Bcl-2 expression by retrograde transport of adenoviral vectors with Cre-loxP recombination system in motor neurons of mutant SOD1 transgenic mice

Yamashita, Satoshi; Mita, Shuji; Arima, Toshiyuki; Maeda, Yasushi; Nishida, Yoshiaki; Murakami, Tatsufumi; Okado, Haruo; Uchino, Makoto

doi:10.1038/sj.gt.3301479

Cited by 23 publications

(12 citation statements)

References 37 publications

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“…In primary embryonic motor neurons, inhibition of pro-apoptotic effects was demonstrated after glutamate insult, whereas in DRG cells the same effects were achieved following a glucose insult. Other anti-apoptotic molecules, Bcl-2 and Bcl-xL, have been shown to prevent apoptosis in vitro and in vivo [Yamashita et al, 2001;Matsuoka et al, 2002;Yamashita et al, 2003;Garrity-Moses et al, 2005a], making them attractive targets for gene-based neuroprotection.…”

Section: Rationale For Therapeutic Interventionmentioning

confidence: 99%

Gene Therapy for Peripheral Nervous System Diseases

Federici

Boulis²

2007

CGT

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Peripheral nerve diseases, also known as peripheral neuropathies, affect 15-20 million of Americans and diabetic neuropathy is the most common condition. Currently, the treatment of peripheral neuropathies is more focused on managing pain rather than providing permissive conditions for regeneration. Despite advances in microsurgical techniques, including nerve grafting and reanastomosis, axonal regeneration after peripheral nerve injury remains suboptimal. Also, no satisfactory treatments are available at this time for peripheral neurodegeneration occurring in motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). Peripheral nerves have the inherent capacity of regeneration. Gene therapy strategies focused on neuroprotection may help optimizing axonal regrowth. A better understanding of the cellular and molecular events involved in axonal degeneration and regeneration have helped researchers to identify targets for intervention. This review summarizes the current state on the clinical experience as well as gene therapy strategies for peripheral neuropathies, including MND, peripheral nerve injury, neuropathic pain, and diabetic neuropathy.

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Section: Rationale For Therapeutic Interventionmentioning

confidence: 99%

Gene Therapy for Peripheral Nervous System Diseases

Federici

Boulis²

2007

CGT

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“…Despite the fact that some investigators have described abnormal axonal transport in the SOD1 mutant mouse,139 other groups have achieved successful delivery using this method 131, 157, 212. Both routes of administration, intramuscular and intraneural, have been used extensively in animal models.…”

Section: Motor Neuron Gene Deliverymentioning

confidence: 99%

Gene‐based treatment of motor neuron diseases

Federici¹,

Boulis²

2005

Muscle and Nerve

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Motor neuron diseases (MND), such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), are progressive neurodegenerative diseases that share the common characteristic of upper and/or lower motor neuron degeneration. Therapeutic strategies for MND are designed to confer neuroprotection, using trophic factors, anti-apoptotic proteins, as well as antioxidants and anti-excitotoxicity agents. Although a large number of therapeutic clinical trials have been attempted, none has been shown satisfactory for MND at this time. A variety of strategies have emerged for motor neuron gene transfer. Application of these approaches has yielded therapeutic results in cell culture and animal models, including the SOD1 models of ALS. In this study we describe the gene-based treatment of MND in general, examining the potential viral vector candidates, gene delivery strategies, and main therapeutic approaches currently attempted. Finally, we discuss future directions and potential strategies for more effective motor neuron gene delivery and clinical translation.

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“…[4][5][6][7][8] CGP 3466B (dibenzo{b,f}oxepin-10-ylmethyl-methyl-prop-2-ynyl-amine), a tricyclic propargylamine structurally related to (-)-deprenyl, was found to inhibit apoptosis in a wide variety of in vitro and in vivo models. [9][10][11][12] The anti-apoptotic effect is probably mediated by the binding of CGP 3466B to glyceraldehyde-3-phosphate dehydrogenase (GAPDH), a ubiquitous enzyme with glycolytic functions that also plays a role in certain apoptotic pathways.…”

Section: Introductionmentioning

confidence: 99%