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  1. Article

    Open Access

    Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

    Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharma...

    Ole B Suhr, Teresa Coelho, Juan Buades, Jean Pouget in Orphanet Journal of Rare Diseases (2015)

  2. Article

    Open Access

    In vivo quantification of formulated and chemically modified small interfering RNA by heating-in-Triton quantitative reverse transcription polymerase chain reaction (HIT qRT-PCR)

    While increasing numbers of small interfering RNA (siRNA) therapeutics enter into clinical trials, the quantification of siRNA from clinical samples for pharmacokinetic studies remains a challenge. This challe...

    Yosef Landesman, Nenad Svrzikapa, Armand Cognetta III, Xuemei Zhang in Silence (2010)

  3. Article

    Annotation of the Drosophila melanogastereuchromatic genome: a systematic review

    The recent completion of the Drosophila melanogaster genomic sequence to high quality and the availability of a greatly expanded set of Drosophila cDNA sequences, aligning to 78% of the predicted euchromatic gene...

    Sima Misra, Madeline A Crosby, Christopher J Mungall, Beverley B Matthews in Genome Biology (2002)