Erratum: Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia (Journal of Clinical Investigation (2006) 116:1 (202-208) doi:10.1172/JCI26210);Intramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegia

Journal of Clinical Investigation

Volumn 116, Issue 1, 2006, Pages 202-208

  Pirozzi, Marinella ^a   Quattrini, Angelo ^b   Andolfi, Gennaro ^a   Dina, Giorgia ^b   Malaguti, Maria Chiara ^b   Auricchio, Alberto ^a   Rugarli, Elena I ^a,c  

^a TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^c National Neurological Institute   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

PARAPLEGIN; PARVOVIRUS VECTOR; PROTEINASE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DISEASE COURSE; DISEASE MODEL; GENE TRANSFER; HEREDITARY MOTOR SENSORY NEUROPATHY; INJECTION; MOTOR PERFORMANCE; MOUSE; NEUROLOGIC DISEASE; NONHUMAN; PERIPHERAL NEUROPATHY; PHENOTYPE; PRIORITY JOURNAL; SPINAL CORD NERVE CELL; SYMPTOM; VIRAL GENE DELIVERY SYSTEM;

EID: 31044456528     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI75082     Document Type: Erratum

References (35)

1. Harding, A.E. 1983. Classification of the hereditary ataxias and paraplegias. Lancet. 1:1151-1154.
2. Harding, A.E. 1984. Hereditary "pure" spastic paraplegia. In The hereditary ataxias and related disorders. Churchill Livingstone. New York, New York, USA. 174-191.
3. Reid, E. 2003. Science in motion: common molecular pathological themes emerge in the hereditary spastic paraplegias. J. Med. Genet. 40:81-86.
4. Schwarz, G.A., and Liu, C.N. 1956. Hereditary (familial) spastic paraplegia: further clinical and pathological observations. Arch. Neurol. Psychiatry. 75:144-162.
5. Fink, J.K. 2003. Advances in the hereditary spastic paraplegias. Exp. Neurol. 184(Suppl. 1):S106-S110.
6. Casari, G., et al. 1998. Spastic paraplegia and OXPHOS impairment caused by mutations in paraplegin, a nuclear-encoded mitochondrial metalloprotease. Cell. 93:973-983.
7. Wilkinson, P.A., et al. 2004. A clinical, genetic and biochemical study of SPG7 mutations in hereditary spastic paraplegia. Brain. 127:973-980.
8. Langer, T. 2000. AAA proteases: cellular machines for degrading membrane proteins. Trends Biochem. Sci. 25:247-251.
9. Langer, T., Kaser, M., Klanner, C., and Leonhard, K. 2001. AAA proteases of mitochondria: quality control of membrane proteins and regulatory functions during mitochondrial biogenesis. Biochem. Soc. Trans. 29:431-436.
10. Nolden, M., et al. 2005. The m-AAA protease defective in hereditary spastic paraplegia controls ribosome assembly in mitochondria. Cell. 123:277-289.
11. Ferreirinha, F., et al. 2004. Axonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transport. J. Clin. Invest. 113:231-242. doi:10.1172/JCI200420138.
12. Mazarakis, N.D., et al. 2001. Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Hum. Mol. Genet. 10:2109-2121.
13. Kaspar, B.K., Llado, J., Sherkat, N., Rothstein, J.D., and Gage, F.H. 2003. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science. 301:839-842.
14. Azzouz, M., et al. 2004. Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J. Clin. Invest. 114:1726-1731. doi:10.1172/JCI200422922.
15. Azzouz, M., et al. 2004. VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature. 429:413-417.
16. LaMonte, B.H., et al. 2002. Disruption of dynein/dynactin inhibits axonal transport in motor neurons causing late-onset progressive degeneration. Neuron. 34:715-727.
17. Mohajeri, M.H., Figlewicz, D.A., and Bohn, M.C. 1998. Selective loss of alpha motoneurons innervating the medial gastrocnemius muscle in a mouse model of amyotrophic lateral sclerosis. Exp. Neurol. 150:329-336.
18. Gregorevic, P., et al. 2004. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat. Med. 10:828-834.
19. Monahan, P.E., and Samulski, R.J. 2000. AAV vectors: is clinical success on the horizon? Gene Ther. 7:24-30.
20. Xiao, W., et al. 1999. Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73:3994-4003.
21. Hildinger, M., and Auricchio, A. 2004. Advances in AAV-mediated gene transfer for the treatment of inherited disorders. Eur. J. Hum. Genet. 12:263-271.
22. Wang, L.J., et al. 2002. Neuroprotective effects of glial cell line-derived neurotrophic factor mediated by an adeno-associated virus vector in a transgenic animal model of amyotrophic lateral sclerosis. J. Neurosci. 22:6920-6928.
23. Dobrowolny, G., et al. 2005. Muscle expression of a local Igf-1 isoform protects motor neurons in an ALS mouse model. J. Cell Biol. 168:193-199.
24. Delettre, C., et al. 2000. Nuclear gene OPA1, encoding a mitochondrial dynamin-related protein, is mutated in dominant optic atrophy. Nat. Genet. 26:207-210.
25. Alexander, C., et al. 2000. OPA1, encoding a dynamin-related GTPase, is mutated in autosomal dominant optic atrophy linked to chromosome 3q28. Nat. Genet. 26:211-215.
26. Zuchner, S., et al. 2004. Mutations in the mitochondrial GTPase mitofusin 2 cause Charcot-Marie-Tooth neuropathy type 2A. Nat. Genet. 36:449-451.
27. Patel, H., et al. 2002. SPG20 is mutated in Troyer syndrome, an hereditary spastic paraplegia. Nat. Genet. 31:347-348.
28. Windpassinger, C., et al. 2004. Heterozygous missense mutations in BSCL2 are associated with distal hereditary motor neuropathy and Silver syndrome. Nat. Genet. 36:271-276.
29. Fink, J.K. 2002. Hereditary spastic paraplegia: the pace quickens. Ann. Neurol. 51:669-672.
30. Auricchio, A., Hildinger, M., O'Connor, E., Gao, G.P., and Wilson, J.M. 2001. Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Hum. Gene Ther. 12:71-76.
31. Auricchio, A., et al. 2001. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum. Mol. Genet. 10:3075-3081.
32. Gao, G.P., et al. 2002. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U. S. A. 99:11854-11859.
33. Gao, G., et al. 2000. Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo. Hum. Gene Ther. 11:2079-2091.
34. Previtali, S.C., et al. 2000. Epitope-tagged P(0) glycoprotein causes Charcot-Marie-Tooth-like neuropathy in transgenic mice. J. Cell Biol. 151:1035-1046.
35. Consiglio, A., et al. 2001. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat. Med. 7:310-316.