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Aav-based gene therapy approaches for the treatment of canavan disease

✍ Scribed by Jonquieres, G.; Mersmann, N.; Teahan, O.; Klugmann, C.B.; Harasta, A.E.; Lutz, B.; Housley, G.D.; Klugmann, M.


Book ID
122468969
Publisher
Informa plc
Year
2013
Tongue
English
Weight
42 KB
Volume
15
Category
Article
ISSN
1465-3249

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Gene therapy, which is the deliberate transfer of DNA for therapeutic purposes, can be defined as the in situ production of a therapeutical material within the diseased cells. The eye is an ideal target for gene therapy, as it is confined to a small space, separated by the retina-blood barrier from