ALS, IGF-1 and gene therapy: 'It's never too late to mend'

Gene Therapy

Volumn 11, Issue 5, 2004, Pages 429-430

  Raoul, Cédric ^a   Aebischer, Patrick ^a  

^a EPFL   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

BRAIN DERIVED NEUROTROPHIC FACTOR; CARDIOTROPHIN 1; CILIARY NEUROTROPHIC FACTOR; COPPER ZINC SUPEROXIDE DISMUTASE; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; NEUROTROPHIC FACTOR; PARVOVIRUS VECTOR; RECOMBINANT PROTEIN; RECOMBINANT SOMATOMEDIN C;

AMYOTROPHIC LATERAL SCLEROSIS; CLINICAL FEATURE; CLINICAL TRIAL; DRUG ACTIVITY; DRUG BIOAVAILABILITY; DRUG BLOOD LEVEL; DRUG HALF LIFE; DRUG SCREENING; HUMAN; NEUROPROTECTION; NONHUMAN; NOTE; PATHOGENESIS; POINT MUTATION; PRIORITY JOURNAL; RISK BENEFIT ANALYSIS; SIDE EFFECT; TRANSGENIC MOUSE; TREATMENT FAILURE; VIRAL GENE DELIVERY SYSTEM;

MUS MUSCULUS; PARVOVIRUS;

EID: 1542269679     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302204     Document Type: Note

References (16)

1. Turner MR, Parton MJ, Leigh PN. Clinical trials in ALS: an overview. Semin Neurol 2001; 21: 167-175.
2. Newbery HJ, Abbott CM. Of mice, men and motor neurons. Trends Genet 2001; 17: S2-S6.
3. Azari MF et al. Behavioural and anatomical effects of systemically administered leukemia inhibitory factor in the SOD1(G93A G1H) mouse model of familial amyotrophic lateral sclerosis. Brain Res 2003; 982: 92-97.
4. Feeney SJ et al. The effect of leukaemia inhibitory factor on SOD1 G93A murine amyotrophic lateral sclerosis. Cytokine 2003; 23: 108-118.
5. Dreibelbis JE et al. Disease course unaltered by a single intracisternal injection of BMP-7 in ALS mice. Muscle Nerve 2002; 25: 122-123.
6. Wang LJ et al. 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 2002; 22: 6920-6928.
7. Bordet T et al. Protective effects of cardiotrophin-1 adenoviral gene transfer on neuromuscular degeneration in transgenic ALS mice. Hum Mol Genet 2001; 10: 1925-1933.
8. Kaspar BK et al. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 2003; 301: 839-842.
9. Haase G et al. Gene therapy of murine motor neuron disease using adenoviral vectors for neurotrophic factors. Nat Med 1997; 3: 429-436.
10. Clement AM et al. Wild-type nonneuronal cells extent survival of SOD1 mutant motor neurons in ALS mice. Science 2003; 302: 113-117.
11. Azzouz M et al. Increased motoneuron survival and improved neuromuscular function in transgenic ALS mice after intraspinal injection of an adeno-associated virus encoding Bcl-2. Hum Mol Genet 2000; 9: 803-811.
12. Lai EC et al. Effect of recombinant human insulin-like growth factor-I on progression of ALS. A placebo-controlled study. The North America ALS/IGF-I Study Group. Neurology 1997; 49: 1621-1630.
13. Borasio GD et al. A placebo-controlled trial of insulin-like growth factor-I in amyotrophic lateral sclerosis. European ALS/IGF-I Study Group. Neurology 1998; 51: 583-586.
14. Paterna JC, Bueler H. Recombinant adeno-associated virus vector design and gene expression in the mammalian brain. Methods 2002; 28: 208-218.
15. Mazarakis ND et al. Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Hum Mol Genet 2001; 10: 2109-2121.
16. Deglon N, Aebischer P. Lentiviruses as vectors for CNS diseases. Curr Top Microbiol Immunol 2002; 261: 191-209.