Data Bases and Tools

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Advisory Committee for Therapeutics (ACT) toolkit

Published by UNEW

AdviceClinical DevelopmentPreclinical ResearchResearch and Drug DevelopmentToolkits for patientsTranslational ResearchTREAT-NMD

The Advisory Committee for Therapeutics (ACT) toolkit provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities. An ACT can provide independent and objective advice to industry and academia, on a real-life therapeutic pathway of drug development programmes. An ACT review can help an applicant to position a candidate compound along a realistic and well-informed pathway to clinical trial and eventual
registration.

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Expression quantitative trait loci: present and future

Published by Philos Trans R Soc Lond B Biol Sci.

eQTLTarget ValidationTool

One of the most prominent directions has been the extensive set of studies on expression quantitative trait loci (eQTLs), namely, the discovery of genetic variants that explain variation in gene expression levels. Such studies have offered promise not just for the characterization of functional sequence variation but also for the understanding of basic processes of gene regulation and interpretation of genome-wide association studies. In this review, we discuss some of the key directions of eQTL research and its implications.

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