Gene therapy for neurologic manifestations of mucopolysaccharidoses

Expert Opinion on Drug Delivery

Volumn 12, Issue 2, 2015, Pages 283-296

  Wolf, Daniel A ^a   Banerjee, Sharbani ^a   Hackett, Perry B ^a   Whitley, Chester B ^b   McIvor, R Scott ^a   Low, Walter C ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

^b UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

Author keywords

Enzyme replacement therapy; Gene therapy; Hunter syndrome; Hurler syndrome; Sanfilippo syndrome

Indexed keywords

CENTRAL NERVOUS SYSTEM AGENTS; GENE THERAPY AGENT; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; RETROVIRUS VECTOR;

BLOOD BRAIN BARRIER; ENZYME REPLACEMENT; EX VIVO STUDY; GENE DELIVERY SYSTEM; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; MUCOPOLYSACCHARIDOSIS; NEUROLOGIC DISEASE; NEUROPATHOLOGY; REVIEW; TRANSPOSON; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; ANIMAL; BRAIN; DEPENDOPARVOVIRUS; GENE VECTOR; GENETICS; METABOLISM; MUCOPOLYSACCHARIDOSES; PATHOPHYSIOLOGY; PROCEDURES;

ANIMALS; BLOOD-BRAIN BARRIER; BRAIN; DEPENDOVIRUS; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MUCOPOLYSACCHARIDOSES;

EID: 84921480276     PISSN: 17425247     EISSN: 17447593     Source Type: Journal    
DOI: 10.1517/17425247.2015.966682     Document Type: Review

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