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Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1125 - 1131
Duchenne muscular dystrophy (DMD) has been a major target for gene therapy development for nearly 30 years. DMD is among the most common genetic diseases, and... 
gene therapy | muscular dystrophy | dystrophin | microdystrophin | AAV | mdx mice | MOUSE SKELETAL-MUSCLE | MEDICINE, RESEARCH & EXPERIMENTAL | SYSTEMIC DELIVERY | CANINE MODEL | TRANSGENIC MDX MICE | ADENOASSOCIATED VIRUS VECTOR | ADENOVIRAL VECTORS | FULL-LENGTH | INTRAVENOUS-INJECTION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IN-VIVO | GENETICS & HEREDITY | EXPRESSION | Dependovirus - genetics | Muscular Dystrophy, Animal - genetics | History, 21st Century | Humans | Muscle, Skeletal - metabolism | Muscular Dystrophy, Animal - pathology | Mice, Inbred mdx | Muscular Dystrophy, Animal - metabolism | Protein Domains | Muscular Dystrophy, Animal - therapy | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Dependovirus - metabolism | Genetic Vectors - chemistry | History, 20th Century | Genetic Vectors - metabolism | Genetic Therapy - history | Muscular Dystrophy, Duchenne - pathology | Dystrophin - chemistry | Genetic Therapy - trends | Animals | Dystrophin - genetics | Dogs | Mice | Muscular Dystrophy, Duchenne - metabolism | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | Mutation | Genetic Therapy - methods | Genetic disorders | Clinical trials | FDA approval | Vectors (Biology) | Muscular dystrophy | Design | Proteins | Genetic counseling | Duchenne's muscular dystrophy | Stem cells | Dystrophy | Gene therapy | Dystrophin | Review
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1132 - 1141
Prior to the first successful bone marrow transplant in 1968, patients born with severe combined immunodeficiency (SCID) invariably died. Today, with a... 
immunodeficiencies | gene therapy | retrovirus | CD40 LIGAND DEFICIENCY | MEDICINE, RESEARCH & EXPERIMENTAL | CHRONIC GRANULOMATOUS-DISEASE | FOAMY VIRUS VECTOR | LENTIVIRAL VECTORS | MURINE MODEL | BONE-MARROW TRANSPLANTATION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | VECTOR-MEDIATED CORRECTION | HEMATOPOIETIC STEM-CELLS | GENETICS & HEREDITY | INSERTIONAL MUTAGENESIS | ADENOSINE-DEAMINASE DEFICIENCY | Immunologic Deficiency Syndromes - pathology | History, 21st Century | Lentivirus - immunology | Humans | Transplantation, Homologous | Lentivirus - genetics | Gammaretrovirus - immunology | Severe Combined Immunodeficiency - pathology | Adenosine Deaminase - deficiency | Immunologic Deficiency Syndromes - immunology | Immunologic Deficiency Syndromes - therapy | Gene Expression | Genetic Vectors - chemistry | Genetic Vectors - immunology | Adenosine Deaminase - genetics | History, 20th Century | Severe Combined Immunodeficiency - therapy | Genetic Therapy - history | Clinical Trials as Topic | Severe Combined Immunodeficiency - immunology | Severe Combined Immunodeficiency - genetics | Gammaretrovirus - genetics | Immunologic Deficiency Syndromes - genetics | Hematopoietic Stem Cell Transplantation - methods | Adenosine Deaminase - immunology | Genetic Therapy - methods | Severe combined immunodeficiency | Disease | Advantages | Syngeneic grafts | Bone marrow transplantation | Metabolism | Medical screening | Patients | Vectors (Biology) | Progenitor cells | Hemopoiesis | Defects | Immunology | Mutagenesis | Metabolites | Lymphocytes | Stem cells | Bone marrow | Gene therapy | Review
Journal Article