(with much hope this group does not sell their patent to Amgen....)

http://www.ncbi.nlm.nih.gov/pubmed/1...?dopt=Abstract

1: Int J Pharm. 2009 Oct 10. [Epub ahead of print]Related Articles, Links
Production of highly pure human glycosylated GDNF in a mammalian cell-line.

Ansorena E, Garbayo E, Lanciego JL, Aymerich MS, Blanco-Prieto MJ.

Department of Pharmacy and Pharmaceutical Technology, School of Pharmacy, University of Navarra, C/Irunlarrea 1, E-31080 Pamplona, Spain.

The administration of glial cell line-derived neurotrophic factor (GDNF) has emerged as a promising strategy for the treatment of several diseases of the nervous system as Parkinson's disease, amyotrophic lateral sclerosis, spinal cord injury and nerve regeneration as well as ocular diseases and drug addictions. A procedure for the purification of human recombinant glycosylated GDNF using a mammalian expression system as the source of the protein is discussed in the present paper. The neurotrophic factor was purified using cation exchange chromatography and gel filtration. A human cell line was chosen as the source of therapeutic protein, since a recombinant protein with a structure and glycosylation pattern equivalent to the native form is desirable for its prospective therapeutic utilization. The activity of the highly pure protein obtained was confirmed with a cell based bioassay. The purified protein is suitable for its in vivo evaluation in animals and for possible subsequent clinical application.

PMID: 19825405 [PubMed - as supplied by publisher]

Viral vectors for neurotrophic factor delivery:
A gene therapy approach for neurodegenerative diseases of the CNS




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Seung Lima, Mikko Airavaarab and Brandon K. Harveyb, ,

aDepartment of Neuroscience, Georgetown University Medical Center, Washington, D.C, 20057, United States

bIntramural Research Program, National Institute on Drug Abuse, National Institutes of Health, Baltimore, MD 21224, United States


Received 18 June 2009; revised 11 October 2009; accepted 11 October 2009.
Available online 17 October 2009.
Abstract
The clinical manifestation of most diseases of the central nervous system results from neuronal dysfunction or loss. Diseases such a stroke, epilepsy and neurodegeneration (e.g. Alzheimer's disease and Parkinson's disease) share common cellular and molecular mechanisms (e.g. oxidative stress, endoplasmic reticulum stress, mitochondrial dysfunction) that contribute to the loss of neuronal function. Neurotrophic factors (NTFs) are secreted proteins that regulate multiple aspects of neuronal development including neuronal maintenance, survival, axonal growth and synaptic plasticity. These properties of NTFs make them likely candidates for preventing neurodegeneration and promoting neuroregeneration.

One approach to delivering NTFs to diseased neurons is through viral vector-mediated gene delivery. Viral vectors are now routinely used as tools for studying gene function as well as developing gene-based therapies for a variety of diseases. Currently, many clinical trials using viral vectors in the nervous system are underway or completed, and seven of these trials involve NTFs for neurodegeneration. In this review, we discuss viral vector-mediated gene transfer of NTFs to treat neurodegenerative diseases of the central nervous system.

Keywords: Neurotrophic factor; Neurodegeneration; Gene therapy; Viral vector; adeno-associated virus; aav; lentivirus; herpes virus; HSV; adenovirus; Parkinson's disease; Alzheimer's disease; amyotrophic lateral sclerosis; stroke; epilepsy; huntington's disease; central nervous system; transgene; clinical trial


Corresponding author. National Institute on Drug Abuse, National Institutes of Health, BRC Suite 200, Rm 06A729, 251 Bayview Blvd, Baltimore, MD 21224. Tel.: +1 443 740 2590

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