Genome Editing: Potential Treatment for Lysosomal Storage Diseases

Current Stem Cell Reports

Volumn 1, Issue 1, 2015, Pages 9-15

  de Carvalho, Talita Giacomet ^a   da Silveira Matte, Ursula ^a   Giugliani, Roberto ^a   Baldo, Guilherme ^a  

^a HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

Author keywords

CRISPR Cas9; Lysosomal storage disorders; TAL effector nucleases; Zinc finger nucleases

Indexed keywords

ZINC FINGER NUCLEASE;

ALPHA 1 ANTITRYPSIN DEFICIENCY; ASPARTYLGLYCOSAMINURIA; BETA THALASSEMIA; DNA DAMAGE; DNA MODIFICATION; DUCHENNE MUSCULAR DYSTROPHY; ENZYME REPLACEMENT; GAUCHER DISEASE; GENE MUTATION; GENE TECHNOLOGY; GENE THERAPY; GENOME EDITING TECHNOLOGY; GLOBOID CELL LEUKODYSTROPHY; GLYCOGEN STORAGE DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LIPIDOSIS; LIPOFUSCINOSIS; LYSOSOME STORAGE DISEASE; MANNOSIDOSIS; MAROTEAUX LAMY SYNDROME; MESENCHYMAL STEM CELL; METACHROMATIC LEUKODYSTROPHY; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; OLIGOSACCHARIDOSES; PARKINSON DISEASE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; RETINA DISEASE; REVIEW; SANFILIPPO SYNDROME; SICKLE CELL ANEMIA; TYROSINEMIA; WOLMAN DISEASE; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84936949109     PISSN: None     EISSN: 21987866     Source Type: Journal    
DOI: 10.1007/s40778-014-0007-8     Document Type: Review

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