT and Gene Therapy Program, Scientific Institute HS Raffaele, Milan, Italy; 5 Dep of Biological and Clinical Sciences, University of Turin, Turin, Italy
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...in Stable Hepatocyte Transduction and Marked ransduction and Marked Improvement in Bone and Joint, Cardiac, and Corneal Manifestations for up to 14 Months
Mark Haskins,1...
929 Neonatal Retroviral V
929 Neonatal Retroviral Vector- Mediated Gene ector -Mediated Gene Therapy for Mucopolysaccharidosis VII in Dogs Results< /b>
in Stable Hepatocyte. .. Internal Medicine and Biochemistry and Molecular Biophysics, Washington University School of Medicine, St Louis, MO, United States; 3 College of Veterinary Medicine, Cornell University,