A generalizable pre-clinical research approach for orphan disease therapy

Orphanet Journal of Rare Diseases

Volumn 7, Issue 1, 2012, Pages

  Beaulieu, Chandree L ^a   Samuels, Mark E ^b   Ekins, Sean ^c   McMaster, Christopher R ^d   Edwards, Aled M ^e   Krainer, Adrian R ^f   Hicks, Geoffrey G ^g   Frey, Brendan J ^e   Boycott, Kym M ^a   MacKenzie, Alex E ^a  

^a CHILDREN S HOSPITAL OF EASTERN ONTARIO   (Canada)

^b UNIVERSITY OF MONTREAL   (Canada)

^c Collaborations in Chemistry ^*  (United States)

^d DALHOUSIE UNIVERSITY   (Canada)

^e UNIVERSITY OF TORONTO   (Canada)

^f Howard Hughes Medical Institute Cold Spring Harbor Laboratory Cold Spring Harbor   (United States)

^g UNIVERSITY OF MANITOBA   (Canada)

Author keywords

Generalizable screening methods; Orphan disease therapy; Preclinical drug development; Translational toolbox

Indexed keywords

ATALUREN; OLIGONUCLEOTIDE; RNA;

BIOINFORMATICS; CHEMICAL INFORMATICS; CLINICAL RESEARCH; CODON; CYSTIC FIBROSIS; DISEASES; DRUG INDUSTRY; FOOD AND DRUG ADMINISTRATION; GENE ACTIVITY; GENETIC DISORDER; HUMAN; LOSS OF FUNCTION MUTATION; MEDICAL LITERATURE; NONHUMAN; ORPHAN DISEASE; PREMATURE TERMINATION CODON; REVIEW; RNA SPLICING; RNA STABILITY; TRANSCRIPTOMICS;

COMPUTATIONAL BIOLOGY; DRUG DISCOVERY; GENETIC DISEASES, INBORN; HUMANS; RARE DISEASES; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84862173050     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-7-39     Document Type: Review

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