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Molecular Therapy, ISSN 1525-0016, 03/2016, Volume 24, Issue 3, pp. 414 - 416
Duchenne muscular dystrophy (DMD) is a fatal disease of skeletal muscle and heart; patients typically die from cardiopulmonary failure with an average life... 
MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | MOUSE MODEL | GENETICS & HEREDITY | RESTORATION | VECTORS | GENOME | Genetic Therapy | Dystrophin - genetics | Muscular Dystrophies - genetics | RNA Editing | Humans | Clustered Regularly Interspaced Short Palindromic Repeats
Journal Article
Human Gene Therapy, ISSN 1043-0342, 11/2017, Volume 28, Issue 11, pp. 113 - 1023
Journal Article
Molecular Therapy, ISSN 1525-0016, 01/2018, Volume 26, Issue 1, pp. 14 - 16
Journal Article
Human Gene Therapy, ISSN 1043-0342, 11/2017, Volume 28, Issue 11, pp. 187 - 1104
Journal Article
Journal Article
Molecular Therapy, ISSN 1525-0016, 01/2015, Volume 23, Issue 1, pp. 4 - 6
Targeted genomic integration typically requires designer nucleases that augment gene targeting efficiencies by creating a double-strand DNA (dsDNA) break at... 
MEDICINE, RESEARCH & EXPERIMENTAL | INTEGRATION | EFFICACY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | FACTOR-IX | IN-VIVO | LIVER | GENETICS & HEREDITY | GENE-THERAPY | HEMOPHILIA | Gene Targeting - methods | Animals | Humans | Factor IX - genetics | Factor IX - metabolism | Female | Male | Hemophilia B - genetics
Journal Article
Journal Article
Cardiovascular Research, ISSN 0008-6363, 05/2015, Volume 108, Issue 1, pp. 4 - 20
Gene therapy is a promising modality for the treatment of inherited and acquired cardiovascular diseases. The identification of the molecular pathways involved... 
Heart failure | Adenylate cyclase | CRISPR | Adeno-associated viral vector | S100A1 | Clinical trials | Cardiovascular disease | miRNA | SERCA2a | Gene therapy | CARDIAC & CARDIOVASCULAR SYSTEMS | ADRENERGIC-RECEPTOR KINASE | ADENOASSOCIATED VIRAL VECTORS | CALCIUM UP-REGULATION |