Drug discovery and development for rare genetic disorders

American Journal of Medical Genetics, Part A

Volumn 173, Issue 9, 2017, Pages 2307-2322

  Sun, Wei ^a   Zheng, Wei ^a   Simeonov, Anton ^a  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

biologics; drug discovery; drug repurposing; genetic disorders; rare diseases; small molecules

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ADENOVIRUS VECTOR; AGALSIDASE BETA; ALIPOGENE TIPARVOVEC; ANTISENSE OLIGONUCLEOTIDE; BIOLOGICAL MARKER; BISPHOSPHONIC ACID DERIVATIVE; CLONAZEPAM; FENRETINIDE; GROWTH HORMONE; HYDROXYUREA; IVACAFTOR; LUMACAFTOR; ORPHAN DRUG; PROTEIN FARNESYLTRANSFERASE INHIBITOR; RETIGABINE; RILUZOLE; SILDENAFIL; TETRABENAZINE; VORINOSTAT;

AMYOTROPHIC LATERAL SCLEROSIS; CUTANEOUS T CELL LYMPHOMA; CYSTIC FIBROSIS; DISEASE MODEL; DRUG EFFECT; DRUG EFFICACY; DRUG REPOSITIONING; DRUG RESEARCH; ENZYME REPLACEMENT; ERECTILE DYSFUNCTION; FABRY DISEASE; FUNDING; GENE THERAPY; GENETIC DISORDER; GLIOBLASTOMA; HAPPY PUPPET SYNDROME; HIGH THROUGHPUT SCREENING; HUMAN; HUNTINGTON CHOREA; HYPERLIPOPROTEINEMIA TYPE 1; INDUCED PLURIPOTENT STEM CELL; NON PROFIT ORGANIZATION; NON SMALL CELL LUNG CANCER; NONHUMAN; OSTEOGENESIS IMPERFECTA; PHARMACOGENETICS; PHENOTYPE; PRIORITY JOURNAL; PROGERIA; PULMONARY HYPERTENSION; RARE DISEASE; RETINA DEGENERATION; REVIEW; SICKLE CELL ANEMIA; STEM CELL TRANSPLANTATION; WHOLE GENOME SEQUENCING; ANIMAL; DRUG DEVELOPMENT; MOLECULAR LIBRARY; TRENDS; UNITED STATES;

ANIMALS; DISEASE MODELS, ANIMAL; DRUG DISCOVERY; GENETIC DISEASES, INBORN; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; RARE DISEASES; SMALL MOLECULE LIBRARIES; UNITED STATES;

EID: 85025437291     PISSN: 15524825     EISSN: 15524833     Source Type: Journal    
DOI: 10.1002/ajmg.a.38326     Document Type: Review

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