Reducing cellular dysfunction caused by mitochondrial gene...

Organic compounds -- part of the class 532-570 series – Organic compounds – Carbohydrates or derivatives

Reexamination Certificate

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C435S325000, C435S366000, C435S368000

Reexamination Certificate

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10687677

ABSTRACT:
A synthetic, nuclear-encoded ND4 gene was linked to a mitochondrial targeting sequence and a FLAG epitope tag. This fusion construct was inserted into a rAAV vector. The ND4 fusion protein was expressed and imported into the mitochondria of cells harboring a mitochondrial DNA mutation (G11778A), where it restored cellular respiration.

REFERENCES:
patent: 2004/0072774 (2004-04-01), Manfredi et al.
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Guy et al., Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Ann Neurol. 52(5): 534-42, 2002, published online Oct. 11, 2002 (a cover page included).
Guy et al., Gene therapy with the ND4 subunit gene recoded in the universal genetic code reverses a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy (LHON), Neurology, (Apr. 9, 2002) vol. 58, No.7 Supplement 3, pp. A508. print. Meeting Info.: 54th Annual Meeting of the American Academy of Neurology. (one page abstract).
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