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Gene therapy for arthritis 185
Cancer gene therapy 185
Brain tumors in cats and dogs 185
Breast cancer in dogs 186
Canine hemangiosarcoma 187
Canine melanoma 187
Canine soft tissue sarcoma 187
Melanoma in horses 188
4. Gene Therapy of Genetic Disorders 189
Introduction 189
Primary immunodeficiency disorders 190
Severe combined immune deficiency 191
Chronic granulomatous disease 193
Wiskott-Aldrich syndrome 193
Purine nucleoside phosphorylase deficiency 194
Major histocompatibility class II deficiency 194
Future prospects of gene therapy of inherited immunodeficiencies 195
Metabolic disorders 195
Adrenoleukodystrophy 196
Canavan disease 196
Lesch-Nyhan syndrome 197
Ornithine transcarbamylase deficiency 197
Phenylketonuria 198
Porphyrias 198
Tetrahydrobiopterin deficiency 199
Lysosomal storage disorders. 199
Batten disease 200
Fabry's disease 201
Farber's disease 201
Gaucher disease 202
Animals models of Gaucher's disease 202
Gene therapy of Gaucher's disease 202
Hunter syndrome 203
Combination of cell and gene therapy for Krabbe's disease 203
Metachromatic leukodystrophy 204
Mucopolysaccharidosis type 1 (Hurler syndrome) 205
Niemann-Pick type A disease 205
Pompe disease 205
Sanfilippo A syndrome 206
Sly syndrome 206
Tay-Sachs disease 207
Future prospects of gene therapy of lysosomal storage disorders 207
Trinucleotide repeat disorders 207
Muscular dystrophies 208
Duchenne muscular dystrophy (DMD) 208
Animal models for gene therapy of DMD 208
Antisense approach to DMD 208
Liposome-mediated gene transfer 209
Myoblast-based gene transfer in DMD 210
Plasmid-mediated gene therapy 210
Post-transcriptional modulation of gene
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