Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
Bowles, Dawn E
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy Feb 2012 - 443-55 p. digital
Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
1525-0024
10.1038/mt.2011.237 doi
Amino Acid Sequence
Animals
Antibodies, Neutralizing--immunology
Antibodies, Viral--immunology
Capsid Proteins--chemistry
Cell Line
Child
Child, Preschool
Dependovirus--genetics
Dystrophin--genetics
Genetic Therapy
Genetic Vectors--administration & dosage
Humans
Male
Mice
Mice, Inbred BALB C
Mice, Inbred C57BL
Models, Molecular
Molecular Sequence Data
Muscle, Skeletal--metabolism
Muscular Dystrophy, Duchenne--genetics
Protein Conformation
Sequence Alignment
T-Lymphocytes--immunology
Transduction, Genetic
Viral Tropism
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. [electronic resource] - Molecular therapy : the journal of the American Society of Gene Therapy Feb 2012 - 443-55 p. digital
Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
1525-0024
10.1038/mt.2011.237 doi
Amino Acid Sequence
Animals
Antibodies, Neutralizing--immunology
Antibodies, Viral--immunology
Capsid Proteins--chemistry
Cell Line
Child
Child, Preschool
Dependovirus--genetics
Dystrophin--genetics
Genetic Therapy
Genetic Vectors--administration & dosage
Humans
Male
Mice
Mice, Inbred BALB C
Mice, Inbred C57BL
Models, Molecular
Molecular Sequence Data
Muscle, Skeletal--metabolism
Muscular Dystrophy, Duchenne--genetics
Protein Conformation
Sequence Alignment
T-Lymphocytes--immunology
Transduction, Genetic
Viral Tropism