Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease

Barry J. Byrne; Shelley Collins; Cathryn Mah; Barbara Smith; Thomas Conlon; Danny Martin; Manuela Corti; Brian Cleaver; Saleem Islam; Lee Ann Lawson

doi:10.1089/humc.2014.2514

Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease

Barry J. Byrne, Shelley Collins, Cathryn Mah, Barbara Smith, Thomas Conlon, Danny Martin, Manuela Corti, Brian Cleaver, Saleem Islam, Lee Ann Lawson

Research output: Contribution to journal › Article › peer-review

34 Citations (Scopus)

Original language	English
Pages (from-to)	134-163
Number of pages	30
Journal	Human Gene Therapy Clinical Development
Volume	25
Issue number	3
DOIs	https://doi.org/10.1089/humc.2014.2514
Publication status	Published - 1 Sept 2014
Externally published	Yes

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10.1089/humc.2014.2514

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Byrne, B. J., Collins, S., Mah, C., Smith, B., Conlon, T., Martin, D., Corti, M., Cleaver, B., Islam, S., & Lawson, L. A. (2014). Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease. Human Gene Therapy Clinical Development, 25(3), 134-163. https://doi.org/10.1089/humc.2014.2514

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title = "Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease",

author = "Byrne, {Barry J.} and Shelley Collins and Cathryn Mah and Barbara Smith and Thomas Conlon and Danny Martin and Manuela Corti and Brian Cleaver and Saleem Islam and Lawson, {Lee Ann}",

year = "2014",

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Byrne, BJ, Collins, S, Mah, C, Smith, B, Conlon, T, Martin, D, Corti, M, Cleaver, B, Islam, S & Lawson, LA 2014, 'Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease', Human Gene Therapy Clinical Development, vol. 25, no. 3, pp. 134-163. https://doi.org/10.1089/humc.2014.2514

Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease. / Byrne, Barry J.; Collins, Shelley; Mah, Cathryn et al.
In: Human Gene Therapy Clinical Development, Vol. 25, No. 3, 01.09.2014, p. 134-163.

Research output: Contribution to journal › Article › peer-review

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T1 - Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease

AU - Byrne, Barry J.

AU - Collins, Shelley

AU - Mah, Cathryn

AU - Smith, Barbara

AU - Conlon, Thomas

AU - Martin, Danny

AU - Corti, Manuela

AU - Cleaver, Brian

AU - Islam, Saleem

AU - Lawson, Lee Ann

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SN - 2324-8637

VL - 25

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JO - Human Gene Therapy Clinical Development

JF - Human Gene Therapy Clinical Development

IS - 3

ER -

Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with pompe disease

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