Literature DB >> 20373247

HD Therapeutics - CHDI Fifth Annual Conference.

Keith T Gagnon1.   

Abstract

The CHDI Fifth Annual HD Therapeutics Conference, held in Palm Springs, CA, included topics covering new therapeutic developments in the field of Huntington's disease (HD). This conference report highlights presentations on biomarkers in HD; emerging topics in drug targeting, such as the lysosomal degradation pathway and target prediction by network-based modeling; understanding phenotype and neuronal circuit dysfunction in animal models; regulation of huntingtin protein expression and function; RNAi and antisense technology to deplete the mutant huntingtin protein; and small-molecule drugs that are progressing quickly through the clinic. Investigational drugs discussed include ALN-HTT (Alnylam Pharmaceuticals Inc/Medtronic Inc), EPI-743 (Edison Pharmaceuticals Inc), LNK-754 (Link Medicine Corp) and pridopidine (NeuroSearch A/S).

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Year:  2010        PMID： 20373247

Source DB:  PubMed          Journal:  IDrugs        ISSN： 1369-7056


  6 in total

Review 1.  Allele-selective inhibition of trinucleotide repeat genes.

Authors:  Masayuki Matsui; David R Corey
Journal:  Drug Discov Today       Date:  2012-01-18       Impact factor: 7.851

Review 2.  Silencing disease genes in the laboratory and the clinic.

Authors:  Jonathan K Watts; David R Corey
Journal:  J Pathol       Date:  2011-11-09       Impact factor: 7.996

Review 3.  The importance of integrating basic and clinical research toward the development of new therapies for Huntington disease.

Authors:  Ignacio Munoz-Sanjuan; Gillian P Bates
Journal:  J Clin Invest       Date:  2011-02-01       Impact factor: 14.808

4.  Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.

Authors:  Jiaxin Hu; Jing Liu; David R Corey
Journal:  Chem Biol       Date:  2010-11-24

Review 5.  Silencing human genetic diseases with oligonucleotide-based therapies.

Authors:  Tamara Martínez; Natalia Wright; Marta López-Fraga; Ana Isabel Jiménez; Covadonga Pañeda
Journal:  Hum Genet       Date:  2013-03-14       Impact factor: 4.132

6.  Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.

Authors:  Keith T Gagnon; Hannah M Pendergraff; Glen F Deleavey; Eric E Swayze; Pierre Potier; John Randolph; Eric B Roesch; Jyoti Chattopadhyaya; Masad J Damha; C Frank Bennett; Christophe Montaillier; Marc Lemaitre; David R Corey
Journal:  Biochemistry       Date:  2010-11-08       Impact factor: 3.162
  6 in total

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