6533b837fe1ef96bd12a331e

RESEARCH PRODUCT

In vivo strategies for drug discovery in myotonic dystrophy disorders.

Manuel Pérez-alonsoIrma Garcia-alcoverArturo López CastelRuben Artero

subject

Drugbusiness.industryDrug discoverymedia_common.quotation_subjectDiseasePharmacologyBioinformaticsmedicine.diseaseMyotonic dystrophyDisease Models AnimalIn vivoDrug DiscoveryMolecular MedicineMedicineAnimalsHumansMyotonic Dystrophybusinessmedia_common

description

Myotonic dystrophy (DM) is a complex neuromuscular genetic disease for which there is currently no valid therapy. The recent development of non-mammal animal models opened up the possibility of performing drug discovery in vivo, using as screening readout phenotypes with underlying molecular parallels to the disease. In this review we discuss the state of the art technologies already used in large scale drug screening and provide guidance for further development of novel technologies.

10.1016/j.ddtec.2012.02.001https://pubmed.ncbi.nlm.nih.gov/24050236