Gene therapy for the treatment of neurological disorders: Metabolic disorders

Methods in Molecular Biology

Volumn 1382, Issue , 2016, Pages 429-465

  Gessler, Dominic J ^a   Gao, Guangping ^a  

^a NONE

Author keywords

AAV; Canavan's disease; CNS; Gene therapy; Metabolic disorders; Niemann Pick disease; Phenylketonuria; Retrovirus

Indexed keywords

ASPARTOACYLASE; PHENYLALANINE 4 MONOOXYGENASE; RECOMBINANT ENZYME; SPHINGOMYELIN; SPHINGOMYELIN PHOSPHODIESTERASE;

ADAPTIVE IMMUNITY; ADENO ASSOCIATED VIRUS; APOPTOSIS; AUTOPHAGY; BLOOD BRAIN BARRIER; BONE MARROW DERIVED MESENCHYMAL STEM CELL; CANAVAN DISEASE; CELL DIFFERENTIATION; CELL GROWTH; CELLULAR IMMUNITY; COMPETITIVE INHIBITION; ENZYME ACTIVITY; ENZYME REPLACEMENT; EX VIVO GENE TRANSFER; FOAM CELL; GENE MUTATION; GENE TARGETING; HUMAN; HYPERPHENYLALANINEMIA; IMMUNOGENICITY; IN VITRO GENE TRANSFER; IN VITRO STUDY; IN VIVO GENE TRANSFER; LIPOSOMAL GENE DELIVERY SYSTEM; LONG TERM SURVIVAL; METABOLIC DISORDER; MOTOR PERFORMANCE; NEUROLOGIC DISEASE; NIEMANN PICK DISEASE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHENYLKETONURIA; PREVALENCE; PROMOTER REGION; PROTEIN SYNTHESIS; STEM CELL GENE THERAPY; THERAPY EFFECT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS RECOMBINANT; WHOLE BODY RADIATION; CLINICAL TRIAL (TOPIC); GENE THERAPY; METABOLIC DISEASES; NERVOUS SYSTEM DISEASES;

CLINICAL TRIALS AS TOPIC; GENETIC THERAPY; HUMANS; METABOLIC DISEASES; NERVOUS SYSTEM DISEASES;

EID: 84948448009     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-4939-3271-9_30     Document Type: Chapter

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