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Gene Therapy - Technologies, Markets and Companies
Date:11/30/2011

9

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

LPL deficiency 197

Ornithine transcarbamylase deficiency 198

Phenylketonuria 198

Porphyrias 199

Tetrahydrobiopterin deficiency 199

Lysosomal storage disorders. 200

Batten disease 201

Fabry's disease 201

Farber's disease 202

Gaucher disease 202

Animals models of Gaucher's disease 202

Gene therapy of Gaucher's disease 203

Hunter syndrome 204

Combination of cell and gene therapy for Krabbe's disease 204

Metachromatic leukodystrophy 205

Mucopolysaccharidosis type 1 (Hurler syndrome) 205

Niemann-Pick type A disease 206

Pompe disease 206

Sanfilippo A syndrome 207

Sly syndrome 207

Tay-Sachs disease 207

Future prospects of gene therapy of lysosomal storage disorders 208

Trinucleotide repeat disorders 208

Muscular dystrophies 208

Duchenne muscular dystrophy (DMD) 208

Animal models for gene therapy of DMD 209

Antisense approach to DMD 209

Exon-skipping technology for DMD 210

Liposome-mediated gene transfer 210

Myoblast-based gene transfer in DMD 211

Plasmid-mediated gene therapy 211

Post-transcriptional modulation of gene expression in DMD 211

Repair of dystrophin gene 212

Routes of administration of gene therapy in DMD 212

Types of dystrophin constructs 212

Viral vectors for DMD 213

Conclusions and future prospects of gene thera
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