HD Insights: Volume 3

HD Insights: Volume 3

HD Insights Volume 3 (PDF)

November 1st, 2012 Heather Hare

An Interview with Dr. Nancy Wexler: Discovering the Huntington Disease Gene

September 1st, 2012 rtullio By: Kristin Darwin One morning in 1968, Dr. Nancy Wexler’s mother, Leonore Wexler, had jury duty in downtown Los Angeles. As Leonore crossed the street on the way to the courthouse, a policeman yelled to her, “How can you be drunk so early in the morning?” Leonore realized that she had been staggering – an
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Meet the Company

September 1st, 2012 rtullio         VITAL SIGNS NAME: Isis Pharmaceuticals HEADQUARTERS: Carlsbad, California STOCK PRICE AS OF 8/21/12: $13.67 52 WEEK RANGE: $6.25 – $14.05 MARKET CAPITALIZATION AS OF 8/21/12: $1.4 billion EMPLOYEES: Approximately 340 Isis Pharmaceuticals is a biotechnology company that develops therapeutic agents that target and modulate RNA. Isis’s primary platform is exploiting antisense
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Research Around the World: Chile

September 1st, 2012 rtullio Alleviating Secretory Pathway Stress in Huntington Disease By: Rene Vidal, PhD and Claudio Hetz, PhD Many clinical trials that use drugs validated in mouse models of HD have failed to alleviate disease progression in humans. Preclinical studies have been performed in transgenic mice of pure genetic backgrounds that overexpress high levels of truncated forms of
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Highlights from the CHDI HD Therapeutics Conference

September 1st, 2012 rtullio By: Lise Munsie, PhD At the 7th Annual CHDI HD Therapeutics Conference in Palm Springs, California, scientists from academia and industry convened to discuss the current state of drug development research aimed at slowing the progression of Huntington disease (HD). Robert Pacifici, CHDI’s chief scientific officer, opened the conference. The first session of the meeting
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The Use of Antisense Oligonucleotides for Gene Silencing

September 1st, 2012 rtullio By: Emily Mitchell Sontag, PhD Huntington disease (HD) is caused by a single gene mutation and is therefore a good candidate for therapeutic gene silencing. While many potential HD therapeutic agents focus on ameliorating toxic effects following intracellular production of the mutant huntingtin protein (mHtt), gene silencing would disrupt the production of mHtt and could
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Research Round-Up

September 1st, 2012 rtullio By: Lise Munsie, PhD In the beginning… In the past year, researchers have published on the use of stem cells to model Huntington disease (HD) and on investigations of stem cell use as a treatment for HD. Sadan and colleagues1 induced mesenchymal stem cells into neurotrophic factor– secreting (NTF) cells, then transplanted the NTF cells
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