Table of contents

Article Outline

 

Introduction: Gene Therapy for Hematologic Disease: Don’t Throw the Baby Out With the Bathwater! 255

Cynthia E. Dunbar

Genetic Manipulation of Hematopoietic Stem Cells 257

André Larochelle and Cynthia E. Dunbar

Gene Therapy for Immunodeficiency Diseases 272

Alain Fischer, Salima Hacein-Bey-Abina, and Marina Cavazzana-Calvo

Gene Therapy for the Hemoglobin Disorders 279

Derek A. Persons and John F. Tisdale

Gene Therapy of the Hemophilias 287

Jay Lozier

New Paradigms for Gene Transfer: RNA Trans-Splicing and Small Interfering RNA as Therapeutic Strategies 297

Christopher E. Walsh

Stem Cell Clonality and Genotoxicity in Hematopoietic Cells: Gene Activation Side Effects Should Be Avoidable 303

C. von Kalle, B. Fehse, G. Layh-Schmitt, M. Schmidt, P. Kelly, and C. Baum

Annual Index 319