Adenovirus in the brain: recent advances of gene therapy for neurodegenerative diseases

Barkats, Martine; Bilang-Bleuel, Alicia; Buc-Caron, Marie-Hélène; Castel-Barthe, M.N.; Corti, Olga; Finiels, Françoise; Horellou, Philippe; Revah, Frédéric; Sabaté, Olivier; Mallet, Jacques

doi:10.1016/s0301-0082(98)00028-8

Cited by 86 publications

(40 citation statements)

References 51 publications

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“…rAd was one of the earliest viral vectors used with success in the brain (Le Gal La Salle et al, 1993) and in animal models of PD (Barkats et al, 1998;Choi-Lundberg et al, 1997). Wild type Ad (wt-Ad) commonly infects humans and is known to cause respiratory infections, conjunctivitis, and gastroenteritis (Shenk, 1996).…”

Section: Recombinant Adenovirus (Rad)mentioning

confidence: 99%

“…In recent years, investigators have developed techniques for engineering the genome of many different viruses to transfer a gene of interest (transgene). Moreover, production procedures have been devised for these vectors in sufficient quantities and of sufficient purity to enable experimentation in animal models of PD and for clinical trials (Barkats et al, 1998;Burton et al, 2003;Mandel and Burger, 2004;Mandel et al, 2006). To illustrate this trend, publications that report gene transfer to treat or study PD have increased over the years ( Fig.…”

Section: Introductionmentioning

confidence: 99%

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Viral vectors for in vivo gene transfer in Parkinson's disease: Properties and clinical grade production

Mandel

Bürger

Snyder

2008

Experimental Neurology

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Because Parkinson's disease is a progressive degenerative disorder that is mainly confined to the basal ganglia, gene transfer to deliver therapeutic molecules is an attractive treatment avenue. The present review focuses on direct in vivo gene transfer vectors that have been developed to a degree that they have been successfully used in animal model of Parkinson's disease. Accordingly, the properties of recombinant adenovirus, recombinant adeno-associated virus, herpes simplex virus, and lentivirus are described and contrasted. In order for viral vectors to be developed into clinical grade reagents, they must be manufactured and tested to precise regulatory standards. Indeed, clinical lots of viral vectors can be produced in compliance with current Good Manufacturing Practices (cGMPs) regulations using industry accepted manufacturing methodologies, manufacturing controls, and quality systems. The viral vector properties themselves combined with physiological product formulations facilitate long-term storage and direct in vivo administration.

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Section: Recombinant Adenovirus (Rad)mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Viral vectors for in vivo gene transfer in Parkinson's disease: Properties and clinical grade production

Mandel

Bürger

Snyder

2008

Experimental Neurology

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“…The differentiation media consisted of FCS, (10%),P/S; IL-la, (100 pg/ml); IL-11, (1 ng/ml); LIF, (1 ng/ml), and GDNF, (10 ng/ml) in 45%DMEM:45% Hamm's F12. At varying time points (1,2,3,4,5,6,7, and 8 hours after incubation, data not shown) the differentiation media was washed off and the cells were plated onto PLL-coated 48-well plates. After 6 days in complete media without cytokines, the cultures were fixed and stained as described above.…”

Section: Preparation Of Cells For Transplantationmentioning

confidence: 99%

Cytokine Induction of Dopamine Neurons from Progenitor Cells

Carvey¹

2000

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“…1 Among the many gene delivery vectors, replication defective adenoviral vectors have been widely used as an effective tool to express therapeutic genes in the brains of rodent models of human neurological diseases including cerebral ischemia. [2][3][4][5][6][7][8] For example, adenoviral vector mediated expression of antiapoptotic genes, such as XIAP, 9,10 Bcl-2 11 or trophic factors, such as glial derived trophic factors, 12,13 conferred potent neuroprotection against cerebral ischemia.…”

Section: Introductionmentioning

confidence: 99%

“…[3][4][5]8,15 Adenoviruses contain a 36 kb double-stranded DNA genome packaged into an icosahedral capsid. Deletion of the E1 and E3 regions of the viral genome renders the adenovirus unable to selfreplicate and minimizes immunogenecity to the host cells, thereby reducing potential toxicity derived from the viral backbone.…”

Section: Introductionmentioning

confidence: 99%

A novel adenoviral vector which mediates hypoxia-inducible gene expression selectively in neurons

2005

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Selective gene expression in neurons is still a challenge. We have developed several expression vectors using a combination of neuron restrictive silencer elements (NRSEs), hypoxia responsive elements (HREs) and CMV minimal promoter (CMVmp). These elements were packaged into replication defective adenovirus to target gene expression selectively in neurons in a hypoxia-regulated manner. Neuronal selectivity and responsiveness to hypoxia of these novel constructs were determined empirically in both neural cell lines and primary cerebellar granule neurons (CGNs). The construct p5HRE-3NRSE exhibited not only the highest level of reporter gene expression in neuronal cells but also in an oxygen concentration-dependent manner when compared with all other constructs. As expected, this construct did not elicit reporter gene expression in non-neuronal cells including human HEK293A and HT29 cells, rat NRK cells, mouse 3T6 cells and 3T3 L1 cells. This construct was packaged into a replication defective adenoviral vector (Ad/5HRE-3NRSE) to determine neuron-selective and hypoxia-inducible gene expression in cultured mouse postmitotic primary CGNs and differentiated human NT2 neurons (NT2/Ns). Remarkably, in response to hypoxia, Ad/5HRE-3NRSE showed strong hypoxia-inducible gene expression selectively in neurons (12-fold induction in CGNs and 22-fold in NT2/Ns), but not in glial cells. Taken together, this vector with restricted gene expression to neurons under the regulation of hypoxia will be a useful tool for investigations of mechanisms of neuronal damage caused by ischemic insult.

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Adenovirus in the brain: recent advances of gene therapy for neurodegenerative diseases

Cited by 86 publications

References 51 publications

Viral vectors for in vivo gene transfer in Parkinson's disease: Properties and clinical grade production

Viral vectors for in vivo gene transfer in Parkinson's disease: Properties and clinical grade production

Cytokine Induction of Dopamine Neurons from Progenitor Cells

A novel adenoviral vector which mediates hypoxia-inducible gene expression selectively in neurons

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