Recombinant adeno-associated virus vectors in the treatment of rare diseases

Expert Opinion on Orphan Drugs

Volumn 3, Issue 6, 2015, Pages 675-689

  Hastie, Eric ^a   Jude Samulski, R ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Adeno associated virus vectors; Gene therapy; Orphan disease; Rare disease

Indexed keywords

ALPHA 1 ANTITRYPSIN; LONG CHAIN ACYL COENZYME A DEHYDROGENASE; PARVOVIRUS VECTOR;

AGE RELATED MACULAR DEGENERATION; DUCHENNE MUSCULAR DYSTROPHY; ENZYME DEFICIENCY; FAMILIAL AMYLOID POLYNEUROPATHY; FRAGILE X SYNDROME; FRIEDREICH ATAXIA; GENE EXPRESSION; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 2; HEMOPHILIA B; HUMAN; HUNTINGTON CHOREA; HURLER SYNDROME; LIMB GIRDLE MUSCULAR DYSTROPHY; MEDICAL INFORMATION; METACHROMATIC LEUKODYSTROPHY; MYASTHENIA; NONHUMAN; PARKINSON DISEASE; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENYLKETONURIA; PRIORITY JOURNAL; RARE DISEASE; RETINITIS PIGMENTOSA; REVIEW; SMITH LEMLI OPITZ SYNDROME; SPINAL MUSCULAR ATROPHY; VIRAL GENE THERAPY; VIRAL TROPISM; VIRUS CAPSID; VIRUS RECOMBINANT;

EID: 84930164222     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2015.1039511     Document Type: Review

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