Assays for the identification and prioritization of drug candidates for spinal muscular atrophy

Assay and Drug Development Technologies

Volumn 12, Issue 6, 2014, Pages 315-341

  Cherry, Jonathan J ^a   Kobayashi, Dione T ^b   Lynes, Maureen M ^c,d   Naryshkin, Nikolai N ^e   Tiziano, Francesco Danilo ^f   Zaworski, Phillip G ^g   Rubin, Lee L ^c,d   Jarecki, Jill ^h  

^a PFIZER INC   (United States)

^b Independent Consultant   (United States)

^c Harvard University   (United States)

^d HARVARD STEM CELL INSTITUTE   (United States)

^e PTC THERAPEUTICS   (United States)

^f CATHOLIC UNIVERSITY   (Italy)

^g PharmOptima   (United States)

^h Families of SMA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBODY; ANTISENSE OLIGONUCLEOTIDE; EPITOPE; INDOPROFEN; MESSENGER RNA; SMALL NUCLEAR RIBONUCLEOPROTEIN; SURVIVAL MOTOR NEURON PROTEIN; SMN2 PROTEIN, HUMAN; SURVIVAL MOTOR NEURON PROTEIN 2;

CELL PROLIFERATION; CELL VIABILITY; CELL VIABILITY ASSAY; CHEMOLUMINESCENCE; DRUG DETERMINATION; DRUG IDENTIFICATION; EMBRYONIC STEM CELL; ENZYME LINKED IMMUNOSORBENT ASSAY; EXON; EXTRACTION; FLUORESCENCE RESONANCE ENERGY TRANSFER; GENE EXPRESSION; GENE THERAPY; GENETICS; HIGH THROUGHPUT SCREENING; HUMAN; IMMUNOASSAY; IN VITRO STUDY; MOLECULE; MOTONEURON; NONHUMAN; PATHOPHYSIOLOGY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PLURIPOTENT STEM CELL; POINT MUTATION; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN LOCALIZATION; PROTEIN STABILITY; QUANTITATIVE ANALYSIS; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; REVIEW; RNA SPLICING; SCREENING; SPINAL MUSCULAR ATROPHY; WESTERN BLOTTING; ANIMAL; DRUG DEVELOPMENT; MUSCULAR ATROPHY, SPINAL; PROCEDURES; TRENDS;

ANIMALS; DRUG DISCOVERY; GENETIC THERAPY; HIGH-THROUGHPUT SCREENING ASSAYS; HUMANS; MUSCULAR ATROPHY, SPINAL; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84906718466     PISSN: 1540658X     EISSN: 15578127     Source Type: Journal    
DOI: 10.1089/adt.2014.587     Document Type: Review

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