Gene therapy for lympho-hematopoietic disorders.
Inherited lympho-hematopoietic disorders are considered to be some of the most amenable targets for development of gene therapy because of their defined molecular biology and pathophysiology and the potential for corrected cells to exhibit profound growth and survival advantages. Recently, several clinical studies have shown that conventional gene transfer technology can produce major beneficial therapeutic effects.
Thrasher
Adrian J
AJ
Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK. a.thrasher@ich.ucl.ac.uk
Gaspar
H Bobby
HB
D015316:Genetic Therapy / Q000009:adverse effects / Q000592:standards; D006402:Hematologic Diseases / Q000628:therapy*; D019337:Hematologic Neoplasms / Q000628:therapy*; D008206:Lymphatic Diseases / Q000628:therapy*; D016254:Mutagenesis, Insertional / Q000502:physiology; D011488:Protein Deficiency / Q000628:therapy; D018121:Receptors, Cytokine / Q000172:deficiency; D012307:Risk Factors; D016511:Severe Combined Immunodeficiency / Q000628:therapy
D018121:Receptors, Cytokine