The final frontier - crossing the blood-brain barrier

EMBO Molecular Medicine

Volumn 5, Issue 5, 2013, Pages 655-657

  Sly, William S ^a   Vogler, Carole ^a  

^a SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Gene therapy; Lysosomal storage diseases; Mucopolysaccharidosis; Sulfamidase; Transcytosis

Indexed keywords

APOLIPOPROTEIN B; BETA GLUCURONIDASE; GLYCOSAMINOGLYCAN; HEPARAN SULFATE; IDURONATE 2 SULFATASE; LOW DENSITY LIPOPROTEIN RECEPTOR; MANNOSE RECEPTOR; PARVOVIRUS VECTOR; SOMATOMEDIN B RECEPTOR;

ADENO ASSOCIATED VIRUS; ARTICLE; AUTOPHAGY; BLOOD BRAIN BARRIER; BRAIN CAPILLARY; CENTRAL NERVOUS SYSTEM; CEREBROSPINAL FLUID; CHEMICAL MODIFICATION; DRUG DELIVERY SYSTEM; DRUG EFFICACY; ENDOCYTOSIS; ENZYME THERAPY; GENE EXPRESSION; HUMAN; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PATHOPHYSIOLOGY; PRIORITY JOURNAL; PROTEIN DEGRADATION;

ANIMALS; BLOOD-BRAIN BARRIER; BRAIN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; GENETIC VECTORS; HUMANS; HYDROLASES; LYSOSOMAL STORAGE DISEASES; MUCOPOLYSACCHARIDOSES; RECEPTOR, IGF TYPE 2; TRANSCYTOSIS;

EID: 84877318308     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.1002/emmm.201302668     Document Type: Article

References (11)

1. Biffi A, Capotondo A, Fasano S, del Carro U, Marchesini S, Azuma H, Malaguti MC, Amadio S, Brambilla R, Grompe M, et al (2006) Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest 116: 3070- 3082
2. Fraldi A, Hemsley K, Crawley A, Lombardi A, Lau A, Sutherland L, Auricchio A, Ballabio A, Hopwood JJ (2007) Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes. Hum Mol Genet 16: 2693- 2702
3. Grubb JH, Vogler C, Sly WS (2010) New strategies for enzyme replacement therapy for lysosomal storage diseases. Rejuvenation Res 13: 229- 236
4. Meng Y, Sohar I, Wang L, Sleat DE, Lobel P (2012) Systemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification. PLoS ONE 7: e40509
5. Rozaklis T, Beard H, Hassiotis S, Garcia AR, Tonini M, Luck A, Pan J, Lamsa JC, Hopwood JJ, Hemsley KM (2011) Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA. Exp Neurol 230: 123- 130
6. Sorrentino NC, D'Orsi L, Sambri I, Nusco E, Monaco C, Spampanato C, Polishchuk E, Saccone P, De Leonibus E, Ballabio A et al (2013) A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA. EMBO Mol Med 5: 675- 690
7. Spencer B, Marr RA, Rockenstein E, Crews L, Adame A, Potkar R, Patrick C, Gage FH, Verma IM, Masliah E (2008) Long-term neprilysin gene transfer is associated with reduced levels of intracellular Abeta and behavioral improvement in APP transgenic mice. BMC Neurosci 9: 109
8. Spencer BJ, Verma IM (2007) Targeted delivery of proteins across the blood-brain barrier. Proc Natl Acad Sci USA 104: 7594- 7599
9. Urayama A, Grubb JH, Sly WS, Banks WA (2008) Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse. Mol Ther 16: 1261- 1266
10. Vogler C, Levy B, Grubb JH, Galvin N, Tan Y, Kakkis E, Pavloff N, Sly WS (2005) Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII. Proc Natl Acad Sci USA 102: 14777- 14782
11. Zhou QH, Boado RJ, Pardridge WM (2012) Selective plasma pharmacokinetics and brain uptake in the mouse of enzyme fusion proteins derived from species-specific receptor-targeted antibodies. J Drug Target 20: 715- 719