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Human Genetics, ISSN 0340-6717, 11/2011, Volume 130, Issue 5, pp. 583 - 605
Dominant negative genetic disorders, in which a mutant allele of a gene causes disease in the presence of a second, normal copy, have been challenging since... 
Human Genetics | Gene Function | Molecular Medicine | Biomedicine | Metabolic Diseases | SMALL INTERFERING RNA | DOUBLE-STRANDED-RNA | DYSTROPHY PROTEIN-KINASE | SICKLE-CELL-ANEMIA | GENETICS & HEREDITY | CENTRAL-NERVOUS-SYSTEM | ALPHA-SYNUCLEIN | BLOOD-BRAIN-BARRIER | HUNTINGTONS-DISEASE | MAMMALIAN-CELLS | MYOTONIC-DYSTROPHY | Parkinson Disease - therapy | Muscular Dystrophies - therapy | Humans | Anemia, Sickle Cell - drug therapy | Parkinson Disease - drug therapy | Spinocerebellar Ataxias - therapy | Genetic Diseases, Inborn - therapy | Molecular Targeted Therapy | Muscular Dystrophies - genetics | Amyotrophic Lateral Sclerosis - drug therapy | Huntington Disease - therapy | Neoplasms - therapy | Genes, Dominant | Neoplasms - genetics | RNA Interference | Spinocerebellar Ataxias - drug therapy | Huntington Disease - drug therapy | Spinocerebellar Ataxias - genetics | Amyotrophic Lateral Sclerosis - therapy | Amyotrophic Lateral Sclerosis - genetics | Alzheimer Disease - therapy | Muscular Dystrophies - drug therapy | Alzheimer Disease - drug therapy | Rats | Clinical Trials as Topic | Parkinson Disease - genetics | Neoplasms - drug therapy | Anemia, Sickle Cell - therapy | Point Mutation | Animals | Huntington Disease - genetics | Mice | Alzheimer Disease - genetics | Genetic Diseases, Inborn - drug therapy | Anemia, Sickle Cell - genetics | Genetic Therapy - methods | Enzymes | Gene mutations | Gene therapy | Catalytic RNA | Health aspects | Genes | Drugs | Parkinson's disease | Neurodegenerative diseases | Tumor cells | RNA-mediated interference | Amyotrophic lateral sclerosis | Antisense RNA | Drug delivery | Single-nucleotide polymorphism | Huntington's disease | Muscular dystrophy | Mutation | Ribozymes | Alzheimer's disease | Movement disorders | Cancer | Pharmaceuticals
Journal Article
1997, Advances in pharmacology, ISBN 0120329417, Volume 40., xvi, 508
Book
Advanced Drug Delivery Reviews, ISSN 0169-409X, 06/2015, Volume 87, pp. 90 - 103
Journal Article