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

37 Citations (Scopus)

Original language	English (UK)
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

Access to Document

10.1089/humc.2014.2514

Cite this

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

@article{3b6815fc1466424387a8ab87fccfa779,

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",

month = sep,

day = "1",

doi = "10.1089/humc.2014.2514",

language = "English (UK)",

volume = "25",

pages = "134--163",

journal = "Human Gene Therapy Clinical Development",

issn = "2324-8637",

publisher = "Mary Ann Liebert Inc.",

number = "3",

}

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

TY - JOUR

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

PY - 2014/9/1

Y1 - 2014/9/1

UR - https://www.scopus.com/pages/publications/84907271281

U2 - 10.1089/humc.2014.2514

DO - 10.1089/humc.2014.2514

M3 - Article

C2 - 25238277

AN - SCOPUS:84907271281

SN - 2324-8637

VL - 25

SP - 134

EP - 163

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

Access to Document

Other files and links

Cite this