Biomedical Engineering Reference
In-Depth Information
26. Airaksinen MS, Saarma M (2002) The GDNF
family: signalling, biological functions and ther-
apeutic value. Nat Rev Neurosci 3:383-394
27. Kordower JH, Palfi S, Chen EY et al (1999)
Clinicopathological fi ndings following intra-
ventricular glial-derived neurotrophic factor
treatment in a patient with Parkinson's disease.
Ann Neurol 46:419-424
28. Nutt JG, Burchiel KJ, Comella CL et al (2003)
Randomized, double-blind trial of glial cell
line-derived neurotrophic factor (GDNF) in
PD. Neurology 60:69-73
29. Slevin JT, Gerhardt GA, Smith CD et al (2005)
Improvement of bilateral motor functions in
patients with Parkinson disease through the
unilateral intraputaminal infusion of glial cell
line-derived neurotrophic factor. J Neurosurg
102:216-222
30. Lang AE, Gill SS, Patel NK et al (2006)
Randomized controlled trial of intraputamenal
glial cell line-derived neurotrophic factor infusion
in Parkinson disease. Ann Neurol 59:459-466
31. Barker RA (2006) Continuing trials of GDNF in
Parkinson's disease. Lancet Neurol 5:285-286
32. Kordower JH, Bjorklund A (2013) Trophic
factor gene therapy for Parkinson's disease.
Mov Disord 28:96-109
33. Bartus RT, Brown L, Wilson A et al (2011)
Properly scaled and targeted AAV2-NRTN
(neurturin) to the substantia nigra is safe,
effective and causes no weight loss: support for
nigral targeting in Parkinson's disease.
Neurobiol Dis 44:38-52
34. Bartus RT, Weinberg MS, Samulski RJ (2014)
Parkinson's disease gene therapy: success by
design meets failure by effi cacy. Mol Ther
22:487-497
35. Mittermeyer G, Christine CW, Rosenbluth
KH et al (2012) Long-term evaluation of a
phase 1 study of AADC gene therapy for
Parkinson's disease. Hum Gene Ther 23:
377-381
36. Kaplitt MG, Feigin A, Tang C et al (2007)
Safety and tolerability of gene therapy with an
adeno-associated virus (AAV) borne GAD
gene for Parkinson's disease: an open label,
phase 1 trial. Lancet 369:2097-2105
37. Palfi S, Gurruchaga JM, Ralph GS et al (2014)
Long-term safety and tolerability of ProSavin,
a lentiviral vector-based gene therapy for
Parkinson's disease: a dose escalation, open-
label, phase 1/2 trial. Lancet 383:1138-1146
38. Kells AP, Forsayeth J, Bankiewicz KS (2012)
Glial-derived neurotrophic factor gene transfer
for Parkinson's disease: anterograde distribu-
tion of AAV2 vectors in the primate brain.
Neurobiol Dis 48:228-235
39. Richardson RM, Kells AP, Rosenbluth KH
et al (2011) Interventional MRI-guided
putaminal delivery of AAV2-GDNF for a
planned clinical trial in Parkinson's disease.
Mol Ther 19:1048-1057
40. Gaudet D, de Wal J, Tremblay K et al (2010)
Review of the clinical development of alipo-
gene tiparvovec gene therapy for lipoprotein
lipase defi ciency. Atheroscler Suppl 11:55-60
41. Gaudet D, Méthot J, Kastelein J (2012) Gene
therapy for lipoprotein lipase defi ciency. Curr
Opin Lipidol 23:310-320
42. Gaudet D, Méthot J, Déry S et al (2013)
Effi cacy and long-term safety of alipogene
tiparvovec (AAV1-LPLS447X) gene therapy
for lipoprotein lipase defi ciency: an open-label
trial. Gene Ther 20:361-369
43. Salmon F, Grosios K, Petry H (2014) Safety pro-
fi le of recombinant adeno-associated viral vectors:
focus on alipogene tiparvovec (Glybera(®)).
Expert Rev Clin Pharmacol 7:53-65
44. Bryant LM, Christopher DM, Giles AR et al
(2013) Lessons learned from the clinical devel-
opment and market authorization of Glybera.
Hum Gene Ther Clin Dev 24:55-56
45. Dismuke DJ, Tenenbaum L, Samulski RJ
(2013) Biosafety of recombinant adeno-
associated virus vectors. Curr Gene Ther
13:434-452
46. Pochon NA, Menoud A, Tseng JL et al (1997)
Neuronal GDNF expression in the adult rat
nervous system identifi ed by in situ hybridiza-
tion. Eur J Neurosci 1997:463-471
47. Akerud P, Alberch J, Eketjäll S et al (2012)
Differential effects of glial cell line-derived
neurotrophic factor and neurturin on develop-
ing and adult substantia nigra dopaminergic
neurons. J Neurochem 73:70-78
48. Sariola H, Saarma M (2003) Novel functions
and signalling pathways for GDNF. J Cell Sci
116:3855-3862
49. Eberling JL, Kells AP, Pivirotto P et al (2009)
Functional effects of AAV2-GDNF on the dopa-
minergic nigrostriatal pathway in parkinsonian
rhesus monkeys. Hum Gene Ther 20:511-518
50. Urabe M, Ding C, Kotin RM (2002) Insect
cells as a factory to produce adeno-associated
virus type 2 vectors. Hum Gene Ther
13:1935-1943
51. Unzu C, Hervás-Stubbs S, Sampedro A et al
(2012) Transient and intensive pharmacologi-
cal immunosuppression fails to improve AAV-
based liver gene transfer in non-human
primates. J Transl Med 10:122
52. Kotin RM (2011) Large-scale recombinant
adeno-associated virus production. Hum Mol
Genet 20:R2-R6
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