Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy

Norma B Romero; Serge Braun; Olivier Benveniste; France Leturcq; Jean-Yves Hogrel; Glenn E Morris; Annie Barois; Bruno Eymard; Christine Payan; Véronique Ortega; Anne-Laure Boch; Lise Lejean; Christine Thioudellet; Brigitte Mourot; Christophe Escot; Aurore Choquel; Dominique Recan; Jean-Claude Kaplan; George Dickson; David Klatzmann; Valérie Molinier-Frenckel; Jean-Gérard Guillet; Patrick Squiban; Serge Herson; Michel Fardeau

doi:10.1089/hum.2004.15.1065

Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy

Norma B Romero, Serge Braun, Olivier Benveniste, France Leturcq, Jean-Yves Hogrel, Glenn E Morris, Annie Barois, Bruno Eymard, Christine Payan, Véronique Ortega, Anne-Laure Boch, Lise Lejean, Christine Thioudellet, Brigitte Mourot, Christophe Escot, Aurore Choquel, Dominique Recan, Jean-Claude Kaplan, George Dickson, David KlatzmannValérie Molinier-Frenckel, Jean-Gérard Guillet, Patrick Squiban, Serge Herson, Michel Fardeau

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

Original language	English
Pages (from-to)	1065-76
Number of pages	12
Journal	Human Gene Therapy
Volume	15
Issue number	11
DOIs	https://doi.org/10.1089/hum.2004.15.1065
Publication status	Published - 2004

Keywords

Adolescent
Adult
Biopsy
Cohort Studies
Dystrophin
Gene Therapy
Gene Transfer Techniques
Genetic Vectors
Histocompatibility Testing
Humans
Immunohistochemistry
Middle Aged
Models, Genetic
Muscle, Skeletal
Muscles
Muscular Dystrophy, Duchenne
Plasmids
Promoter Regions, Genetic
RNA, Messenger
Reverse Transcriptase Polymerase Chain Reaction
Time Factors

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.1089/hum.2004.15.1065

Cite this

Romero, N. B., Braun, S., Benveniste, O., Leturcq, F., Hogrel, J.-Y., Morris, G. E., Barois, A., Eymard, B., Payan, C., Ortega, V., Boch, A.-L., Lejean, L., Thioudellet, C., Mourot, B., Escot, C., Choquel, A., Recan, D., Kaplan, J.-C., Dickson, G., ... Fardeau, M. (2004). Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Human Gene Therapy, 15(11), 1065-76. https://doi.org/10.1089/hum.2004.15.1065

@article{815bd0d8e94241ad889c166a1aa81563,

title = "Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy",

keywords = "Adolescent, Adult, Biopsy, Cohort Studies, Dystrophin, Gene Therapy, Gene Transfer Techniques, Genetic Vectors, Histocompatibility Testing, Humans, Immunohistochemistry, Middle Aged, Models, Genetic, Muscle, Skeletal, Muscles, Muscular Dystrophy, Duchenne, Plasmids, Promoter Regions, Genetic, RNA, Messenger, Reverse Transcriptase Polymerase Chain Reaction, Time Factors",

author = "Romero, {Norma B} and Serge Braun and Olivier Benveniste and France Leturcq and Jean-Yves Hogrel and Morris, {Glenn E} and Annie Barois and Bruno Eymard and Christine Payan and V{\'e}ronique Ortega and Anne-Laure Boch and Lise Lejean and Christine Thioudellet and Brigitte Mourot and Christophe Escot and Aurore Choquel and Dominique Recan and Jean-Claude Kaplan and George Dickson and David Klatzmann and Val{\'e}rie Molinier-Frenckel and Jean-G{\'e}rard Guillet and Patrick Squiban and Serge Herson and Michel Fardeau",

year = "2004",

doi = "10.1089/hum.2004.15.1065",

language = "English",

volume = "15",

pages = "1065--76",

journal = "Human Gene Therapy",

issn = "1043-0342",

publisher = "Mary Ann Liebert Inc.",

number = "11",

}

Romero, NB, Braun, S, Benveniste, O, Leturcq, F, Hogrel, J-Y, Morris, GE, Barois, A, Eymard, B, Payan, C, Ortega, V, Boch, A-L, Lejean, L, Thioudellet, C, Mourot, B, Escot, C, Choquel, A, Recan, D, Kaplan, J-C, Dickson, G, Klatzmann, D, Molinier-Frenckel, V, Guillet, J-G, Squiban, P, Herson, S & Fardeau, M 2004, 'Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy', Human Gene Therapy, vol. 15, no. 11, pp. 1065-76. https://doi.org/10.1089/hum.2004.15.1065

TY - JOUR

T1 - Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy

AU - Romero, Norma B

AU - Braun, Serge

AU - Benveniste, Olivier

AU - Leturcq, France

AU - Hogrel, Jean-Yves

AU - Morris, Glenn E

AU - Barois, Annie

AU - Eymard, Bruno

AU - Payan, Christine

AU - Ortega, Véronique

AU - Boch, Anne-Laure

AU - Lejean, Lise

AU - Thioudellet, Christine

AU - Mourot, Brigitte

AU - Escot, Christophe

AU - Choquel, Aurore

AU - Recan, Dominique

AU - Kaplan, Jean-Claude

AU - Dickson, George

AU - Klatzmann, David

AU - Molinier-Frenckel, Valérie

AU - Guillet, Jean-Gérard

AU - Squiban, Patrick

AU - Herson, Serge

AU - Fardeau, Michel

PY - 2004

Y1 - 2004

KW - Adolescent

KW - Adult

KW - Biopsy

KW - Cohort Studies

KW - Dystrophin

KW - Gene Therapy

KW - Gene Transfer Techniques

KW - Genetic Vectors

KW - Histocompatibility Testing

KW - Humans

KW - Immunohistochemistry

KW - Middle Aged

KW - Models, Genetic

KW - Muscle, Skeletal

KW - Muscles

KW - Muscular Dystrophy, Duchenne

KW - Plasmids

KW - Promoter Regions, Genetic

KW - RNA, Messenger

KW - Reverse Transcriptase Polymerase Chain Reaction

KW - Time Factors

U2 - 10.1089/hum.2004.15.1065

DO - 10.1089/hum.2004.15.1065

M3 - Article

C2 - 15610607

SN - 1043-0342

VL - 15

SP - 1065

EP - 1076

JO - Human Gene Therapy

JF - Human Gene Therapy

IS - 11

ER -